Sarepta says FDA may approve gene therapy for smaller population, shares fall

May 24 (Reuters) – Sarepta Therapeutics Inc (SRPT.O) said on Wednesday the U.S. drug regulator could potentially approve the company’s gene therapy for a muscle wasting disorder for a smaller patient group initially, sending the company’s shares down 10%.

The Food and Drug Administration (FDA) also deferred an accelerated approval decision related to the company’s gene therapy for Duchenne muscular dystrophy (DMD) to June 22 from May 29, adding that the regulator needs more time to complete the review of SRP-9001.

The FDA indicated it could potentially grant an accelerated approval for the therapy, called SRP-9001, initially for use in Duchenne patients aged 4 and 5. The drug was tested in DMD patients who could walk, with the mean age group of about 7 years.

The smaller-than-expected patient age group is a net negative for the company in the near term, Credit Suisse analyst Judah Frommer said.

Sarepta’s announcement on Wednesday followed a meeting of FDA’s external advisers less than two weeks ago, where the panel narrowly backed an accelerated approval for the gene therapy with 8-6 votes.

DMD is a muscle-wasting disorder that is estimated to affect one in 3,500 male births worldwide, according to the National Organization for Rare Disorders.

Sarepta’s shares were trading at $132.39 before the bell on Wednesday.

Reporting by Aditya Samal and Leroy Leo in Bengaluru; Editing by Shinjini Ganguli

Our Standards: The Thomson Reuters Trust Principles.

Source: Reuters