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Peay H.L.,Parent Project Muscular Dystrophy | Fischer R.,Parent Project Muscular Dystrophy
Clinical Therapeutics | Year: 2014

Background There is growing agreement that regulators performing benefit-risk evaluations should take patients' and caregivers' preferences into consideration. The Patient-Focused Drug Development Initiative at the US Food and Drug Administration offers patients and caregivers an enhanced opportunity to contribute to regulatory processes by offering direct testimonials. This process may be advanced by providing scientific evidence regarding treatment preferences through engagement of a broad community of patients and caregivers. Objective In this article, we demonstrate a community-engaged approach to measure caregiver preferences for potential benefits and risks of emerging therapies for Duchenne muscular dystrophy (DMD). Methods An advocacy oversight team led the community-engaged study. Caregivers' treatment preferences were measured by using best-worst scaling (BWS). Six relevant and understandable attributes describing potential benefits and risks of emerging DMD therapies were identified through engagement with advocates (n = 5), clinicians (n = 9), drug developers from pharmaceutical companies and academic centers (n = 11), and other stakeholders (n = 5). The attributes, each defined across 3 levels, included muscle function, life span, knowledge about the drug, nausea, risk of bleeds, and risk of arrhythmia. Cognitive interviewing with caregivers (n = 7) was used to refine terminology and assess acceptability of the BWS instrument. The study was implemented through an online survey of DMD caregivers, who were recruited in the United States through an advocacy group and snowball sampling. Caregivers were presented with 18 treatment profiles, identified via a main-effect orthogonal experimental design, in which the dependent variable was the respondents' judgment as to the best and worst feature in each profile. Preference weights were estimated by calculating the relative number of times a feature was chosen as best and as worst, which were then used to estimate relative attribute importance. Results A total of 119 DMD caregivers completed the BWS instrument; they were predominately biological mothers (67.2%), married (89.9%), and white (91.6%). Treatment effect on muscle function was the most important among experimental attributes (28.7%), followed by risk of heart arrhythmia (22.4%) and risk of bleeding (21.2%). Having additional postapproval data was relatively the least important attribute (2.3%). Conclusions We present a model process for advocacy organizations aiming to promote patient-centered drug development. The community-engaged approach was successfully used to develop and implement a survey to measure caregiver preferences. Caregivers were willing to accept a serious risk when balanced with a noncurative treatment, even absent improvement in life span. These preferences should inform the Food and Drug Administration's benefit-risk assessment of emerging DMD therapies. This study highlights the synergistic integration of traditional advocacy methods and scientific approach to quantify benefit-risk preferences. © 2014 The Authors.


