The U.S. Food and Drug Administration (FDA) has denied the approval of arimoclomol, a heat shock protein amplifier intended for the treatment of Niemann-Pick disease 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.
The FDA issued the Complete Response Letter (CRL) stating that the drug will not be approved based on the data presented in the application. Details of the CRL are not known but the developers of arimoclomol, Orphazyme, noted that the decision to not approve the drug application included the need for additional qualitative and quantitative evidence to further support the validity of the 5-domain NPC Clinical Severity Scale (NPCCSS) used in the phase 2/3 trial. A primary endpoint of the phase 2/3 clinical trial was progression in disease severity as measured by the 5-domain NPCCSS. This is a disease-specific measure of disease progression consisting of the five clinically most relevant domains to patients with NPC, caregivers and physicians.
Additionally, the FDA noted in the CRL that data beyond the single phase 2/3 clinical trial are needed to support the benefit-risk assessment of the NDA.
“We are disheartened by the outcome of the FDA’s review, given the urgent need for a new therapeutic option for NPC, but we remain committed to working with the regulators, with the goal of delivering arimoclomol to families managing this challenging disease,” said Orphanzyme CEO, Christophe Bourdon.
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