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GAITHERSBURG, Md.--(BUSINESS WIRE)--Sigma-Tau Pharmaceuticals, Inc., a leader in the development and commercialization of medicines for patients with rare diseases, today announced that the company has changed its name to Leadiant Biosciences, Inc. reaffirming the company’s continued strong commitment to the patient communities it serves. The announcement coincides with Rare Disease Day 2017, a global campaign to raise awareness of rare diseases and improve access to available treatments and medical representation for people, and their caregivers, whose lives are impacted by these conditions. Now in its 10th year, Rare Disease Day is an annual celebration organized by the European Organization for Rare Diseases (EURORDIS) and the National Organization for Rare Disorders (NORD). This year’s theme, With Research, Possibilities Are Limitless, underscores the importance of collaborative research in the drug-development process and recognizes the contributions of patients and families in advocating for increased investment in rare disease research. “Rare Disease Day is the perfect time to unveil our new name and reaffirm our commitment to the study of rare diseases, which has been an integral part of our heritage dating back to 1984 when we became only the fourth company in the world to receive an Orphan Drug Designation in the U.S.,” said Michael Minarich, chief executive officer, Leadiant Biosciences, Inc. “In 2017, Leadiant Biosciences will realize several important and exciting milestones in our product pipeline, as well as continuing multiple ongoing clinical trials.” For more information about the vision, mission and work of Leadiant Biosciences, Inc. visit www.leadiant.com. For more information about Rare Disease Day 2017, visit www.rarediseaseday.org or www.rarediseaseday.us. Leadiant Biosciences, Inc. (formerly Sigma-Tau Pharmaceuticals, Inc.) is a U.S.-based, wholly owned subsidiary of Leadiant Biosciences S.p.A., a research-based pharmaceutical company dedicated to the development and commercialization of medicines for patients with rare diseases. Based in Gaithersburg, Maryland, Leadiant Biosciences, Inc. dedicates considerable scientific and financial resources to the research, development, and distribution of novel and effective therapies that address patient needs and improve quality of life. For more information, visit www.leadiant.com.


News Article | February 28, 2017
Site: globenewswire.com

CAMBRIDGE, Mass., Feb. 28, 2017 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (NASDAQ:EDIT), a leading genome editing company, announced the company has joined forces with 30 million Americans and health care advocates around the world today for Rare Disease Day®. Rare Disease Day is an annual awareness day dedicated to elevating public understanding of rare diseases. This year’s theme focuses on research and the critical role it plays in identifying, diagnosing, and ultimately treating rare diseases. “At Editas Medicine, we have the bold vision to unlock the potential of CRISPR to design and develop genome editing therapies for patients suffering from genetically-defined and genetically-treatable diseases, including rare diseases,” said Katrine Bosley, President and Chief Executive Officer of Editas Medicine. “It’s a privilege to join organizations like EURORDIS and NORD, as well as our colleagues within the industry, to raise awareness of the importance of research, funding, and partnerships for the millions of people living with rare diseases across the world and their families.” CRISPR is a dynamic, versatile tool that can be programmed to target specific stretches of genetic code and edit DNA at precise locations in the human genome. The technology allows researchers to permanently modify genes and has the potential to create medicines with a durable treatment effect following a single dose. Editas Medicine is currently focused on using its CRISPR technology to treat diseases for which there are few or no available treatments, including a rare inherited eye disease called Leber congenital amaurosis type 10, or LCA10, that appears at birth or in the first few months of life and leads to significant vision loss. According to the National Institutes of Health (NIH), nearly one in 10 Americans lives with a rare disease—collectively affecting 30 million people—and nearly half of these patients are children. There are approximately 6,000 rare diseases, 95 percent of which have no FDA-approved therapies. Rare Disease Day was established in Europe in 2008 by EURORDIS, the organization representing rare disease patients in Europe, and is now observed in more than 80 nations. Rare Disease Day is sponsored in the U.S. by the National Organization for Rare Disorders (NORD)®, the largest and leading independent, nonprofit organization committed to the identification, treatment, and cure of rare diseases. About Editas Medicine Editas Medicine is a leading genome editing company dedicated to treating patients with genetically-defined diseases by correcting their disease-causing genes. The company was founded by world leaders in genome editing, and its mission is to translate the promise of genome editing science into a broad class of transformative genomic medicines to benefit the greatest number of patients. Forward-Looking Statements This press release contains forward-looking statements and information within the meaning of the Private Securities Litigation Reform Act of 1995. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "target," "should," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption "Risk Factors" included in the Company's most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.


