Of four new "exon-skipping" therapies - intended for a smaller group of patients with specific genetic mutations - three are from Sarepta. In a mid-stage trial, Sarepta's gene therapy was able to produce a mini version of the dystrophin protein needed to help keep muscles intact, but did not improve patient clinical outcomes like their ability to walk and stand.Ī late-stage study to confirm the treatment's effectiveness is underway and initial data from it is expected by December.Ĭurrent options for DMD patients are limited. ![]() Currently, approved steroids and some therapies for specific genetic mutations only control symptoms and slow disease progression. The agency's decision comes after several delays and questions over the effectiveness of the therapy, which will be sold under the brand name Elevidys.Įlevidys could change the way that DMD patients, who rarely survive beyond their thirties, are treated. The world's most expensive treatment, a gene therapy for bleeding disorder hemophilia from Australian drugmaker CSL Ltd (CSL.AX), carries a list price of $3.5 million. The therapy's wholesale acquisition cost is $3.2 million, CEO Douglas Ingram said on a conference call with investors. ![]() It was initially seeking approval for all DMD patients who can walk. ![]() Sarepta said on Thursday the Food and Drug Administration had approved the treatment for children aged between 4 and 5 years who can walk. health regulator has granted accelerated approval to Sarepta Therapeutics' (SRPT.O) first-of-its-kind gene therapy for Duchenne muscular dystrophy (DMD), an inherited progressive muscle-wasting disorder that almost always affects young boys.
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