CAMBRIDGE, Mass.--(BUSINESS WIRE)--Eli Lilly and Company, one of the top 15 global pharmaceutical companies, has joined the Collaborative Trajectory Analysis Project (cTAP), a unique partnership of many stakeholders all committed to accelerating scientific discovery and bringing new treatments to Duchenne Muscular Dystrophy (DMD) patients more rapidly. “Lilly is proud to support the efforts of cTAP, which is making a real difference in our understanding of the progression of DMD, and how variations across patients make it particularly difficult to design effective clinical trials,” said Kraig Kinchen, M.D., senior medical director of Lilly’s Bio-medicines Core Team. “cTAP is a model that brings together the collective talent of a multi-stakeholder community. We all share a common interest in speeding the development of potentially effective therapies for Duchenne patients and their families.” Debra Miller, founder and CEO of CureDuchenne, a leading advocacy group in the fight against Duchenne, said: “Advancing our scientific knowledge of Duchenne and the way in which its progression varies across patients is cTAP’s mission. CureDuchenne was the founding advocacy group supporting cTAP and we are thrilled by Lilly’s contribution.” “By sharing trial data, Lilly is showing real leadership,” added Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy. “PPMD has a long history with Lilly and their tadalafil trial, and we believe the data collected from our community will be valuable to the mission of cTAP.” Duchenne Muscular Dystrophy is a progressive, fatal disease of boys characterized by gradual weakening of muscles. It is the most common fatal genetic disorder diagnosed in childhood. Most Duchenne patients die in their 20s. Though a Phase III human clinical trial of tadalafil, a phosphodiesterase inhibitor, in Duchenne ultimately failed to meet its targets for proving efficacy in slowing the decline in patients’ ability to walk, the data generated by the trial are essential to expanding scientific understanding of the rate of decline in boys with Duchenne. cTAP has characterized the variable rates at which Duchenne patients that exhibit similar symptoms often progress. This variation makes it difficult to design clinical drug trials that can definitively prove the efficacy of new treatments. Because Duchenne is a rare disease, with only about 20,000 new cases worldwide each year, it is virtually impossible to conduct large-scale trials with many patients, an approach that can be used to overcome variability in more prevalent disorders. “Through our collaboration with cTAP we have been able to create new models that eliminate much of the statistical variation we see in patients in clinical trials of experimental Duchenne treatments,” said Professor Nathalie Goemans, head of the Neuromuscular Reference Center for Children at the University Hospitals in Leuven, Belgium. “With these new data from Lilly, we will be able to assess how well our predictive tools – developed using natural history data – perform in the placebo arm of a clinical trial for a Duchenne treatment. That’s why Lilly’s participation is so critically important.” The Collaborative Trajectory Analysis Project, or cTAP, is a novel group enabling leading clinical experts to solve the most urgent problems in Duchenne drug development. cTAP is a dynamic alliance of scientists, drug companies, patient advocacy organizations, and registries and clinical centers in DMD across Europe and the U.S. The collaboration also brings leaders in biostatistical outcomes research to the fight against Duchenne. cTAP is curating and growing what is already the largest natural history database of DMD. This enables it to develop near-term solutions for some of the key problems in designing clinical trials and analyzing their results. To learn more, please visit ctap-duchenne.org. Duchenne Muscular Dystrophy is a uniformly fatal, progressive, muscle-wasting disease affecting about one in 3,500-6,000 male live births. Patients with Duchenne lack the ability to make dystrophin, a protein crucial to muscle function. As their muscles deteriorate, they progressively lose the ability to walk, feed themselves, turn over in bed, and ultimately to breathe. While there is no cure, the past decade has seen an explosion in research, resulting in more than 15 therapies entering clinical development, with some receiving limited approval. Learn more about Duchenne at cureduchenne.org and parentprojectmd.org.


HACKENSACK, N.J., Feb. 16, 2017 /PRNewswire-USNewswire/ -- Ann & Robert H. Lurie Children's Hospital of Chicago (Lurie Children's) became the 14th clinic named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to...