Rare Diseases affect 30 million people in the United States and 350 million people globally. Despite this size, it lacks a strong unified voice as only 15% of rare diseases have organizations or foundations providing support or driving research, halting the progress that could be made. Mediaplanet’s cross-platform edition of “Rare Diseases” will call upon researchers, universities, students, companies, policy makers, and clinicians to do more research and to make readers aware of the importance of advocating for the rare disease community. Sam Buck, a child fighting VWM, graces the cover of the print publication. Through an exclusive interview with Mediaplanet, his mother Allyson opens up about her families struggle with rare diseases “The pediatrician told us that there’s no treatment, no cure- there’s nothing” Allyson recalls. Allyson shares how to make the best of such a difficult situation, and how to answer an unlucky diagnosis with rare courage. "It does get easier” Allyson shares. The print component of “Rare Diseases” is distributed within the weekend edition of USA Today with a circulation of approximately 750,000 copies and an estimated readership of 1.5 million. The digital component is distributed nationally through a vast social media strategy and across a network of top news sites and partner outlets. To explore the digital version of the campaign, click here. This edition of “Rare Diseases” was made possible with the support of The Buck Family, NORD (National Organization for Rare Disorders), NIH office of Rare Disease Research, Global Genes, C1 Consulting, Alexion Pharmaceuticals, Aegerion Pharmaceuticals, Incyte, Versartis, Fibrocell, Amicus Therapeutics, AmeriSourceBergen, and Illumina. About Mediaplanet Mediaplanet is the leading independent publisher of content marketing campaigns covering a variety of topics and industries such as Health, Education, Lifestyle, Business and Technology, and Corporate Social Responsibility. We turn consumer interest into action by providing readers with motivational editorial, pairing it with relevant advertisers and distributing it within top newspapers and online platforms around the world. Please visit http://www.mediaplanet.com for more on who we are and what we do.


DUBLIN, Ireland, March 02, 2017 (GLOBE NEWSWIRE) -- Horizon Pharma plc (NASDAQ:HZNP), a biopharmaceutical company focused on improving patients’ lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs, today announced support of a program developed by the National Organization for Rare Disorders (NORD) to help people with Urea Cycle Disorders (UCDs) seeking assistance with high out-of-pocket costs associated with the purchase of medical foods and supplements for their low-protein dietary needs.   “Patients' health care needs extend beyond medications and we commend Horizon for being willing to support programs that are aimed at overcoming obstacles to other important treatments,” said Catherine Blansfield, RN, vice president of patient services at NORD.  “This donation provides financial support for products, which are essential for good health but, unfortunately, are not commonly covered by health insurance benefits.” The program is independently administered by NORD and available to all people living with UCDs in the United States who meet the NORD eligibility criteria.  Horizon provided a charitable grant to help fund the program and has no involvement or input on its administration. “I have to significantly limit the amount of protein I consume to avoid elevated ammonia levels, but the flipside is that too little protein can deprive my body of core amino acids, and that can also lead to high ammonia levels,” said Denise Z., who lives with a type of UCD called Citrullinemia Type 1.  “Medical foods and supplements that provide these core nutrients are vital to my health, and that is why this program is so important to those of us in the UCD community who struggle with the day-to-day challenges of living with a UCD.” UCDs are rare genetic disorders that affect approximately 1 in 35,000 people in the United States.  It is caused by an enzyme deficiency in the urea cycle, a process that is responsible for converting excess ammonia from the bloodstream and ultimately removing it from the body.  Because of this, people with a UCD experience hyperammonemia, or elevated ammonia levels in their blood that can then reach the brain where it can cause irreversible brain damage, coma or death.  UCD symptoms may first occur at any age depending on the severity of the disorder, with more severe defects presenting earlier in life.  Those with UCDs adhere to a low-protein diet to decrease the nitrogen load of the urea cycle, and receive essential nutrient supplementation to help achieve normal growth and metabolic stability.1 “At Horizon, we are always looking for ways to be supportive, beyond our therapies, for people with rare diseases,” said Robert Metz, senior vice president, business operations and external affairs, Horizon Pharma plc.  “We’ve received feedback directly from people living with UCDs, their caregivers and health care professionals about the financial burden that those who require medical foods and supplements face, and are thrilled to support NORD’s UCDs Medical Foods and Supplements Financial Assistance Program.” To learn more about the UCDs Medical Foods and Supplements Financial Assistance Program, patients or their caregivers should contact NORD at 877-333-1860 or UCD@rarediseases.org. About Horizon Pharma plc Horizon Pharma plc is a biopharmaceutical company focused on improving patients' lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs.  The Company markets 11 medicines through its orphan, rheumatology and primary care business units.  For more information, please visit www.horizonpharma.com.  Follow @HZNPplc on Twitter or view careers on our LinkedIn page.


