A BMO Capital survey revealed that about 90% of physicians will continue prescribing Elevidys gene therapy to Duchenne muscular dystrophy (DMD) patients, despite recent deaths.
The survey of 30 physicians further revealed that approximately 35% of respondents are prescribing Elevidys at a similar rate as before the incidents. In a scenario where an ambulatory Elevidys patient death occurs — the biggest current risk for Sarepta — more than 40% of physicians would still continue prescribing the therapy without waiting for safety updates.
This year, Sarepta has reported two fatal acute liver failure cases in non-ambulatory patients, out of a total of approximately 140 non-ambulatory patients treated with Elevidys globally to date.
Back in July, Sarepta said it would voluntarily and temporarily pause all shipments of Elevidys in the U.S., following a public dispute with the FDA. A week later, the agency notified Sarepta that it could lift the voluntary pause for ambulatory patients.
DMD disease progression tends to follow a predictable pattern, with initial symptoms appearing between 2 and 5 years of age, progressing to loss of ambulation by the early teens and subsequent development of cardiorespiratory complications. Respiratory and cardiac complications are the main causes of death in patients with DMD, which carries a life expectancy of around 30 years.
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