Sarepta’s New Treatment for Duchenne Muscular Dystrophy Approved by FDA, $1 Million Yearly Cost Despite Lack of Confirmation Studies

March 3, 2021

Sarepta Therapeutics has created a third drug for use in Duchenne Muscular Dystrophy (DMD), termed Amondys 45. It recently received approval from the FDA. The pharma company’s three DMD drugs: Amondys 45, Exondys 51, and Vvondys 53, will reach approximately a third of patients with DMD.  However, Sarepta has been slow to complete confirmation studies. As such, ICER has been critical regarding these drugs, which could hit a yearly cost of $1 million. This year, Sarepta’s revenue is expected to increase by 30% to reach $686 million.

“All of Sarepta’s drugs have been granted accelerated approval by the FDA based on their ability to produce a tiny amount of the muscle protein dystrophin missing in these patients, even though there is no established proof that more dystrophin improves muscle function or slows the progression of the disease.” Read more here.

(Source: Adam Feuerstein, STAT News, 2/25/21)

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