News Article | February 28, 2017
Site: www.businesswire.com

WILMINGTON, Del.--(BUSINESS WIRE)--Incyte Corporation (Nasdaq:INCY) joins the National Organization for Rare Disorders (NORD), the European Organization for Rare Diseases (EURODIS) and other rare disease health organizations and advocates in recognizing Rare Disease Day 2017. “ Raising awareness and educating the global community about rare diseases is critical to helping improve the lives of patients and families affected by these conditions,” said Hervé Hoppenot, CEO of Incyte. “ Rare Disease Day provides an opportunity to honor and give a voice to those impacted by rare diseases, including the estimated 200,000 individuals in the U.S. living with myeloproliferative neoplasms (MPNs), a closely related group of rare blood cancers.” MPNs are chronic, progressive blood cancers that can strike anyone at any age. MPNs occur when the bone marrow cells that produce the body's blood cells develop and function abnormally. The three main MPNs are myelofibrosis (MF), polycythemia vera (PV) and essential thrombocythemia (ET). This year, as part of its ongoing MPN educational efforts, Incyte teamed up with ABC’s General Hospital to raise awareness about PV. The storyline and partnership with actress Finola Hughes, whose character on General Hospital was diagnosed with PV, aim to raise awareness and inspire patients and caregivers impacted by these under-recognized blood cancers. To learn more, visit www.VoicesofMPN.com. Finola Hughes commented, “ I am honored to lend my voice on and off the set of General Hospital to help more people understand the impact MPNs, including PV, can have on patients and their families.” Rare Disease Day is an important time to recognize and honor the patients, doctors, caregivers, advocates and organizations that contribute to bringing understanding, compassion and strength to those living with rare disease like MPNs. The MPN Heroes Recognition Program, now in its fifth year, is sponsored by Incyte in collaboration with CURE Media Group, publishers of CURE® magazine. The MPN Heroes® program recognizes individuals and organizations who have contributed to the MPN community by going above and beyond to make a real difference. Nominations for the 2017 MPN Heroes program will be accepted online, by mail or by phone beginning on Rare Disease Day through September, 14, 2017. To learn more about the program or to submit a nomination, visit http://www.mpnheroes.com for more details. On Rare Disease Day, MPN patients, physicians and caregivers are also encouraged to share their stories, show their support and help raise awareness of these rare disorders via the Voices of MPN website and Facebook page. Those impacted by MPNs are invited to join in a ‘Voices of MPN Ask an MPN Expert’ Facebook Live Chat tonight, February 28, 2017 at 8:30 PM Eastern Time, where a medical expert in the field will answer questions about MPNs. Visit the Voices of MPN Facebook page to join in. Myeloproliferative neoplasms (MPNs) are a closely related group of blood cancers in which the bone marrow cells that produce the body’s blood cells develop and function abnormally.1 The three main myeloproliferative neoplasms are myelofibrosis (MF), polycythemia vera (PV) and essential thrombocythemia (ET).1 MPNs are progressive blood cancers that can strike anyone at any age, but they are more common in older adults. Estimates of the prevalence of MPNs vary, but analysis of claims data suggests there may be as many as 200,000 people in the U.S. living with MF, PV or ET.2 Incyte Corporation is a Wilmington, Delaware-based biopharmaceutical company focused on the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit the Company’s website at www.incyte.com. MPN Heroes and Voices of MPN are registered trademarks of Incyte Corporation. 1 MPN Research Foundation. “Understanding MPNs.” Available at: http://www.mpnresearchfoundation.org/overview-page 2 Data on file.


