FDA Greenlighted Sarepta’s Gene Therapy for DMD

FDA Greenlighted Sarepta's Gene Therapy for DMD
FDA Greenlighted Sarepta's Gene Therapy for DMD. Credit | REUTERS

United States – Sarepta Therapeutics Inc. had its gene therapy permitted by the U.S. FDA for individuals with Duchenne Muscular Dystrophy who were four years old and above on Thursday; the company’s stock surged 36% in after-hours trading.

The agency provided regular approval to patients who were four years old and above who could walk, while accelerated approval was granted to patients who could not walk, as reported by Reuters.

Approval Details and Therapy Challenges

Elevidys gene therapy had faced issues of regulatory hold and efficacy in earlier years and, especially after it failed to meet some of the basic targets of its final phase clinical trial.

Motor function in patients 4 to 7 years old did not improve significantly more with High-Dose vs. low-dose therapy vs. placebo at 52 weeks, but all the other secondary goals in the trial were met, and no new safety concerns were revealed.

Patient and Expert Perspectives

According to Debra Miller, founder and CEO of CureDuchenne, which is a nonprofit organization carrying out patient support and research funding for DMD, told Reuters before the decision that goals, for instance, walk tests and endurance while at play, became sometimes challenging to record.

“I believe, from a parent’s standpoint, you could watch your child deteriorate and hope for something better, or you can take advantage of something now,” Miller said.

Impact and Future Steps

Muscular Dystrophy affects approximately one in every 3,500 male births globally, as per the National Organization for Rare Disorders. It affects skeletal and heart muscles, and the condition deteriorates rapidly with age; the patient is likely to die from it by the time he/she is 25 years old, as postulated by the Cleveland Clinic.

The widened approval was a ‘’homerun scenario,’’ J. P. Morgan analysts said in a note.

The FDA approved this therapy in June last year on an accelerated basis. It is the first of its kind for DMD in patients aged four to five who can still walk. Premarket approval enables the agency to approve treatment before final evidence confirms that it is of value.

The expanded approval would probably mean that Sarepta needs to increase its output, although as of the time the news was published, CEO Doug Ingram said the company is ‘in a good place from a manufacturing standpoint.’

Further authorization for the application of the therapy in Duchenne patients who cannot ambulate may depend however on affirming the clinical effectiveness in a confirmatory trial, as reported by Reuters.

Continued Research and Licensing

The company has stated that Elevidys should not be used in patients who have mutations in the DMD gene.

Roche (ROG. S) has licensed the gene therapy for commercialization in the other markets than the United States from Sarepta.