Sarepta was stunned by the rejection of Vyondys 53. Now it's stunning everyone with a surprise accelerated approval
Sarepta has a friend in the FDA after all. Four months after the agency determined that it would be wrong to give Sarepta an accelerated approval for their Duchenne MD drug golodirsen, regulators have executed a stunning about face and offered the biotech a quick green light in any case.
It was the agency that first put out the news late Thursday, announcing that Duchenne MD patients with a mutation amenable to exon 53 skipping will now have their first targeted treatment: Vyondys 53, or golodirsen. Having secured the OK via a dispute resolution mechanism, the biotech said the new drug has been priced on par with their only other marketed drug, Exondys 51 — which for an average patient costs about $300,000 per year, but since pricing is based on weight, that sticker price can even cross $1 million.
Sarepta shares $SRPT surged 23% after-market to $124.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 151,200+ biopharma pros reading Endpoints daily — and it's free.