Once again the FDA has taken a hard line on an effort to seek a quick approval for a new drug that treats Duchenne muscular dystrophy. And while it’s not Sarepta this time, at least one prominent analyst says the agency’s tough stance highlights the odds of a looming rejection.
It was Santhera’s turn today to disappoint investors. The Swiss biotech announced this morning that the agency will require investigators to finish another, confirmatory Phase III study for their Duchenne’s drug, putting off any prospect of a U.S. approval by more than three years.
Santhera (SIX: SANN) had hoped that the FDA would let them file now, working with late-stage data on Raxone, or idebenone. The drug is designed to improve lung function in patients by energizing weakened muscle cells, guarding mitochondria. European regulators at the CHMP are reviewing the drug now, but the FDA isn’t going to budge.
Santhera’s stock plunged 35%, and RBC’s Simos Simeonidis, who’s adopted a skeptical view of Sarepta’s chances at the FDA, sees this as another case that would indicate the agency won’t be lenient with Sarepta, which was exposed to a harsh takedown by FDA insiders unimpressed with their data on a tiny group of patients.
“The bull thesis on SRPT has been that the FDA would be “lenient” and “forgiving” with weaker datasets in DMD, because of the devastating nature of the disease and the lack of available drugs for this patient community,” notes Simeonidis. “However, time after time, we see evidence that this is NOT the case: 1) BMRN: rejection, 2) PTCT: RTF, 3) SRPT: Negative Vote in the panel, 4) SANN: don’t file until you run a confirmatory Phase III trial.”
Sarepta’s volatile shares were down about 5% in midday trading.
The bull enthusiasts, though, will note that the FDA has already made several exceptions for Sarepta, including its latest decision to delay ruling on its marketing application for an accelerated approval until investigators get a chance to search for fresh evidence of dystrophin production among patients treated with eteplirsen. And they may yet decide that Sarepta will be the exception that proves the rule.
In an opposing opinion, Baird’s Skorney says the Santhera setback is meaningless when it comes to figuring out where Sarepta sits with regulators. His statement:
“We think the negative SRPT market reaction to FDA’s request that Santhera run a second Phase 3 study before submitting a NDA for their DMD drug is wrong. We believe this is more likely an issue specific with Santhera’s drug/trial rather than a broader regulatory signal as to what the hurdle for DMD approval is. If this was indeed the regulatory hurdle, eteplirsen never would have made it this far.”
“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,” commented Thomas Meier, PhD, CEO of Santhera. “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.”
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