Arimoclomol treatment.

On May 15, 2022, KemPharm, Inc., a specialty pharmaceutical company focused on rare central nervous system diseases like NPC, announced its acquisition from Orphazyme of Arimoclomol. This action supports the confidence of NPC clinicians in the benefit of Arimoclomol. “We believe the efficacy signal for Arimoclomol in NPC is convincing and that there is a viable regulatory path that could enable a successful NDA resubmission” said Travis Mickle, Ph.D., KemPharm’s President and CEO.  Patients will continue receiving Arimoclomol under an expanded access program until a determination on approval is made.