Recently, the US Food and Drug Administration has approved the first gene therapy to treat a rare and devastating muscle disease but limited the approval to kids ages 4 and 5 based on available evidence.
The US Food and Drug Administration hasĀ approvedĀ the first gene therapy to treat a rare and devastating muscle disease but limited the approval to kids ages 4 and 5 based on available evidence.
The drug, from biotech company Sarepta Therapeutics, will need to prove in an ongoing clinical trial that it improves physical function and mobility in patients with Duchenne muscular dystrophy in order to stay on the market, the FDA said Thursday. The therapy was approved under the accelerated approval pathway, which clears medicines for diseases where theyāre urgently needed based on data suggesting theyāre likely to confer clinical benefits.
The group for whom the drug was approved, children ages 4 and 5, is about 6% of the population of people with Duchenne muscular dystrophy, according to Brian Abrahams, an analyst with Wall Street firm RBC Capital Markets. He had estimated that Sarepta would price the drug at $2.8 million per patient, which could bring in $2.1 billion in revenue from the initial group eligible for the drug.
While eye-popping, such a pricetag isnāt out of step with other one-time gene therapies, which have topped $3 million per patient in recent years. But Elevidys will become the second most expensive drug in the world, after the $3.5 million hemophilia treatment Hemgenix.
(CNN)