FDA approves gene therapy drug

(CNN/KYMA, KECY) – A panel of outside advisers to the Food and Drug Administration voted to grant accelerated approval to an experimental gene therapy.

SRP-9001 treats Duchenne muscular dystrophy in a single dose.

However, it hasn't been clinically proven and the FDA has expressed safety concerns.

But, parents of children who have received it got the votes they needed.

Parents told stories about the therapy keeping their kids walking longer than if they hadn't received it.

Kids with the disease generally lose the ability to walk by their teens.

The advisers ultimately voted 8 to 6 to put the drug on a path to go to market without robust clinical evidence.

The FDA does not have to follow that recommendation.

The agency is expected to make a final decision by the end of the month.