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... The Food and Drug Administration (FDA) on Thursday announced expanded approval for a gene therapy to treat Duchenne muscular dystrophy (DMD) -- despite the fact that it failed a Phase III clinical trial last year and that the approval came over the objections of three of FDA's own expert review teams and two of its directors.

In fact, the decision to expand the approval of the therapy -- called Elevidys (delandistrogene moxeparvovec-rokl) -- appears to have been decided almost entirely by Peter Marks, Director of the FDA's Center for Biologics Evaluation and Research.

Elevidys initially gained an FDA approval last year, also over objections from staff.

The therapy intravenously delivers a transgene that codes for select portions of a protein called dystrophin in healthy muscle cells; the protein is mutated in patients with DMD. Last year's initial approval occurred under an accelerated approval process and was only for use in DMD patients ages 4 and 5 who are able to walk. In the actions Thursday, the FDA granted a traditional approval for the therapy and opened access to DMD patients of all ages, regardless of ambulatory status.

"Today's approval broadens the spectrum of patients with Duchenne muscular dystrophy eligible for this therapy, helping to address the ongoing, urgent treatment need for patients with this devastating and life-threatening disease," Marks said in the announcement Thursday. "We remain steadfast in our commitment to help advance safe and effective treatments for patients who desperately need them."

Criticism

The move, which follows a string of controversies in recent years of the FDA issuing questionable approvals over the assessments of advisors and its own staff, has quickly drawn criticism from agency watchers. ...