WASHINGTON, DC--(Marketwired - Feb 14, 2017) - The National Academies of Sciences, Engineering and Medicine today issued a report that examines the scientific, clinical, ethical, legal and social implications of human genome editing. The Alliance for Regenerative Medicine (ARM) believes that genomic medicines, including genome editing, hold great promise for the treatment of a multitude of hereditary and acquired diseases where there is presently no effective treatment available. The Alliance for Regenerative Medicine (ARM) applauds the National Academies of Sciences, Engineering and Medicine for its very thorough and thoughtful report on the current scientific, technical, ethical, and policy issues relating to human genome editing. We support the need for responsible and ethically appropriate approaches to research and clinical use of these technologies following the seven guiding principles outlined in the report, as well as the need for continued public engagement and dialogue. We also commend the Academies for recognizing the profound impact genome editing will have on the development of a new class of medicines for many patients with presently incurable diseases. We believe the report's recommendations that "existing regulatory infrastructure and processes for reviewing and evaluating somatic gene therapy to treat or prevent disease and disability" are sensible and will help create a safe path toward eventual clinical adoption and regulatory approval of therapeutics based on somatic cell genome editing. In addition, we note the report's recommendations on heritable germline genome editing and the strict criteria to be met before ever considering clinical study. ARM will continue to monitor developments related to these applications, but until safety is proven and the risks associated with long-term consequences, both intended and unintended, are fully evaluated, we will remain solely focused on realizing the full therapeutic potential of somatic cell genome editing. Further, we must be satisfied that all relevant moral and ethical issues have been addressed and that a broad societal consensus exists as to the benefits and risks associated with editing the germline. ARM believes that advances in the field of gene therapy, including somatic cell genome editing, have the potential to profoundly and positively impact the practice of medicine for currently incurable genetic diseases, such as muscular dystrophy, sickle cell disease (SCD), cystic fibrosis, hemophilia, adrenoleukodystrophy (ALD), Alpha-1 Antitrypsis Deficiency (AATD), and Transthyretin Amyloidosis (ATTR), as well as acquired diseases such as cancer, certain forms of heart disease, HIV, Hepatitis B virus, and other infectious diseases. It is estimated that 30 million Americans, or 1 in every 10 people, are afflicted with one of the approximately 7,000 rare diseases. Two thirds of those affected are children. The National Organization for Rare Disorders (NORD) estimates that for 95 percent of these diseases no FDA-approved treatment currently exists,(1) and the few treatments that are available generally address the symptoms and not the underlying genetic cause of the disease. As a result, these treatments must be administered for the duration of a patient's life. In contrast, genome editing offers the very real potential to bring hope to rare disease patients through development of a broad range of new technologies to precisely target and modify the genetic material of a patient's cells. By removing, repairing, or replacing a defective gene or genes, these therapies hold the promise of potentially curing a broad range of diseases with a single treatment. Similarly, in diseases such as cancer, HIV, and beta-thalessemia, genome editing is being employed to modify T cells and hematopoietic stem cells ex-vivo. The modified cells are then delivered to the patient to treat and potentially cure the underlying disease. These programs build upon early successes and several advanced programs based on somatic cell gene replacement therapies. According to a recent white paper titled, "Therapeutic Gene Editing," published by the American Society of Gene & Cell Therapy (ASGCT), "the successful development of effective treatments based on genome editing could shift today's approach from a lifetime of symptom management for hereditary diseases to tomorrow's ideal of making a one-time curative repair or change to an individual's affected gene. The goal is a long lasting, perhaps life-long effect that minimizes or even eliminates disease."(2) Diseases involving multiple genes may also be treatable if the therapy can alter specific genes affecting the course of the disease. ARM represents a number of companies and research institutions that use various gene therapy and genome editing technology platforms, including CRISPR/Cas9, zinc finger nucleases (ZFNs), homing endonucleases, vector-driven homologous recombination, transcription activator-like effector-based nucleases (TALEN) and meganucleases, amongst others to design therapeutics that address a wide range of, hereditary and acquired diseases. About The Alliance for Regenerative Medicine The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 250 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit http://www.alliancerm.org. 1. National Organization for Rare Disorders (2015). NORD developing 20 natural history studies for 20 rare diseases (Press Release). https://rarediseases.org/fda-awards-nord-250000-grant-to-support-the-development-of-20-natural-history-studies-for-rare- disease-research/. 2. American Society of Gene & Cell Therapy (2016). Therapeutic Gene Editing: An American Society of Gene & Cell Therapy White Paper. http://www.asgct.org/UserFiles/file/TherapeuticGeneEditingWP_Nov21_v1.pdf.


