Despite a restricted FDA label, Sarepta Therapeutics has seen early commercial success with its Duchenne muscular dystrophy (DMD) gene therapy Elevidys. This year, the company aims to dramatically grow the drug's reach.
The FDA is set to decide on a potential label expansion—as well as a potential conversion of the drug’s accelerated approval to a traditional one—by June 21. Out of the two moves, Sarepta is prioritizing the “broadest possible label” over the potential accelerated approval conversion, CEO Douglas Ingram said on the company's fourth-quarter and full-year earnings conference call.
With two quarters of the drug’s launch under its belt, the company is finally in a spot that it had to “move heaven and Earth to reach,” Ingram said.
The company seeks a new label to cover "the treatment of DMD patients with a confirmed mutation in the DMD gene.” Currently, the drug is only approved to treat ambulatory patients ages 4 to 5.
The restricted patient population presents several “unique executional challenges,” Chief Customer Officer Dallan Murray noted on the call.
For one, many patients in the age group are not yet diagnosed, considering the average age of DMD diagnosis is 5 years old in the U.S. Families then need to digest the diagnosis and assess their treatment options before starting the pre-treatment process for Elevidys, which includes antibody testing.
Essentially, securing access to the gene therapy in a tight window before aging out of eligibility is “a race against time,” Murray said.
Plus, the currently approved population is relevantly small in terms of total numbers, meaning Sarepta will be “quickly” working through its eligible patients over the first half of this year.
Despite missing the primary endpoint in a pivotal study, Ingram repeatedly emphasized that “the science justifies” the removal of the label restriction.
“It's the right answer from the science. It's the right answer from a policy perspective. And most of all, it's the right answer for patients living with Duchenne muscular dystrophy, which I hope will be all of our north stars in this regard,” the CEO said.
Still, even the restricted population has been fruitful for Sarepta. Elevidys brought in total sales of $200.4 million last year, with $131.3 million coming from the year’s fourth quarter. Elevidys won its FDA nod in June 2023.
The therapy’s early market success stands to prove that “gene therapies can be commercially viable,” Murray noted.