Posted by Michael Wonder on 28 Sep 2016
Why Sarepta's $300K price for DMD drug invalidates reasons for accelerated approval
26 September 2016 - The Editor of BioCentury argues the case for why Sarepta Therapeutics should not have been allowed to have it both ways.
Despite the fact that Sarepta Therapeutics Inc. did many, many things wrong in the development of Exondys 51 eteplirsen, in early May we took the position that FDA should grant accelerated approval to treat Duchenne muscular dystrophy.
We still believe accelerated approval was the right decision. Unfortunately, judging by the $300,000 annual net cost for a drug that at this point is only "reasonably likely" to produce a clinical benefit, it looks like Sarepta is continuing to get it wrong.
Unless the company engages in risk-sharing or pay-for-performance deals with payers, the high price will prevent broad access to the drug — and giving patients broad access was the best reason to grant this drug accelerated approval.