(PRLEAP.COM) February 24, 2017 - Fibrolamellar. You have probably never heard of it, but it is a fatal liver cancer that strikes teens and young adults. You also most likely never heard of Niemann-Pick Type C disease (NPC) a debilitating and fatal genetic metabolic disorder affecting young children's visual, cognitive and motor abilitiesBoth of these rare diseases are speaking out this week on behalf of the over 7000 rare diseases for National Rare Disease Day , where, here in Connecticut, statewide activities will generate awareness for rare diseases, which collectively affect 1 in 10 Americans, and are typically the costliest, and deadly.John Hopper, Executive Director of the Fibrolamellar Cancer Foundation (FCF) , and Phil Marella, Co-founder of Dana's Angels Research Trust (DART) - both headquartered in Greenwich, CT have become visible spokespersons and among the national leaders for their respective rare disease categories in the state of Connecticut and across the nation.John Hopper, who is also co-chair of the GI Cancers Alliance and Board director for the National Pancreas Foundation said, "Rare cancers represent a large percentage of all cancers, and the majority of rare diseases. Fibrolamellar is a very rare and aggressive liver cancer that attacks teens and young adults who are typically very healthy, yet rarely diagnosed until Stage IV. To date there are few curative treatments which is why sponsoring research is a priority for the foundation. We are proud to bring awareness to all of the rare diseases that affect so many people."Phil Marella,Trustee of DART said, "NPC is a rare cholesterol storage disease that takes children piece by piece before it takes their lives. Working with our collaborators in an effort called Support Of Accelerated Research for NPC (SOAR-NPC), DART has helped advance medical research, which includes five clinical drug trials, one ongoing in Phase 3 by Vtesse, Inc. with over 20 sites worldwide. Our son Andrew, 17 years old, who is in the Vtesse trial, has been one of the many beneficiaries of our efforts. While we have been slowing down the progression of the disease, we must stop it and ultimately find a cure."During the Hartford event, patients, caregivers, medical professionals, industry representatives and state legislators will share their stories and support for the estimated 357,000 residents living with a rare disease in Connecticut.Government officials are backing both these foundation's mission of building a collaborative community to support research, broaden education and build patient communities around rare diseases such as Fibrolamellar and Niemann-Pick Type C. Many important decisions related to rare diseases are made at the state level, including newborn screening; support services to help families cope with complex medical needs; an environment that promotes innovative medical research and product development; and insurance practices that assure patient access to medically-necessary therapies. The implementation of the Affordable Care Act has highlighted the increasingly important role of state policies and programs in assuring that the health care needs of Americans are addressed.State Senator Scott Frantz said, "Ensuring quality health care for all includes those with rare diseases. Federal and State support should include studies that can result in more effective and less costly treatments for rare diseases. Affected families deserve no less attention than those families with more common and widespread illnesses."State Representative Fred Camillo added, "As someone whose family was personally affected by rare cancer, this day has special meaning. The day a parent is told their child has a rare disease such as fibrolamellar or niemann pick type c - with no cure- is the day their entire life is changed- forever. The emotional and financial stress impacts home, work, and community for these families. We, in Connecticut and beyond, need to collectively focus on ways to accelerate support, and leverage our assets to help organizations like FCF and DART."To hear more about Rare Disease Day here in Connecticut, tune into your local news on February 28th or attend the Hartford forum at the State Capital. Details are available at: http://www.rarediseaseday.us/events/locations FCF, a public 501c3 nonprofit organization based in Greenwich, CT, was founded in 2009 by 27-year-old Tucker Davis, who lost his life to the disease in 2010. The foundations mission is to:To date, the foundation has funded nearly $6 million in research at major research institutions in the US and Internationally. 100% of donations go to research with all operating costs funded by the Charles and Marna Davis Foundation. www.fibrofoundation.org DART is a public 501c3nonprofit organization that was founded in 2002 by Andrea and Phil Marella in an effort to save the lives of two of their four children suffering from Niemann-Pick Type C disease-as well as the many others afflicted. Sadly, the Marella's daughter, Dana, at 19, passed away in the Summer of 2013. But the effort continues supporting pivotal NPC research; research that may also help millions of people suffering from Ebola, HIV/Aids, heart disease, stroke, Alzheimer's disease and other disorders that appear to be related to cholesterol metabolism. To date, DART, an all-volunteer organization, has raised over $4 million for NPC research.Rare Disease Day is an annual awareness day celebrated around the world dedicated to elevating public understanding of rare diseases and calling attention to the special challenges faced by patients. The goal of Rare Disease Day, organized by the National Organization for Rare Disorders (NORD)® (Danbury CT national headquarters) is to foster collaborative efforts across all stakeholders, especially government, to increase awareness, improve care support, encourage research, and advocate resources- financial and others- to address this growing population- in hopes for a healthier community.Common challenges facing rare disease patients and families include but are not limited to:For more information about Rare Disease Day in the U.S., go to www.RareDiseaseDay.us. For information about global activities, visit www.RareDiseaseDay.org ).To search for information about rare diseases, visit NORD's website, www.RareDiseases.org. 20 Horseneck Lane, 2nd Floor, Greenwich, CT 06830T: 203-862-319615 East Putnam Ave., #117Greenwich, CT 06830203-861-2063


