FDA approves previously rejected Sarepta treatment for Duchenne

The FDA approved Sarepta Therapeutics' drug to treat Duchenne muscular dystrophy Dec. 12 after rejecting the same drug in August. 

In the agency's approval notice, it did not specify why it rejected the drug in the first place or what caused it to reverse course and approve it now. 

Duchenne is a rare genetic disorder that causes progressive muscle deterioration and weakness. People typically notice the first symptoms of it in infants between the ages of 3 and 5, and symptoms worsen over time. 

The drug, called Vyondys 53, is designed to treat about 8 percent of Duchenne patients whose disease is caused by a specific DNA error. In clinical trials for the drug, it produced a small increase in a muscle protein called dystrophin that is normally missing in Duchenne patients. 

Sarepta, a Cambridge, Mass.- based drugmaker, has yet to prove that Vyondys 53 can improve muscle function or slow progression of the disease, according to STAT

When the FDA rejected the drug in August, Sarepta said it was because of the risk of infections from infusion ports and kidney toxicity seen in animal experiments. In a separate Dec. 12 news release from Sarepta, the company said it had filed an appeal with the FDA and resolved those concerns, STAT reported. 

The drugmaker said it will price Vyondys 53 similarly to its other Duchenne drug, Exondys 51, which can cost as much as $1 million per year, depending on the dosage needed. 

Vyondys 53 was approved under the FDA's accelerated approval process,  designed for drugs that show promising results but have not been confirmed. 

"In making this decision, the FDA considered the potential risks associated with the drug, the life-threatening and debilitating nature of the disease and the lack of available therapy," the FDA wrote. 

Under the approval conditions, the FDA is requiring Sarepta to conduct a clinical trial to confirm the drug's clinical benefit and to assess whether it can improve motor function in Duchenne  patients. If it fails, the FDA may withdraw approval. 

Read the full article here

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