News Article | November 3, 2016
Site: globenewswire.com

ALLSCHWIL, SCHWEIZ und ROCKVILLE (MD), USA - 3. November 2016 - Actelion Ltd (SIX: ATLN) und ReveraGen BioPharma, Inc., ein privates Unternehmen, das proprietäre therapeutische Produkte im Bereich der neuromuskulären und entzündlichen Krankheiten erforscht und entwickelt, gaben heute den Abschluss einer Vereinbarung bekannt. Im Rahmen dieser Vereinbarung erhält Actelion eine Exklusivoption zur Einlizensierung des Leitkandidaten von ReveraGen, Vamorolone, zur Behandlung von Muskeldystrophie Duchenne. Die Option kann in zwei verschiedenen Entwicklungsphasen des Wirkstoffs ausgeübt werden. ReveraGen entwickelt Vamorolone in klinischen Studienprogrammen parallel in den USA und in Europa. Die klinischen Studien der Phase I wurden Ende 2015 abgeschlossen - sie wurden im Rahmen von Venture-Philanthropy-Verträgen von Organisationen wie der Muscular Dystrophy Association (USA), Joining Jack (UK), Duchenne Research Fund (UK) und Duchenne Children's Trust (UK) finanziert. Actelion hat mit ReveraGen BioPharma, Inc., einer Gesellschaft nach dem Recht des Staates Delaware, USA, eine Lizenz-, kooperative Entwicklungs- und Kommerzialisierungsvereinbarung zur Erforschung und gemeinsamen Entwicklung von Vamorolone, einem nicht-hormonalen Steroidmodulator primär zur Behandlung von Muskeldystrophie Duchenne (DMD) abgeschlossen. Bislang hat Actelion insgesamt USD 10 Millionen an ReveraGen gezahlt, welche als F&E-Ausgaben ausgewiesen wurden. Gemäss der Vertragsbedingungen wird Actelion in den nächsten drei Jahren auch F&E-Aktivitäten unterstützen, wobei der Höchstbetrag dafür bei USD 1 Million pro Jahr liegt. Weiterhin erwarb Actelion eine Option auf die globalen Exklusivrechte zur Einlizensierung von Vamorolone zu einem beliebigen Zeitpunkt, jedoch nicht später als nach Erhalt der Ergebnisse der IIb-Studie. Wenn die Option ausgeübt wird, hat ReveraGen einen Anspruch auf Milestone-Zahlungen in Höhe von USD 165 Millionen für die Indikation DMD, und auf weitere USD 190 Millionen für drei weitere Indikationen, abhängig vom Erreichen bestimmter Meilensteine in den Bereichen F&E, Registrierung und Kommerzialisierung.  Desweiteren wird Actelion gestaffelte Lizenzgebühren im ein- bis zweistelligen Bereich auf den Nettoumsatz von Vamorolone zahlen. Vamorolone hat sowohl in den USA als auch in Europa den Status als Orphan Drug erhalten. ReveraGen entwickelt Vamorolone in parallelen klinischen Studienprogrammen in den USA und in Europa. Die klinischen Studien der Phase I wurden Ende 2015 abgeschlossen. Gegenwärtig läuft ein Programm der Phase IIa, das die Sicherheit und Wirksamkeit von Vamorolone bei vier- bis siebenjährigen steroidnaiven Jungen mit Duchenne untersucht, d.h. von Patienten, die noch nicht mit Prednison oder Deflazacort behandelt wurden. Das klinische DMD-Programm wird in Zusammenarbeit zwischen CINRG Group und Newcastle University (Kate Bushby und Michela Guglieri) entwickelt und durchgeführt. Ein Programm der Phase IIb ist in Planung. ReveraGen BioPharma, Inc., ist ein privates pharmazeutisches Unternehmen, das 2008 gegründet wurde und seinen Sitz in Rockville (MD), USA hat. Der einzige Vermögenswert von ReveraGen, Vamorolone, ist ein proprietärer Wirkstoff für die Behandlung von Muskeldystrophie Duchenne. Die Entwicklung von Vamorolone erfolgt auf der Basis von Venture Philanthropy, in Zusammenarbeit mit staatlichen Institutionen wie den US National Institutes of Health, der Europäischen Gemeinschaft und internationalen Organisationen, einschliesslich der Foundation to Eradicate Duchenne, Parent Project Muscular Dystrophy, Save our Sons und anderen. Insgesamt wurden bis heute von Regierungsstellen und Interessengruppen USD 20 Millionen zur Verfügung gestellt. Actelion: Andrew C. Weiss Senior Vice President, Head of Investor Relations & Corporate Communications Actelion Pharmaceuticals Ltd, Gewerbestrasse 16, CH-4123 Allschwil +41 61 565 62 62 http://www.actelion.com


