A self-test for H.I.V. in Harare, Zimbabwe. The every-six-months injection was found to provide better protection than the current oral drug for what’s called pre-exposure prophylaxis, also taken as a daily pill.
Enlarge/ Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research within the Food and Drug Administration on March 18, 2021 in Washington, DC. (credit: Getty | Susan Walsh)
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.
In high-tech labs, workers are generating data to train A.I. algorithms to design better medicine, faster. But the transformation is just getting underway.
Chips in a container at Terray Therapeutics in Monrovia, Calif. Each of the custom-made chips has millions of minuscule wells for measuring drug screening reactions quickly and accurately.
In high-tech labs, workers are generating data to train A.I. algorithms to design better medicine, faster. But the transformation is just getting underway.
Chips in a container at Terray Therapeutics in Monrovia, Calif. Each of the custom-made chips has millions of minuscule wells for measuring drug screening reactions quickly and accurately.
The agency’s staff analysis suggests that approval of the illegal drug known as Ecstasy for treatment of PTSD is far from certain, with advisers meeting next week to consider the proposed therapy.
A seizure of the drug MDMA, known as Ecstasy or molly. It and other psychoactive drugs are still classified as illegal drugs with a potential for abuse.
Martha Warren, 65, of Westerly, R.I., found out last year that she had small cell lung cancer, and joined the tarlatamab clinical trial. She said she now feels as good as before her diagnosis.
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