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Zevra Therapeutics’ drug, Miplyffa, for treating a rare and fatal genetic disorder will have a wholesale acquisition cost ranging between $40,000 and $106,000 per month depending on dosage, the company said on a conference call on Monday.
At the highest dose the annual price comes to about $1.3 million, while H.C. Wainwright analysts estimate the average net pricing of up to $600,000 per year.
The average monthly price of the drug would come to $85,000 based on dosing seen in its expanded access program, the company added.
The oral drug is to be given three times a day, with the exact dosage ranging from 47 milligram to 124 milligram depending on an individual’s body weight.
The U.S. health regulator on Friday approved the drug making it the first treatment to get a nod for Neimann-Pick disease type C (NPC) – a rare genetic condition that affects the nervous system and other organs.
Miplyffa, in combination with miglustat, was cleared to treat neurological symptoms associated with NPC in adults and children 2 years of age and older.
Miplyffa: US Approves Treatment for Niemann-Pick Disease-Type C
Zevra expects Miplyffa to be commercially available within eight to 12 weeks.
Niemann-Pick disease affects the body’s ability to break down and transport fats such as cholesterol and lipids, inside cells – which leads to the abnormal buildup of fats.
The company estimates that 900 people are living with NPC in the U.S., of which about one-third have been diagnosed with the rare, fatal neurodegenerative disease.
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