Back in late 2014, BioMarin ($BMRN) put up $680 million in cash for a risky gamble. It bought up Prosensa and its failed Duchenne drug drisapersen, betting that it could leverage its experience on rare drugs and take it on to a near-term regulatory approval.
On Tuesday evening, the biotech conceded a near-complete failure. After seeing the door slammed in its face at the FDA, European regulators at the CHMP sent a clear message that the same fate awaited BioMarin’s application across the Atlantic. BioMarin is now withdrawing its application, ending its work on drisapersen and three follow-on therapies: BMN 044, BMN 045 and BMN 053, now in Phase II studies.
But BioMarin vows to make a comeback, eventually.
“Our plan now is to invest in research of next generation oligonucleotides with the goal of making a safe and effective treatment available for boys with this devastating disorder,” said Jean-Jacques Bienaimé, BioMarin's chairman and chief executive officer. BioMarin has also been fighting to protect its patents on using antisense oligonucleotides for exon 51 skipping, winning an initial ruling last fall against Sarepta ($SRPT).
Prosensa's drug failed to beat a placebo in significantly improving boys' ability to complete a 6-minute walk test--the classic measure of success in this field. Soon after, GlaxoSmithKline ($GSK) walked out of its collaboration and washed its hands of the drug. But left to its own devices, the small biotech completed fresh analysis of extension study data that backed a theory that providing the drug earlier while extending treatment could delay disease progression.
That argument, though, was slapped down by outside and inside experts at the FDA, who failed to see any evidence of efficacy. The same experience awaited PTC Therapeutics ($PTCT), which saw its rival application spurned by an agency that refused to even accept it for review. Only Sarepta, which was subjected to a blistering internal review at the FDA, is still awaiting a final decision in the U.S. on the Duchenne drug eteplirsen following two delays, though a panel voted to reject. And PTC is waiting to see if European regulators will keep its drug on the market after failing its pivotal study.
Bienaimé indicated to me back in April that the final fate of drisapersen rested entirely on the Europeans’ attitude. An approval in Europe could have pointed the way forward, but a rejection raised questions of just how practical it was to carry on.
The biotech has experienced several setbacks recently, including a secondary endpoint miss for pegvaliase in March that raised a cloud of doubt about its prospects.
- here's the release