This pathway provides earlier patient access to promising new drugs while the company conducts clinical trials to verify the predicted clinical benefit.The accelerated approval of Exondys 51 is based on the surrogate endpoint of dystrophin increase in skeletal muscle observed in some Exondys 51-treated patients. Sarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for 6 Limb-girdle muscular dystrophy diseases (LGMD), Charcot-Marie-Tooth (CMT), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development. Citi Group analyst Joel Beatty said it was now more likely Sarepta will be able to win approval for Duchenne treatment casimersen and gene therapies it is developing. Garthwaite, a professor at Northwestern’s Kellogg School of Management, suggested payers should negotiate some sort of reduced pricing or pay a penalty for drugs approved through the accelerated process that turn out to not be effective. The required study is designed to assess whether Exondys 51 improves motor function of DMD patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping. Reprints. Jefferies analyst Michael Yee said the FDA’s change of heart could have implications for Biogen Inc’s (Biogen scrapped development of aducanumab in March, but seven months later surprised investors by saying it would pursue approval after analyzing additional data that showed it had some benefit. The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). “In rare diseases, new drug development is especially challenging due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders. HC Wainwright analyst Debjit Chattopadhyay found the FDA reversal to be confounding. An official website of the United States government The company’s chief executive in November said it expects to provide additional updates to regulators by the end of next year. 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 that the company must conduct after approval.”DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. On Dec. 12, the US Food and Drug Administration (FDA) granted accelerated approval to golodirsen (Vyondys 53) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. REUTERS/Jason Reed/File Photo Sarepta said it addressed the kidney toxicity issue in the drug’s label, which recommends monthly monitoring of patients. The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). It is the second exon-skipping, disease-modifying drug to treat DMD, the most common childhood form of muscular dystrophy. In 2016, the FDA approved the company’s first DMD treatment, Exondys 51, bowing to intense pressure from patient advocates even as an outside panel of experts and the agency’s own reviewers questioned the drug’s effectiveness. The surprise approval was not the first for Sarepta. Sarepta in a statement said it had appealed the FDA rejection through a formal dispute resolution request. Patients typically succumb to the disease in their 20s or 30s; however, disease severity and life expectancy vary.Exondys 51 was approved under the accelerated approval pathway, which provides for the approval of drugs that treat serious or life-threatening diseases and generally provide a meaningful advantage over existing treatments. Healthcare economist Craig Garthwaite said he believes the FDA’s desire to make new medicines available much faster will be problematic. Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD.“Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “We’re allowing things to come to market with far less evidence that they work and having people effectively pay the price for those drugs as if they did,” he said. Sarepta already has a marketed DMD drug, Exondys 51, which performed well since launch with sales set to reach $300 million in 2018. Sarepta must still submit additional data on the efficacy of Exondys to the FDA. The approval is contingent on results of a post-approval trial expected to conclude by 2024, the FDA said. It is the most common type of People with DMD progressively lose the ability to perform activities independently and often require use of a wheelchair by their early teens. A clinical benefit of Exondys 51, including improved motor function, has not been established. Company shares, which were cut by half after the August rejection, jumped to $132.05 on Friday from a Thursday close of $100.46. CAMBRIDGE, Mass., June 26, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the Company has completed the submission of a rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for casimersen (SRP-4045). If the drug “was not approvable four months ago, it cannot suddenly become approvable without significant new data, and potentially reflects some contrived decision making within the agency,” Chattopadhyay said. Approval under this pathway can be based on adequate and well-controlled studies showing the drug has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit to patients (how a patient feels or functions or whether they survive).


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