CORAL GABLES, Fla., Feb. 27, 2017 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), (Catalyst) a biopharmaceutical company focused on  developing  and  commercializing  innovative  therapies  for  people  with  rare  debilitating diseases, today announced that  it  has  joined  forces  with  30  million  Americans  and  health  care  advocates around the world for Rare Disease Day® on February 28. Rare Disease Day is an annual awareness day dedicated to elevating public understanding of rare diseases and calling attention to the special challenges they present to patients and families. Catalyst continues to support patient organizations that provide disease awareness for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS). These are rare diseases in which patients are often misdiagnosed with other diseases prior to getting a definitive diagnosis, thus educating the patient and physicians at medical congresses is an important outreach to the rare disease community to facilitate proper and prompt diagnoses. "Completing one phase 3 clinical study and enrolling a second phase 3 clinical study for LEMS indicates our commitment to the LEMS community for patients to have access to an FDA approved treatment" says Patrick J. McEnany, Chairman and CEO of Catalyst. "In addition, we have expanded our CMS phase 3 clinical study to include adults in recognition of the need to provide access to a potential treatment across the entire CMS population. We are pleased to continue to support the efforts of the rare disease community to find ways to broaden the awareness of rare diseases and improve access to treatments.” According to the National Institutes of Health (NIH), a disease is rare if it affects fewer than 200,000 people. Nearly 1 in 10 Americans live with a rare disease—affecting 30 million people—and nearly half of these patients are children. There are more than 7,000 rare diseases and only approximately 450 FDA-approved medical treatments. Rare Disease Day takes place every year on the last day of February (February 28 or February 29 in a leap year)—the rarest date on the calendar—to underscore the nature of rare diseases and what patients face. It was established in Europe in 2008 by EURORDIS, the organization representing rare disease patients in Europe, and is now observed in more than 80 nations. Rare Disease Day is sponsored in the U.S. by the National Organization for Rare Disorders (NORD)®, the largest and leading independent, nonprofit organization committed to the identification, treatment, and cure of rare diseases and one which Catalyst actively supports.  Additionally, Catalyst supports Global Genes in their efforts to continue to get patients with rare diseases to take an active role in raising awareness about their disease. For more information about Rare Disease Day in the U.S., go to http://www.rarediseaseday.us.  For information about global activities, go to www.rarediseaseday.org. To search for information about rare diseases, visit NORD’s website www.rarediseases.org and Global Gene’s website https://globalgenes.org/world-rare-disease-day. Catalyst Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, including Lambert-Eaton myasthenic syndrome (LEMS), congenital myasthenic syndromes (CMS), infantile spasms, and Tourette's Disorder. Firdapse® has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the treatment of LEMS and Orphan Drug designation for LEMS, CMS and myasthenia gravis. Firdapse is the first and only approved drug in Europe for symptomatic treatment in adults with LEMS. Catalyst is also developing CPP-115 to treat infantile spasms, epilepsy and other neurological conditions associated with reduced GABAergic signaling, like post-traumatic stress disorder and Tourette's Disorder. CPP-115 has been granted U.S. orphan drug designation for the treatment of infantile spasms by the FDA and has been granted E.U. orphan medicinal product designation for the treatment of West Syndrome by the European Commission.  In addition, Catalyst is developing a generic version of Sabril® (vigabatrin). This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including whether the receipt of breakthrough therapy designation for Firdapse will expedite the development and review of Firdapse by the FDA or the likelihood that the product will be found to be safe and effective, the timing on Catalyst's second trial evaluating Firdapse for the treatment of LEMS and whether the trial will be successful, whether Catalyst's assumptions in its updated business plan will be accurate and the impact of unanticipated events or delays in projected activities on Catalyst's cash requirements and on Catalyst's ability to get to an accepted NDA submission for Firdapse without the need for additional funding, what clinical trials and studies will be required before Catalyst can resubmit an NDA for Firdapse for the treatment of CMS and whether any such required clinical trials and studies will be successful, whether the investigator-sponsored study evaluating Firdapse for the treatment of MuSK-MG will be successful, whether any NDA for Firdapse resubmitted to the FDA will ever be accepted for filing,  the timing of any such NDA filing or acceptance, whether, if an NDA for Firdapse is accepted for filing, such NDA will be given a priority review by the FDA, whether Firdapse will ever be approved for commercialization, whether Catalyst will be the first company to receive approval for amifampridine (3,4-DAP), giving it 7-year marketing exclusivity for its product, whether CPP-115 will be determined to be safe for humans, what additional testing will be required before CPP-115 is "Phase 2 ready", whether CPP-115 will be determined to be effective for the treatment of infantile spasms, post-traumatic stress disorder, Tourette's Disorder or any other indications, whether Catalyst can successfully design and complete a bioequivalence study of its version of vigabatrin compared to Sabril that is acceptable to the FDA, whether any such bioequivalence study the design of which is acceptable to the FDA will be successful, whether any ANDA that Catalyst submits for a generic version of Sabril will be accepted for filing, whether any ANDA for Sabril accepted for filing by the FDA will be approved (and the timing of any such approval), whether any of Catalyst's product candidates will ever be approved for commercialization or successfully commercialized, and those other factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2015 and its other filings with the U.S. Securities and Exchange Commission (SEC), could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are available from the SEC, may be found on Catalyst's website, or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date.


