The US Food and Drug Administration has approved Miplyffa (arimoclomol) for the treatment of Niemann Pick type C (NPC), the first approved therapy for this indication.
NPC is a rare lysosomal disorder characterized by variable clinical signs, depending on the age of onset. It is caused by changes in either the NPC1 or NPC2 gene. Signs and symptoms may include prolonged unexplained neonatal jaundice or cholestasis, isolated unexplained splenomegaly, progressive, often severe neurological symptoms such as:
- Cognitive decline
- Cerebellar ataxia
- Vertical supranuclear gaze palsy (VSPG)
- Dysarthria
- Dysphagia
- Dystonia
- Seizures
- Gelastic cataplexy
- Psychiatric disorders
Arimoclomol is approved for use in combination with the enzyme inhibitor miglustat for the treatment of neurological symptoms caused by NPC in adults and children 2 years of age and older.
A randomized, double-blind, placebo-controlled 12 month clinical trial tested the safety and efficacy of arimoclomol in NPC patients ages 2 to 19 years of age. Fifty patients were randomized 2:1 with arimoclomal or placebo orally three times daily. Additionally, 78% of patients also received miglustat as background treatment.
As measured by the rescored 4-domain NPC Clinical Severity Scale (R4DNPCCSS), arimoclomal, in combination with miglustat, resulted in slower disease progression when compared to placebo in combination with miglustat. The R4CNPCCSS measures disease progression through ambulation, speech, swallow, and fine motor skills.
Prescribing information for arimoclomal contains a warning for hypersensitivity reactions such as hive and angioedema. The most common side effects included upper respiratory infection, diarrhea, and decreased weight.
With this approval, Zevra Therapeutics also receives a Priority Review Voucher that can be redeemed to receive priority review for another product or sold to another company.
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To learn more about NPC and other rare lysosomal disorders, visit https://checkrare.com/diseases/lysosomal-storage-disorders/