News Article | November 3, 2016
Site: globenewswire.com

Vamorolone, a novel compound for the treatment of Duchenne Muscular Dystrophy, holds potential to better preserve muscle function and prolong ambulation for the patient, without some of the side effects associated with glucocorticoid therapy. ALLSCHWIL, SWITZERLAND, and ROCKVILLE (MD), USA - 03 November 2016 - Actelion Ltd (SIX: ATLN) and ReveraGen BioPharma, Inc., a privately held company engaged in the discovery and development of proprietary therapeutic products for neuromuscular and inflammatory diseases, announced today that they have entered into an agreement. By this agreement, Actelion has obtained an exclusive option to in-license ReveraGen's lead compound vamorolone for the treatment of Duchenne Muscular Dystrophy at two different stages in its development. Vamorolone is a novel compound that has the potential to preserve muscle function and prolong ambulation, without some of the known side effects associated with corticosteroids currently in use. This is important especially for very young patients with Duchenne, where glucocorticoid therapy is not appropriate due to these side effects, which include growth stunting and immune suppression. Jean-Paul Clozel, MD and Chief Executive Officer of Actelion, commented: "We have been very impressed by the pioneering work performed by Dr. Hoffman and his team at ReveraGen, and by the encouragement the development of vamorolone receives from the patient community. With our proven scientific, regulatory and marketing expertise in the area of orphan drugs, Actelion is ideally positioned to support the development of this new therapeutic approach, to benefit the boys affected by Duchenne Muscular Dystrophy and help those who care for them." Eric Hoffman, Chief Executive Officer of ReveraGen, commented: "Duchenne has been the focus of our efforts for many years, and we now have the chance to slow the progression of this devastating disease. The project has already greatly benefited from broad philanthropic support, and we are delighted with the option agreement, which will bring Actelion's scientific and commercial competencies to the table, enabling vamorolone to rapidly reach patients with Duchenne and their families." ReveraGen is pursuing parallel clinical development for vamorolone in both the US and Europe. Phase I clinical trials were completed in late 2015, funded through venture philanthropy contracts by organizations including the Muscular Dystrophy Association (USA), Joining Jack (UK), Duchenne Research Fund (UK) and Duchenne Children's Trust (UK). A Phase IIa program is currently underway and investigates the safety and efficacy of vamorolone in 4-7 year old steroid-naïve boys with Duchenne (patients that have not taken prednisone or deflazacort). The DMD clinical program is being developed and run by a collaboration between the CINRG group and Newcastle University (Kate Bushby and Michela Guglieri). A Phase IIb program is in the planning stage. ABOUT THE AGREEMENT Actelion signed a license, collaborative development and commercialization agreement with ReveraGen BioPharma, Inc., a corporation organized under the laws of Delaware, US, to research and co-develop vamorolone, a non-hormonal steroid modulator primarily for the treatment of Duchenne Muscular Dystrophy ("DMD"). To date Actelion has paid a total of USD 10 million to ReveraGen which has been expensed as R&D costs. Under the terms of the agreement Actelion will also support R&D activities up to a maximum amount of USD 1 million p.a. for the next three years. In addition, Actelion acquired an option to obtain the exclusive worldwide license rights on vamorolone at any time but not later than following the receipt of the Phase IIb study results. If the option is exercised, ReveraGen will be entitled to receive up to USD 165 million in development and regulatory milestones for the DMD indication and up to USD 190 million for three further indications depending on achievement of certain development, regulatory approval and commercialization milestones. Furthermore, Actelion will pay increasing tiered single- to double-digit royalties on the net sales of vamorolone. ABOUT VAMOROLONE Vamorolone is the first-in-human steroid-like compound that shows signs of effectively separating a number of sub-properties seen in corticosteroids. Animal models indicate that vamorolone retains sub-properties associated with efficacy (namely anti-inflammatory effects), and in addition shows new membrane stabilization and mineralocorticoid receptor antagonist sub-properties, all without the growth stunting and immune suppression side effects of corticosteroids. Treatment with vamorolone in the earlier stages of DMD holds potential to better preserve muscle function than is seen currently, without some of the side effects that have limited the use of corticosteroids in very young children in particular. Vamorolone has received Orphan Drug Designation in both the US and Europe. ReveraGen is pursuing parallel clinical development for vamorolone in both the US and Europe. Phase I clinical trials have been completed in late 2015. A Phase IIa program is currently underway and investigates the safety and efficacy of vamorolone in 4-7 year old steroid-naïve DMD boys (patients that have not taken prednisone or