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 ...

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

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 ...

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 ...

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 Announces Approval of Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) in Japan Provided by GlobeNewswire Jun 25, 2025, 11:00:00 AM ...

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 ...