Nearly two years after their narrow PhIII failure, Orphazyme gets speedy look at the FDA
Orphazyme’s bet that, despite missing both primary endpoints on its pivotal study, they would find welcoming ears at the FDA has paid off, for now.
The Danish biotech announced today that the FDA has accepted the application for their experimental drug arimoclomol in the rare neurodegenerative condition Niemann-Pick disease Type C and given it priority review. Although drugs that fail pivotal studies rarely receive a priority look at the agency, the distinction should come with little surprise. Orphazyme has signaled repeatedly since the 2019 results came out that the FDA viewed an application favorably and, the FDA gave it breakthrough designation that November.
The agency set a PDUFA date for March 17, 2021. Orphazyme’s stock jumped 8.38% on the news, from 83.5 krones to 90.5 krones ($13.3 to $14.3).
“We look forward to collaborating with the FDA as they complete their review of this NDA to address the unmet medical need in NPC, and meanwhile are working to expand our U.S. activities in preparation for potential commercial availability next year,” CEO Kim Stratton said in a statement.
Orphazyme, a one-drug company targeting a string of neurodegenerative disorders, came out it with results from a 50-person Phase II/III study on Niemann-Pick type C patients, their lead indication, in January 2019. Arimoclomol failed to hit both primary endpoints, both a standard scale for assessing symptoms in patients, called the NPC Clinical Severity Scale, and the Clinical Global Impression of Improvement, a self-rating tool that the FDA requested. It also failed several secondary endpoints.
Still, the drug only narrowly missed significance on the first primary endpoint, where they saw both a 74% reduction in disease severity and a p-value of 0.0506. And the company noted that two pre-specified subgroup analyses reached significance, with disease progression slowing in patients over the age of 4 and in patients who had been taking an Actelion drug sometimes prescribed for the disease. All endpoints were against placebo.
In a degenerative condition with no significant treatments, Orphazyme was willing to bet that was enough to take to regulators. So far, they’ve been proven right.
Orphazyme, eyeing funds for commercialization, filed for its first US offering earlier this month, penciling in an initial figure of $115 million for the raise. In addition to their lead indication, they also have pivotal studies ongoing for ALS and sporadic inclusion body myositis, a progressive muscle disorder.