deflazacort). The DMD clinical program is being developed and run by a collaboration between the CINRG group and Newcastle University (Kate Bushby and Michela Guglieri). A Phase IIb program is in the planning stage. ABOUT DUCHENNE MUSCULAR DYSTROPHY (DMD) Duchenne Muscular Dystrophy is a devastating and aggressive genetic disorder where progressive muscle wasting leads to increasing weakness, disability and early death. It is caused by a mutation of the gene which codes for dystrophin, a protein that is essential to maintain normal muscle function. If dystrophin is absent, the stability of the muscle cell membranes is reduced - the muscle cells are easily damaged and die. Duchenne is an X-linked disease, such as hemophilia and red-green color blindness. As the dystrophin gene is located on the X chromosome - of which females have two, but males only one - females have a "backup" if one of the genes is flawed, and will produce enough dystrophin to preserve muscle function. Females can however be carriers, giving the X chromosome with the mutated dystrophin gene to their sons, who will likely develop Duchenne or Becker Muscular Dystrophy, a milder form of muscular dystrophy related to Duchenne, depending on the mutation. It is estimated that approximately 1 in every 3,500 live male births is affected, with about 20,000 new cases each year worldwide. Onset typically occurs in early childhood, with early motor developmental milestones being delayed. By age four or five, mobility and movement problems like falling or trouble climbing stairs become increasingly apparent. As muscles continue to weaken, the boys begin to require increased physical support and have to use a wheelchair around the age of 7-13 years. With the disease progressing, arms, chest and neck muscles can also be affected and the heart muscle and diaphragm may weaken. By young adulthood, the condition causes premature death, mostly due to respiratory or cardiac failure resulting from extreme muscle weakness. REVERAGEN BIOPHARMA, INC. ReveraGen BioPharma, Inc. is a private pharmaceutical company, founded in 2008 and headquartered in Rockville (MD), USA. ReveraGen's single asset, vamorolone, is a proprietary compound for the treatment of Duchenne Muscular Dystrophy. Vamorolone development has progressed through venture philanthropy, partnering with government programs such as the US National Institutes of Health, European Community, and international stakeholder foundations, including The Foundation to Eradicate Duchenne, Parent Project Muscular Dystrophy, Save Our Sons, and others. In total, approximately USD 20 million has been provided by government and stakeholders to date. ACTELION LTD Actelion Ltd. is a leading biopharmaceutical company focused on the discovery, development and commercialization of innovative drugs for diseases with significant unmet medical needs. Actelion is a leader in the field of pulmonary arterial hypertension (PAH). Our portfolio of PAH treatments covers the spectrum of disease, from WHO Functional Class (FC) II through to FC IV, with oral, inhaled and intravenous medications. Although not available in all countries, Actelion also has treatments approved by health authorities for a number of specialist diseases including Type 1 Gaucher disease, Niemann-Pick type C disease, Digital Ulcers in patients suffering from systemic sclerosis, and mycosis fungoides type cutaneous T-cell lymphoma. Founded in late 1997, with now over 2,500 dedicated professionals covering all key markets around the world including Europe, the US, Japan, China, Russia and Mexico, Actelion has its corporate headquarters in Allschwil / Basel, Switzerland. Actelion shares are traded on the SIX Swiss Exchange (ticker symbol: ATLN) as part of the Swiss blue-chip index SMI (Swiss Market Index SMI®). All trademarks are legally protected. For further information, please contact: For Actelion: Andrew C. Weiss Senior Vice President, Head of Investor Relations & Corporate Communications Actelion Pharmaceuticals Ltd, Gewerbestrasse 16, CH-4123 Allschwil +41 61 565 62 62 http://www.actelion.com The above information contains certain "forward-looking statements", relating to the company's business, which can be identified by the use of forward-looking terminology such as "estimates", "believes", "expects", "may", "are expected to", "will", "will continue", "should", "would be", "seeks", "pending" or "anticipates" or similar expressions, or by discussions of strategy, plans or intentions. Such statements include descriptions of the company's investment and research and development programs and anticipated expenditures in connection therewith, descriptions of new products expected to be introduced by the company and anticipated customer demand for such products and products in the company's existing portfolio. Such statements reflect the current views of the company with respect to future events and are subject to certain risks, uncertainties and assumptions. Many factors could cause the actual results, performance or achievements of the company to be materially different from any future results, performances or achievements that may be expressed or implied by such forward-looking statements. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described herein as anticipated, believed, estimated or expected.


