Another company had a setback in the quest to offer treatment to Duchenne muscular dystrophy patients. The FDA notified Santhera Pharmaceuticals ($SANN) that it would require the results from another Phase III trial, which is expected to take an additional three years.
This follows a negative panel for another DMD drug from Sarepta Therapeutics ($SRPT) in April that was followed by a blown-through May PDUFA date--and a request for more data that came in early June, which the company had said it would respond to “in the coming weeks.”
For its part, Santhera said the agency will require the results of a trial that had been slated to be confirmatory, rather than part of a regulatory submission. The FDA requested results from the SIDEROS trial in glucocorticoid-using patients. The company had hoped for a review of its Raxone (idebenone) to treat DMD patients specifically in patients who are not taking concomitant glucocorticoids.
"While we are disappointed that the FDA does not support our plan to file an NDA for Raxone under subpart H for patients not using concomitant glucocorticoids, we now have clarity that successful completion of the SIDEROS trial will provide the necessary data to support NDA filing for Raxone in all DMD patients irrespective of the glucocorticoid use status," said Santhera CEO Thomas Meier in a statement.
He added that enrollment "in the SIDEROS trial will start shortly and we are committed to working closely with the FDA, clinical experts and the DMD patient community to make Raxone available for all DMD patients in the U.S. as quickly as possible."
Last month, the European Medicines Agency (EMA) opted to accept the filing specifically for Raxone in DMD patients with respiratory function decline who are not taking concomitant glucocorticoids. The company expects a decision from the agency in the first quarter of 2017.
The Santhera setback could point to further difficulties for Sarepta’s eteplirsen, argued RBC Capital analyst Simos Simeonidis in a July 14 note.
“We believe today’s news makes a positive decision on eteplirsen even more unlikely. Here are the facts: FDA just notified a company with positive data from a randomized, placebo-controlled trial, with 5-6 times the number of more patients (n=64) than Sarepta’s trial that these data are not sufficient to even be reviewed for approval, and that they should come back in 3 years after they run another Phase III trial, primarily because they need to see confirmation of these data from a trial with a more appropriate endpoint,” he argued.
The small cap was down by more than one-third on the news to a market cap of about $350 million.
- here is the release
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