After being ushered onto the inside lane at FDA, regulators hand Amryt a CRL for rare disease drug
Amryt may have earned a priority review for its drug to treat rare cases of epidermolysis bullosa (EB), but they got handed a CRL for their trouble in any case.
The biotech reported Monday morning that the agency is demanding “additional confirmatory evidence of effectiveness for Oleogel-S10 in EB.” Now the biotech says they will follow up with the FDA to determine exactly what that means.
It wouldn’t be too surprising, though, if the agency is holding out for a new late-stage trial to prove the therapy works as billed, treating patients with a disorder that makes their skin as fragile as butterfly wings. The therapy hit on the primary endpoint — with 41% of the patients in the drug arm seeing wound closure in 45 days, compared to 28% in the control arm — but missed on a number of key secondary endpoints.
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