News Article | February 28, 2017
Site: globenewswire.com

OXFORD, United Kingdom, Feb. 28, 2017 (GLOBE NEWSWIRE) -- Summit Therapeutics plc (NASDAQ:SMMT) (AIM:SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy (‘DMD’) and Clostridium difficile infection, recognises the tenth annual Rare Disease Day taking place today, 28 February 2017. The Rare Disease Day 2017 theme, ‘with research, possibilities are limitless,’ emphasises the importance of scientific research in helping to understand, diagnose and treat rare diseases that affect millions of people and their families worldwide. Summit seeks to remain at the forefront of utrophin modulation research through its strategic alliance with the University of Oxford, under the guidance of Professor Kay Davies. The collaboration is focussed on developing future-generation utrophin modulators for the potential treatment of all patients with the progressive muscle wasting disorder, DMD. To date, the research team has identified two series of novel utrophin modulators, one of which has a mechanism of action potentially distinct from ezutromid, the Company’s lead utrophin modulator that is in a Phase 2 clinical trial in DMD patients. “In our quest to bring a potentially disease-modifying treatment to all patients with DMD, we have collaborated with the preeminent expert in utrophin modulation biology, Professor Kay Davies, and her research team at the University of Oxford,” said Glyn Edwards, Chief Executive Officer of Summit. “We applaud EURORDIS, the organisation representing rare disease patients in Europe, for bringing an annual spotlight to the plight of millions of people affected by rare diseases and in this year, recognising the immense impact that research is having and will continue to have for those living with rare diseases.” In the European Union a rare disease is defined as one that affects fewer than 5 in 10,000 of the general population, while in the United States, it is defined as a disease that affects fewer than 200,000 people. There are between 6,000 and 8,000 known rare diseases with around five new rare diseases described in the literature each week. Rare diseases are often chronic and life threatening and include rare conditions, such as childhood cancers, and some other well-known conditions including cystic fibrosis and DMD. Rare Disease Day takes place on the last day of February each year, and its objective is to raise awareness among the general public and decision-makers about rare diseases and their impact on patients' lives. Rare Disease Day was launched in Europe in 2008 by EURORDIS. It is now observed in more than 80 nations, and is sponsored in the US by the National Organization for Rare Disorders (NORD). For more information, please visit www.rarediseaseday.org. About Utrophin Modulation in DMD DMD is a progressive muscle wasting disease that affects around 50,000 boys and young men in the developed world. The disease is caused by different genetic faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of all muscles. There is currently no cure for DMD and life expectancy is into the late twenties. Utrophin protein is functionally and structurally similar to dystrophin. In preclinical studies, the continued expression of utrophin has a meaningful, positive effect on muscle performance. Summit believes that utrophin modulation has the potential to slow down or even stop the progression of DMD, regardless of the underlying dystrophin gene mutation. Summit also believes that utrophin modulation could potentially be complementary to other therapeutic approaches for DMD. The Company’s lead utrophin modulator, ezutromid, is an orally administered, small molecule. DMD is an orphan disease, and the US Food and Drug Administration (‘FDA’) and the European Medicines Agency have granted orphan drug status to ezutromid. Orphan drugs receive a number of benefits including additional regulatory support and a period of market exclusivity following approval. In addition, ezutromid has been granted Fast Track designation and Rare Pediatric Disease designation by the FDA. About Summit Therapeutics Summit is a biopharmaceutical company focused on the discovery, development and commercialisation of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programs focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficile infection. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc). For more information, please contact: Forward-looking Statements Any statements in this press release about Summit’s future expectations, plans and prospects, including but not limited to, statements about the clinical and preclinical development of Summit’s product candidates, the therapeutic potential of Summit’s product candidates, and the timing of initiation, completion and availability of data from clinical trials, and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," and similar expressions, constitute forward looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of future clinical trials, availability and timing of data from on-going and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials or preclinical studies will be indicative of the results of later clinical trials, expectations for regulatory approvals, availability of funding sufficient for Summit’s foreseeable and unforeseeable operating expenses and capital expenditure requirements and other factors discussed in the "Risk Factors" section of filings that Summit makes with the Securities and Exchange Commission including Summit’s Annual Report on Form 20-F for the fiscal year ended January 31, 2016. Accordingly readers should not place undue reliance on forward looking statements or information. In addition, any forward looking statements included in this press release represent Summit’s views only as of the date of this release and should not be relied upon as representing Summit’s views as of any subsequent date. Summit specifically disclaims any obligation to update any forward-looking statements included in this press release.


