Based on the incidence of NPC estimated at one in 100,000 to 130,000 live births, Zevra estimates approximately 1,800 individuals with NPC in the U.S. and Europe combined—approximately 900 in ...

Miglustat (Opfolda, Yargesa, Zavesca) has been approved in the European Union, Canada, and Japan and is considered a standard of care for treating progressive neurological complications in NPC ...

Cantor analysts expect Zevra’s drug to pull around $75 million in 2025 sales. Zevra currently has enough cash and expected future sales of Miplyffa and its urea cycle disorder med Olpruva to ...

Zevra has sold its US Food and Drug Administration (FDA) priority review voucher (PRV) for $150m to an undisclosed party. The US-based company received the PRV when its Niemann-Pick disease type C ...

Miplyffa was the first therapy approved for Niemann-Pick disease type C in the US. It is indicated for use alongside Johnson & Johnson’s (J&J’s) Zavesca (miglustat). Zavesca has long been used as the ...

The oral medication miglustat (Zavesca) may help slow the progression of neurological symptoms associated with NPC. In the fall of 2024, the FDA approved the oral medication arimoclomol ...

Miglustat(Zavesca) generic is an enzyme inhibitor, prescribed for Gaucher disease. It prevents the body from producing the fatty substances. More info about Miglustat ...

Until now there were no FDA-approved drugs for the disease, although the FDA-approved Gaucher’s disease drug miglustat (Zavesca), a glucosylceramide synthase inhibitor, is often used off-label ...

Of the 50 patients enrolled in the trial, 39 also received miglustat as a background treatment during the study. Miglustat, brand name Zavesca, is a Johnson & Johnson drug approved in the U.S. for ...

In the trial, Miplyffa plus miglustat halted disease progression through 12 months of treatment as shown by a 0.2 point decrease from baseline on the rescored 4-domain NPC Clinical Severity Scale ...

Friday, the FDA approved Zevra Therapeutics, Inc.'s (NASDAQ:ZVRA) Miplyffa (arimoclomol), an oral medication for Neimann-Pick disease, type C (NPC). Miplyffa, in combination with Johnson & Johnson ...

Friday, the FDA approved Zevra Therapeutics, Inc.'s (NASDAQ:ZVRA) Miplyffa (arimoclomol), an oral medication for Neimann-Pick disease, type C (NPC). Miplyffa, in combination with Johnson & Johnson's ...