{"id":9376,"date":"2019-12-13T11:51:32","date_gmt":"2019-12-13T11:51:32","guid":{"rendered":"https:\/\/laforcedmd.com\/?p=9376"},"modified":"2022-05-03T18:06:27","modified_gmt":"2022-05-03T18:06:27","slug":"fda-grants-accelerated-approval-to-vyondys-53","status":"publish","type":"post","link":"https:\/\/laforcedmd.com\/en\/fda-grants-accelerated-approval-to-vyondys-53\/","title":{"rendered":"FDA grants accelerated approval to Vyondys 53"},"content":{"rendered":"

Sarepta Therapeutics Announces FDA Approval of VYONDYS 53\u2122 (golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53<\/h4>\n

Source: Sarepta Therapeutics, Inc., Dec 12, 2019 – Read the original news here<\/a><\/span>–\u00a0<\/em><\/span><\/p>\n

The U.S. Food and Drug Administration granted accelerated approval to Vyondys 53 (golodirsen)<\/strong> injection to treat Duchenne muscular dystrophy (DMD) patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping<\/strong>. It is estimated that about 8% of patients<\/strong> with DMD have this mutation.<\/p>\n

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Billy Dunn<\/span>, M.D., acting director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research<\/strong>\u00a0– “The FDA recognizes the urgent need for new medical treatments for serious neurological disorders and we have a long-standing commitment to working with researchers, drug companies and patients to facilitate the development and approval of treatments for rare diseases. With today’s accelerated approval, patients with Duchenne muscular dystrophy who have a confirmed mutation of the dystrophin gene amenable to exon 53 skipping will now have available the first treatment targeted specifically for this disease subtype. Use of the accelerated approval pathway will make Vyondys 53 available to patients based on initial data and we look forward to learning more about the drug’s clinical benefit from the ongoing confirmatory clinical trial.”<\/p><\/blockquote>\n

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Doug Ingram, president and chief executive officer, Sarepta –<\/strong> “Today is monumental for Sarepta and, more importantly, for the DMD community. VYONDYS 53, our second approved exon-skipping RNA therapy for DMD, may treat up to 8% of the DMD community, representing those patients who have a confirmed exon 53 amenable mutations.\u00a0 Along with EXONDYS 51\u00ae (eteplirsen), we now offer treatment options for approximately 20% of those with DMD in the U.S.”<\/p><\/blockquote>\n

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Pat Furlong, founding president and chief executive officer, Parent Project Muscular Dystrophy (PPMD link here<\/a><\/span>)<\/strong> – “With the approval of VYONDYS 53, up to another 8% of Duchenne families will have a therapy to treat this devastating disease. For 25 years, PPMD has been working with researchers, clinicians, industry, and the Duchenne community to find treatments for all people living with Duchenne. And while we need to ensure that these approved therapies are accessible for patients, today we celebrate this approval and thank Sarepta for their continued leadership in the fight to end Duchenne.”<\/p><\/blockquote>\n

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More about Vyondys 53 (golodirsen)<\/h4>\n

Like Exondys 51, golodirsen, which Sarepta hopes to sell under the name Vyondys 53, is designed to treat a group of Duchenne patients with a particular type of mutation. Exondys 51 works for about 13% of DMD patients\u2014those whose disease is amenable to exon 51 skipping. If approved, golodirsen would offer treatment to patients with a mutation in exon 53\u2014about 8% of the DMD population.<\/strong><\/p>\n

VYONDYS 53 is priced at parity to EXONDYS 51, the price of which has not increased since its launch in 2016. Patients and physicians can access more information at\u00a0www.SareptAssist.com<\/a><\/span>\u00a0or by calling 1-888-727-3782.<\/p>\n

Important Safety Information for VYONDYS 53<\/a><\/span><\/p>\n

Approval of Vyondys 53<\/h4>\n

Vyondys 53 was approved under the accelerated approval pathway, which provides for the approval of drugs that treat serious or life-threatening diseases and generally offer a meaningful advantage over existing treatments. Approval under this pathway can be based on adequate and well-controlled studies showing the drug affects a surrogate endpoint that is reasonably likely to predict clinical benefit to patients. This pathway provides earlier patient access to promising new drugs while the company conducts clinical trials to verify the predicted clinical benefit.<\/p>\n

The accelerated approval of Vyondys 53 is based on the\u00a0surrogate endpoint<\/a>\u00a0of an increase in dystrophin production in the skeletal muscle observed in some patients treated with the drug. The FDA has concluded that the data submitted by the applicant demonstrated an increase in dystrophin production that is reasonably likely to predict clinical benefit in patients with DMD who have a confirmed mutation of the dystrophin gene amenable to exon 53 skipping<\/strong>. A clinical benefit of the drug, including improved motor function, has not been established. In making this decision, the FDA considered the potential risks associated with the drug, the life-threatening and debilitating nature of the disease and the lack of available therapy. Read more here.<\/a><\/span><\/p>\n

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The Status<\/h4>\n