Zevra Therapeutics’ Miplyffa™ (Arimoclomol) Receives u.s. Fda Approval as Treatment for Niemann-Pick Disease Type C

The first NPC drug approved by the FDA, MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older. [...] “The approval of MIPLYFFA is a monumental milestone for NPC patients and their family members in the U.S. [...] With this labeled indication, patients will now have more access to treatments to tackle this devastating disease.” The approval of MIPLYFFA for the treatment of NPC is based on the totality of the data in the New Drug Application (NDA), which included additional evidence supporting trial endpoints, FDA-preferred analyses, and additional confirmatory evidence, both clinical and nonclinical. [...] Zevra will immediately initiate its launch activities for MIPLYFFA, which is expected to be commercially available in the U. S. [...] in eight to 12 weeks. Launch of AmplifyAssist™ —Comprehensive Support for Patients Zevra is committed to assisting those whose lives are affected by NPC to overcome the barriers and challenges that may impact their treatment journey. [...] The mission of the program is to support the individual needs of eligible patients and those who care for them. [...] MIPLYFFA was granted Breakthrough Therapy designation, Rare Pediatric Disease designation, Orphan Drug designation, and Fast Track designation by the FDA for the treatment of NPC. [...] INDICATIONS AND USAGE MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. [...] These increases occurred mostly in the first month of MIPLYFFA treatment and were not associated with changes in glomerular function. [...] Participation in these programs is subject to the laws and regulations of each jurisdiction under which each respective program is operated.