Proactive Investors - Sarepta Therapeutics Inc (NASDAQ:SRPT) shares soared almost 35% after the US Food and Drug Administration (FDA) approved the expanded use of its gene therapy Elevidys for patients with Duchenne muscular dystrophy (DMD).
DMD is a rare genetic condition which causes weakness and the wasting away of the body’s muscles, with symptoms including trouble walking and running, falling frequently, fatigue and learning disabilities or difficulties.
The regulator expanded the drug’s approval to include ambulatory and non-ambulatory individuals four years of age and older with DMD with a confirmed mutation in the DMD gene.
Elevidys had been previously approved for ambulatory individuals four through five years of age with DMD with a confirmed mutation in the DMD gene.
The FDA also granted accelerated approval for non-ambulatory patients and, consistent with this, Sarepta said it will conduct and submit the results of a randomized, controlled trial to verify and confirm the clinical benefit of Elevidys in patients with DMD who are non-ambulatory.
“Representing many years of dedicated research, development, investment and creative energy, the expansion of the Elevidys label to treat Duchenne patients aged four and above, regardless of ambulatory status, is a defining moment for the Duchenne community,” Sarepta CEO Doug Ingram said in a statement.
FDA Center for Biologics Evaluation and Research director Dr Peter Marks added: “[This] approval broadens the spectrum of patients with Duchenne muscular dystrophy eligible for this therapy, helping to address the ongoing, urgent treatment need for patients with this devastating and life-threatening disease.”
Shares of Sarepta were up 34.9% at $166.20 in early trade on Friday.