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

In response to outrage from patients and lawmakers, Marathon Pharmaceuticals has delayed the launch of an $89,000 drug for Duchenne muscular dystrophy. The company had announced the annual list price for Emflaza, which is a steroid, after the Food and Drug Administration approved the drug Thursday. Emflaza is approved as an orphan drug, which means it is intended to treat a rare disease. Duchenne is an inherited disorder that causes muscles to become weak. There is no cure for the condition, which mainly affects boys, but some drugs, including Emflaza, are used to lessen symptoms. For years, many American patients have imported deflazacort, the generic version of Emflaza, for about $1,200 a year. But because the medicine wasn't approved in the U.S., the cost of the medicine wasn't typically covered by insurers. That contrast in price between became a flash point Monday as Sen. Bernie Sanders, I-Vt., and Rep. Elijah Cummings, D-Md., sent a letter to Marathon on Monday morning demanding answers about the $89,000 price for a drug that isn't new. It has been used routinely by Duchenne patients in the U.S. since at least 2005. "We believe Marathon is abusing our nation's 'orphan drug' program, which grants companies seven years of market exclusivity to encourage research into new treatments for rare diseases — not to provide companies like Marathon with lucrative market exclusivity rights for drugs that have been available for decades," Sanders and Cummings wrote. Marathon said FDA approval would help more patients get the drug. "Our goal in commercializing Emflaza all along has been to make it available to that broader set of patients who prior to FDA approval have not had access to the therapy," said a statement attributed to Marathon CEO Jeffrey Aronin that was read Monday to a meeting of parents, patients and advocates in Washington, D.C. "We are pausing our launch, which has not yet taken place. We have not sold any new product and will pause that process." But patients and their families cried foul. "What you're doing is robbing my insurance company," said Dana Edwards, a mother from New Jersey whose 12-year-old has taken deflazacort, the generic version of the drug, since he was 5. Pat Furlong, the president and founder of Parent Project Muscular Dystrophy, which sponsored the Monday conference, read the statement to an outraged crowd in a conference room at the Mayflower Hotel. "Since last week's approval, they've heard from all of us," he said. Furlong told the audience that complaints from patients helped to prompt Marathon's action. The company, Furlong read, will continue to offer patients an expanded access program, which allows about 800 patients to receive the drug from the company. More can join that program free, and patients can continue importing the drug from Canada or "wherever they are getting it," the statement said. In January, Kaiser Health News and NPR published an investigation that found the orphan drug law intended to help desperate patients is being manipulated by drugmakers for financial gain. Republican Rep. Robert Aderholt, the chairman of the appropriations subcommittee that funds the FDA, called Marathon's announcement a "tipping point." And he said the astronomical increase in deflazacort's price "makes me question whether the current construct of how the FDA approves orphan drugs does more harm than good if companies have found a way to game the system." At Monday's meeting, Marathon executives presented a slideshow on financial assistance for patients, as well as details on the drug's expected availability. Emflaza was set to hit the market by mid-March, said Eric Messner, vice president of sales and marketing for Marathon. Messner said the drug would cost $50,000 to $54,000 after rebates and discounts to insurers. "I know there has been a lot of discussion about that. How did we arrive at the price?" Messner said. An audience member interrupted Messner asking if the company had talked to patients and families about the price. He offered assurance that they did and said there would be an ongoing dialogue with patient groups. "We expect that patients will experience a low or zero out-of-pocket experience," he said. Late Monday, Marathon released an official statement and open letter to the Duchenne community, promising that "price should not be a barrier" for patients. "Put simply we expect patients will pay a standard copay of typically $20 or less per prescription. Edwards, the New Jersey mother, said she hopes President Trump will fulfill his vow to bring down high drug prices. Kaiser Health News' coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation.


WASHINGTON, Dec. 7, 2016 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) – applauded today's final Congressional approval of the 21st Century Cures legislation. The bill,...


News Article | September 20, 2016
Site: www.biosciencetechnology.com

Federal regulators on Monday granted tentative approval to the first drug for muscular dystrophy, following an intense public campaign from patients and doctors who pushed for the largely unproven medication. The approval came despite an internal dispute among Food and Drug Administration officials that ultimately had to be resolved by the agency's chief. Some staffers said there was little evidence the drug worked. Adding to the high-stakes decision, officials faced direct pleas from patients' families, politicians and physicians. The FDA cleared Sarepta Therapeutics' Exondys 51 for a rare form of Duchenne muscular dystrophy, a deadly inherited disease that affects boys. Company officials said the drug would cost about $300,000 per year. Like other makers of high-cost specialty drugs, Sarepta said it would offer financial assistance to help families get the drug. It's the first FDA approval for the degenerative condition, which causes muscle weakness, loss of movement and eventually death. The approval was based on a company study of just 12 boys. The agency is requiring Sarepta to conduct a larger study examining whether Exondys 51 results in improved movement and function for patients. If the study fails to shows it helps, the FDA said it could withdraw the drug. Duchenne's muscular dystrophy is a rare disease, affecting about 1 of every 3,600 boys worldwide and usually causing death by age 25, according to the National Institutes of Health. The new drug targets a genetic mutation that affects about 13 percent of Duchenne's patients. Previously there were no U.S.-approved drugs to fight the disease, though steroid drugs have been used to slow the loss of muscle strength. Pat Furlong, a patient advocate who lost two sons to the disease, called the announcement "an extraordinary win." "I think this is a collaborative effort that shows the FDA, companies and the patient community can work together toward a single goal, and that is improving the lives of patients," said Furlong, founder and president of Parent Project Muscular Dystrophy, a nonprofit that helped fund travel and other expenses related to the study. The FDA cleared Sarepta's drug under its accelerated approval program, reserved for drugs that show promising early results that have not been confirmed. The drug acts on a protein called dystrophin, which plays a role in the growth of muscle fibers. The 12-patient study showed an increase in dystrophin "that is reasonably likely to predict" benefit in some patients, the FDA said in its announcement "Accelerated approval makes this drug available to patients based on initial data, but we eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial," said Dr. Janet Woodcock, director of the FDA's drug center. At a public meeting in April, an outside panel of experts did not support the drug's approval, voting 7-3 that it did not show effectiveness in treating the disease. The FDA is not required to follow the advice of its advisory panels, though it often does. The final ruling was made by FDA Commissioner Dr. Robert Califf. According to agency memos posted to the FDA's website after the announcement, Califf said that he would defer to Woodcock's "judgment and authority" to approve the drug, despite opposition by several other agency staffers. FDA staff said at the April meeting that they "strongly encouraged" Sarepta to conduct a larger, more comprehensive study of its drug with a randomly selected control group of patients receiving a placebo - considered the gold standard of study design. "Ultimately, it appears to us that the FDA bowed to external pressure from patient advocates and others who demanded that a safe and potentially efficacious drug be made available," said Leerink Swann analyst Joseph Schwartz, in a note to investors. Shares of Sarepta Therapeutics rose more than 73 percent Monday to close at $48.94 in regular trading. The company is based in Cambridge, Massachusetts.


