Miplyffa: Zevra announces price for Niemann-Pick Disease-Type C drug
- 23-Sep-2024
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 […]
Miplyffa: US Approves First Treatment for Niemann-Pick Disease-Type C
- 20-Sep-2024
The US Food and Drug Administration (FDA) approved Miplyffa (arimoclomol), an oral medication for the treatment of Neimann-Pick disease, type C (NPC). Miplyffa, in combination with the enzyme inhibitor miglustat, is approved to treat neurological symptoms associated with NPC in adults and children 2 years of age and older. Miplyffa is the first drug approved […]