FDA Approves Miplyffa for Ultra Rare Niemann-Pick Disease Type C

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The FDA has approved Miplyffa (arimoclomol) to treat Niemann-Pick Disease Type C (NPC), an extremely rare and progressive neurodegenerative disease, according to the details of a recent news release. Drug manufacturer Zevra Therapeutics Inc. also announced the launch of AmplifyAssist, a patient support hotline that offers insurance coverage education, disease information and prescription refill assistance. Patients can visit the AmplifyAssist website or call 1-888-668-4198.

NPC is a lysosomal storage disorder. When someone has NPC, their body is unable to transport fatty lipids inside their cells, which leads to a buildup substances such as cholesterol in body tissues, including brain tissue, according to the National Organization for Rare Disorders. This accumulation leads to fatal tissue damage. Only about 300 patients in the United States have been diagnosed with NPC, but it’s estimated there are at least another 600 who remain undiagnosed.

Miplyffa is approved for patients ages two and older and is to be used in combination with miglustat, which addresses the neurological manifestations of the disease, such as double vision and ataxia. About 80% of NPC patients are currently on or have been on miglustat, according to the investor call.

Miplyffa is given as a capsule, orally three times a day with or without food. Exact dosage is 47 mg, 62 mg, 93 mg or 124 mg and determined by body weight. It is expected to be priced at $9.50 per milligram, the investor call details revealed.

The Miplyffa approval is based on new drug application data, which includes evidence from a yearlong trial to determine safety and effectiveness. Effectiveness was measured using the 4-domain NPC Clinical Severity Scale (R4DNPCCSS). The combination of Miplyffa and miglustat resulted in a decrease of 0.2 points from baseline, compared with 1.9 progression points for patients taking only miglustat.

“Until now, those living with NPC have had no FDA-approved treatment to combat this devastating disease,” Laurie Turner, Family Services Manager, National Niemann-Pick Disease Foundation (NNPDF) said in the news release. “For more than 30 years, NNPDF and the community have been working to find treatments for NPC.”

The most common adverse reactions of Miplyffa were upper respiratory infection, diarrhea and weight loss seen in at least 15% of patients.

Miplyffa will be available in the United States within the next eight to 12 weeks.