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

HACKENSACK, N.J., Feb. 28, 2017 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) announced the 500th patient application to their highly successful Decode Duchenne program. Decode Duchenne is a nationwide program to assist individuals with Duchenne muscular dystrophy in...


BURLINGTON, Mass. & LIESTAL, Switzerland--(BUSINESS WIRE)--Santhera Pharmaceuticals, a Swiss specialty pharmaceutical company focused on the development of innovative treatments for rare mitochondrial and neuromuscular diseases, announced that it will be establishing U.S. operations in Burlington, Mass. Santhera’s U.S. operations are being led by Todd Bazemore, Chief Operating Officer of Santhera Pharmaceuticals (USA), Inc. The U.S. operations will initially be staffed to focus on regulatory and clinical operations support, medical affairs, patient advocacy liaison and commercial strategy. “A strong presence in the U.S. is an important next step in our mission to advance novel treatments for Duchenne muscular dystrophy and other rare neuromuscular and mitochondrial diseases,” said Todd Bazemore, COO of Santhera U.S. “We chose the metropolitan Boston area for our U.S. operations because it is one of the main centers for the biotech and pharma business globally with a unique confluence of academia, life science companies, and clinical expertise.” Duchenne muscular dystrophy (DMD) is one of the most common and devastating types of muscle degeneration and leads to progressive muscle weakness starting at an early age. DMD is a genetic, degenerative disease that occurs almost exclusively in males with an incidence of up to 1 in 3,500 live male births worldwide.1 DMD is characterized by a loss of the protein dystrophin, ultimately resulting in muscle weakness and wasting and early morbidity and mortality due to respiratory failure. In DMD, the progressive respiratory muscle weakness results in respiratory symptoms such as inability to cough and clear mucus, frequent airway infections, sleep-disordered breathing and the need for assisted ventilation. Addressing the progressive loss of respiratory function is a hallmark in DMD and an urgent unmet medical need for these patients. In a Phase III clinical trial (DELOS) conducted in DMD patients not taking glucocorticoids, Santhera’s lead therapeutic candidate, idebenone, demonstrated a clinically significant slowing in the loss of respiratory function, regardless of the underlying genetic mutation. Santhera is now enrolling patients in a new Phase III trial (SIDEROS) in 60 centers across the United States and Europe to confirm the efficacy of idebenone in 266 patients who are currently taking a stable dose of glucocorticoids. The first patient was enrolled at the University of Kansas Medical Center (KUMC), Department of Neurology in September with Jeffrey Statland, MD. In the U.S., the SIDEROS trial will be conducted in 24 study centers with the goal of having this trial fully enrolled by end of 2017. If successful, this study will provide data that supports the use of idebenone in all DMD patients experiencing respiratory decline irrespective of their glucocorticoid use or genetic mutation. “Idebenone is generating a significant interest among researchers, physicians and the patient community. In slowing the loss of respiratory function in all patients irrespective of their mutational status, this drug candidate has the potential to expand the current treatment paradigm for DMD,” said Thomas Meier, PhD, CEO of Santhera. “Our presence here in the U.S. will enable us to expand our engagement with physicians, their patients, and families as the SIDEROS trial moves forward while we continue our efforts to find a regulatory path for early approval.” “There is high interest from investigators and the patient community in therapies that hold the promise of maintaining respiratory function in patients with DMD,” added Oscar Henry Mayer, MD, Medical Director of the Pulmonary Function Testing Laboratory at the Children’s Hospital of Philadelphia and Lead SIDEROS Investigator for the U.S. “A patient and caregiver survey conducted by Parent Project Muscular Dystrophy clearly demonstrated that the DMD community highly values treatment options for respiratory complications.” SIDEROS is a phase III, double-blind, randomized, placebo-controlled trial with idebenone