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Pignatti F.,The European Medicines Agency | Ashby D.,Imperial College London | Brass E.P.,University of California at Los Angeles | Eichler H.-G.,The European Medicines Agency | And 14 more authors.
Clinical pharmacology and therapeutics | Year: 2015

Structured frameworks for benefit-risk analysis in drug licensing decisions are being implemented across a number of regulatory agencies worldwide. The aim of these frameworks is to aid the analysis and communication of the benefit-risk assessment throughout the development, evaluation, and supervision of medicines. In this review, authors from regulatory agencies, pharmaceutical companies, and academia share their views on the different frameworks and discuss future directions. © 2015 American Society for Clinical Pharmacology and Therapeutics.

News Article | March 17, 2015
Site: techcrunch.com

Nintendo is being unusually candid about its product roadmap, with news that it will work on mobile titles with partner DeNA, and a reveal of its next console project – or at least the fact that it’s working on one. Nintendo’s Chief Executive Satoru Iwata told a press conference today that its next-generation console will be called NX, and is currently under development with a “brand new concept.” What Nintendo didn’t do was give us any indication of what NX is, or when it might arrive. Iwata did say that we’ll learn more about it next year, meaning it could make an initial appearance at E3 2016 or at its own dedicated unveiling. The news might be a candid admission that Wii U hasn’t been the home console Nintendo had hoped for, which might lead to a fast-forward refresh in the form of the NX, vs. an update cycle more in time with its competitors Sony and Microsoft. Nintendo’s purpose with this press conference was to elaborate on its decision to ship smartphone games and describe the form they would take (they won’t just port existing games to mobile platforms, Iwata says, since that would “ruin the value of Nintendo’s IP”). The NX mention in this context seems like a reassurance regarding the company’s continued plans to innovate in the dedicated gaming market, but if Iwata is hoping for a 2016 reveal, that would still be an accelerated refresh cycle vs. previous console generations. That “brand new concept” tidbit is interesting: It could just be straightforward marketing bluster, but Nintendo also probably knows it needs to wow with a Wii U follow-up. It’d be interesting to see them offer a take on VR, given its growing popularity among hardware companies, but it could also be a machine more in line with the competition in terms of power under the hood and media service offerings.

News Article | October 23, 2015
Site: news.yahoo.com

By Ben Hirschler LONDON (Reuters) - A first-in-class drug from Amgen based on a tumour-killing virus was given a green light by European regulators on Friday, paving the way for its approval within a couple of months. The European Medicines Agency (EMA) said its experts had recommended approval of Imlygic, also known as talimogene laherparepvec or "T-Vec", for treating melanoma, making it another option among several new drugs for the most deadly form of skin cancer. “Viral immunotherapy represents a completely new way of treating cancer, so it’s extremely exciting to see T-Vec become the first treatment of this type to gain the green light from European regulators," said Paul Workman, Chief Executive of The Institute of Cancer Research, London.

A vector, which is used to transport the healthy gene into the patient's cells, is seen in this undated handout image provided by GlaxoSmithKline on April 1, 2016. REUTERS/Grant Thompson/GSK/Handout via Reuters More LONDON (Reuters) - The world's first life-saving gene therapy for children, developed by Italian scientists and GlaxoSmithKline, has been recommended for approval in Europe, boosting the pioneering technology to fix faulty genes. The European Medicines Agency (EMA) said on Friday it had endorsed the therapy, called Strimvelis, for a tiny number of children with ADA Severe Combined Immune Deficiency (ADA-SCID) for whom no matching bone marrow donor is available. Around 15 children a year are born in Europe with the ultra-rare genetic disorder, which leaves them unable to make a type of white blood cell. They rarely survive beyond two years unless their immune function is restored with a suitable bone marrow transplant. SCID is sometimes known as “bubble baby” disease, since children born with it have immune systems so weak they must live in germ-free environments. Strimvelis is expected to secure formal marketing authorization from the European Commission in a couple of months, making it the second gene therapy to be approved in Europe, after UniQure's Glybera, which treats a rare adult blood disorder. The U.S. Food and Drug Administration has yet to approve any gene therapies but a growing number of U.S. biotech companies, such as Bluebird Bio, have products in development. Other large pharmaceutical companies are also eyeing the field, including Bristol-Myers Squibb, which has a tie-up with UniQure. Research into gene therapy goes back a quarter of a century but the field has experienced many setbacks, including the high-profile death of an American patient in 1999 and some disastrous clinical trial results in the late 1990s and early 2000s. Now, though, optimism is building, helped by the discovery of better ways to carry replacement genes into cells. Martin Andrews, head of GlaxoSmithKline's rare diseases unit, believes the technology is proving itself, although it remains at an early stage of development. "We're on page one of chapter one of a new medicine text book," he told Reuters. A host of challenges still need to be overcome, including the complexity of delivering a product like GSK's new treatment, which requires bone marrow cells to be taken from the patient, processed and injected back. Trickiest of all may be pricing, given the tiny market for a therapy like Strimvelis. UniQure's Glybera made history in 2014 as the first drug to carry a $1 million price tag. GSK is not putting a price on its product but a source close to the company said that, if approved, Strimvelis would cost “very significantly less than $1 million”. GSK has several other gene therapies under development with researchers at Fondazione Telethon and Ospedale San Raffaele in Italy, including treatments for metachromatic leukodystrophy and Wiskott-Aldrich syndrome that could be submitted for regulatory approval in the next couple of years. Its Strimvelis treatment for ADA-SCID is also being lined up for submission to U.S. regulators, although Andrews said this would not happen before the end of next year.

News Article
Site: www.rdmag.com

The European Medicines Agency (EMA) launched a new initiative Monday called PRIME, which is designed to help expedite the regulatory process for drug makers working on promising medications for rare diseases. PRIME stands for PRIority MEdicines. Its first phase requires participants submit an application.

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