News Article | February 15, 2017
Site: www.npr.org

Republican Sen. Chuck Grassley, chairman of the Senate Judiciary Committee, has opened an inquiry into potential abuses of the Orphan Drug Act that may have contributed to high prices on commonly used drugs. In a statement, Grassley said the inquiry is "based on reporting from Kaiser Health News" and strong consumer concern about high drug prices. "My staff is meeting with interested groups and other Senate staff to get their views on the extent of the problem and how we might fix it," Grassley wrote. A six-month Kaiser Health News investigation published in January found that the orphan drug program intended to help desperate patients is being manipulated by drugmakers. While the companies aren't breaking the law, they are using the Orphan Drug Act to secure lucrative incentives and gain monopoly control of rare disease markets where drugs often command astronomical price tags. KHN's investigation, which was published and aired by NPR, found that many drugs that now have orphan status aren't entirely new. More than 70 were drugs first approved by the Food and Drug Administration for mass market use. Those include cholesterol blockbuster Crestor, Abilify for psychiatric disorders and rheumatoid arthritis drug Humira, the best-selling drug in the world. Others are drugs that have received multiple exclusivity periods for two or more rare conditions. About 80 drugs fall into this latter category, including cancer drug Gleevec and wrinkle-fighting drug Botox. Before the Orphan Drug Act passed, drugs for rare diseases were often abandoned during development, hence the name orphan. The patient populations were simply too small for the drugs to be financially viable for companies, they said. Grassley, the senior senator from Iowa, is well positioned to call for changes in the law's incentives. The Judiciary Committee he oversees has jurisdiction over anticompetitive and patent-related issues. Grassley made headlines late last year calling for hearings to demand that drugmaker Mylan justify its price hikes for the lifesaving EpiPen. Grassley's office has reached out to staff members of the Senate Health, Education, Labor and Pensions committee, which has jurisdiction over the FDA. When asked this week about a possible update to the law, the committee lauded the Orphan Drug Act for helping millions of families and said it "will continue oversight of the law to ensure it is working as intended." But there are questions over whether the law is working as intended. Dr. Robert Califf, who left his post last month as commissioner of the FDA, said it's time to review the orphan drug program and the incentives offered to corporations. While the law has been very successful in bringing effective treatments to patients with rare disease, Califf said, it's also the case that when a program "reaches a mature phase it's important to reassess it." Califf went on to say, "It's understandable that whenever you put a financial incentive in place in the United States, people will try to figure out how to take advantage of it — almost all of them within the law. So, I'm not talking about illegal activity but ... there is a game of cat and mouse that goes on." Government incentives for orphan drug development are needed because the research involved is costly, risky and uncertain, Anne Pritchett, vice president of policy and research at PhRMA, the drug industry's lobbying group, said in an interview in late 2016. This week, a PhRMA spokeswoman said the group hadn't heard of any movement on changing or updating the Orphan Drug Act. Dr. Martin Makary, a professor at Johns Hopkins University School of Medicine and a vocal critic of the high prices of orphan drugs, has suggested that drugmakers should repay some of the federal incentive money once a drug reaches a $1 billion in annual sales. Former U.S. Rep. Henry Waxman, D-Calif., who pioneered the Orphan Drug Act in the 1980s, thinks Congress should tackle prescription drug pricing on a variety of fronts, and that an update to the Orphan Drug Act should be part of the solution. But Peter Saltonstall, president of the National Organization for Rare Disorders, said his group has heard "no energy" for changing the law now. "I think the Orphan Drug Act, at least from our perspective, has been successful," Saltonstall said, adding that there are other drug pricing concerns that could be addressed by Congress. Today, more than 450 orphan drugs have been approved. Obtaining orphan designation for a product is a popular business strategy for manufacturers. Drugs approved to treat rare diseases made up 40 percent of new drugs approved by the FDA in 2016 and nearly 50 percent of new drugs approved in 2015. The drugs are also expensive, at $111,820 for a year's treatment with an orphan drug, on average, in 2014 compared with $23,331 for a mass market drug. Kaiser Health News is an editorially independent newsroom that is part of the nonpartisan Henry J. Kaiser Family Foundation. KHN's coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation.

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