in approximately 266 DMD patients receiving concomitant glucocorticoids. Patients with declining respiratory function on any stable glucocorticoid treatment scheme and irrespective of the underlying dystrophin mutation or ambulatory status will be eligible. Study participants will receive either idebenone (900 mg/day; given as 2 tablets 3 times a day with meals) or placebo for 78 weeks (18 months). The primary endpoint of the trial is the change from baseline to week 78 in forced vital capacity % predicted (FVC%p). Secondary endpoints include changes from baseline in % predicted peak expiratory flow (PEF%p), time to first 10% decline in FVC and change from baseline in inspiratory flow reserve. Patients completing the trial will be offered the opportunity to enroll in an open label extension study in which all patients receive idebenone. The study will be conducted at about 60 centers in the United States and Europe. Patients wishing to enroll in the study should contact their neuromuscular clinic physician. Further information about the study is available under www.clinicaltrials.gov. Duchenne muscular dystrophy (DMD) is one of the most common and devastating types of muscle degeneration and results in rapidly progressive muscle weakness. DMD is characterized by a loss of the protein dystrophin, leading to cell damage, impaired calcium homeostasis, elevated oxidative stress and reduced energy production in muscle cells. This results in progressive muscle weakness and wasting and early morbidity and mortality due to respiratory failure. Idebenone is a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1) capable of stimulating mitochondrial electron transport, reducing and scavenging reactive oxygen species (ROS) and supplementing cellular energy levels. DELOS was a phase III, double-blind, placebo-controlled trial which randomized 64 patients, not taking concomitant glucocorticoids, to receive either idebenone (900 mg/day) or matching placebo. The trial met its primary endpoint and demonstrated that idebenone can slow the loss of respiratory function and reduces bronchopulmonary complications. The statistically significant and clinically relevant outcomes of the phase III DELOS study were published: Buyse et al., The Lancet 2015, 385:1748-1757; McDonald et al., Neuromuscular Disorders 2016, 26: 473–480 and Buyse et al., Pediatric Pulmonology 2016: http://dx.doi.org/10.1002/ppul.23547. The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) and the Swiss regulatory authorities Swissmedic are currently assessing a Marketing Authorization Application (MAA) for idebenone under the name RAXONE® in DMD patients with respiratory function decline who are not taking concomitant glucocorticoids. The indication would include patients who previously were treated with glucocorticoids or in whom glucocorticoid treatment is not desired, not tolerated or is contraindicated. Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative pharmaceutical products for the treatment of orphan mitochondrial and neuromuscular diseases. Santhera's lead product RAXONE® (idebenone) is authorized in the European Union, Norway, Iceland and Liechtenstein for the treatment of Leber's hereditary optic neuropathy (LHON). For Duchenne muscular dystrophy (DMD), the second indication for RAXONE®, Santhera has filed a Marketing Authorization Application (MAA) in the European Union and Switzerland. In collaboration with the U.S. National Institute of Neurological Disorders and Stroke (NINDS) Santhera is developing RAXONE® in a third indication, primary progressive multiple sclerosis (PPMS), and omigapil for congenital muscular dystrophy (CMD), all areas of high unmet medical need. For further information, please visit the Company's website www.santhera.com. This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. These statements could be affected by, among other things, risks and factors referred to in the Risk Factors section on Santhera’s web site (http://www.santhera.com/investors-and-media/investor-toolbox/risk-factors). Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements. 1 National Institutes of Health, U.S. National Library of Medicine, Genetics Home Reference, Duchenne and Becker muscular dystrophy, https://ghr.nlm.nih.gov/condition/duchenne-and-becker-muscular-dystrophy#statistics

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