FILE PHOTO: The headquarters of the U.S. Food and Drug Administration (FDA) is seen in Silver Spring, Maryland November 4, 2009. REUTERS/Jason Reed/File Photo
(This December 12 story corrects headline and paragraph 1 of the Dec. 12 story to remove reference to “early approval”)
(Reuters) – The U.S. Food and Drug Administration on Thursday approved Sarepta Therapeutics Inc’s second treatment for Duchenne muscular dystrophy (DMD).
The company’s shares surged about 28% to $129 in extended trading after being halted ahead of the news.
The FDA in August had declined to approve the company’s marketing application for the drug, citing safety concerns including the risk of infection and kidney toxicity.
The health regulator said on Thursday the company has to conduct trial to confirm the drug’s clinical benefit. The company said it expects the trial to conclude by 2024.
DMD is a rare, genetic disorder that hampers muscle movement mainly in men, affecting one in every 3,500 to 5,000 males. Many patients die at a relatively young age from the disorder, which is caused by the absence of dystrophin, a muscle building protein.
The company said the new treatment Vyondys 53 is priced at parity to its first DMD drug, Exondys 51, which is priced at about $300,000 per patient per year.
Reporting by Dania Nadeem, Anuron Kumar Mitra and Shivani Singh in Bengaluru; Editing by Sriraj Kalluvila and Uttaresh.V