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 ...

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 ...

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

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 ...

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 ...

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 ...

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 ...

Using the original 5-Domain NPCCSS score, there was a treatment difference of -1.40 in favor of arimoclomol (95% CI -2.76 to -0.03, P=0.0456), corresponding to a 65% reduction in annual disease ...

Amicus Therapeutics has released a post-hoc analysis from the PROPEL study, published in Muscle and Nerve, which evaluated the efficacy of cipaglucosidase alfa-atga combined with miglustat (cipa ...

Miplyffa, in combination with Johnson & Johnson's JNJ enzyme inhibitor Zavesca (miglustat), is approved to treat neurological symptoms associated with NPC in adults and children two years of age ...

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 ...

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 ...