CBER Director Says Muscular Dystrophy Gene Therapy Deserved Accelerated Approval

The FDA’s June 22 Accelerated Approval of Sarepta’s gene therapy Elevidys for Duchenne muscular dystrophy (DMD) is appropriate, despite an apparent disagreement within the department, according to CBER Director Peter Marks.

In an unusual publicly released letter describing CBER’s decision-making process, Marks said Elevidys’ phase 2 data show that the engineered protein is associated with improved clinical outcomes in patients with the progressive genetic disorder, which has no other treatment.

“After carefully considering the totality of the available data and information, I have determined that accelerated approval is appropriate,” he said.

Although the committee voted 8-6 in favor of accelerated approval, “even some of the advisors who voted in favor of approval ... expressed concerns about the strength of the clinical evidence,” Marks wrote.

The division’s staff reviewers apparently feel differently, Marks said in the letter.

“In reviewing the documentation provided in the CBER Clinical Review, Clinical Pharmacology Review, and the Statistical Review, which do not recommend approval based upon their overall evaluation of the data submitted, as well as in reviewing the CDER Division of Pharmacometrics consult, I agree in large part with the efficacy assessments provided,” the letter said.

Read the letter here.

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