Joslyn Crowe, executive director of the National Niemann-Pick Disease Foundation (NNPDF), describes the Niemann-Pick community’s reaction to the FDA denying the approval of arimoclomol for the treatment of Niemann-Pick Type C (NPC).

NPC is a disabling neurogenetic disorder that has been diagnosed prenatally, neonatally, during childhood, and even into adulthood. This very rare genetic disorder is marked by progressive motor dysfunction and a highly variable symptom profile and onset of symptoms. It can result in the patient’s death soon after birth or manifest as a chronic disorder with symptoms worsening slowly over time.

Following the news that the FDA sent a complete response letter (CRL) regarding arimoclomol, Ms. Crowe notes that the community was devastated. The degenerative nature of NPC, she says, does not allow patients to wait a long time for a drug approval. Thus, according to Ms. Crowe, the denial of arimoclomol is likely to lead to many unnecessary patient deaths. 

Though she does not know the FDA’s rationale, it is suspected that the FDA denied the approval based on “swallowing” being a domain in the 5-domain NPCCSS in the study data presented to the FDA. The study, which is ongoing, has a primary endpoint of change in the NPC disease severity based on the NPCCSS scores. Therefore, “swallowing” is directly connected to the primary endpoint which, Ms. Crowe, believes the FDA has a problem with. This is troublesome because a number of clinical trials in NPC, as well as other rare neurodegenerative disorders, use swallowing as part of their primary endpoint. This CRL thus may set a precedent that makes it near impossible for other NPC drugs to be approved. 

To stay up to date with the latest news in rare disease, sign up for our weekly newsletter here.