It’s been just six months since Zevra Therapeutics scored an FD | Niemann-Pick disease type C therapy Miplyffa became ...

Zevra Therapeutics Inc (ZVRA) marks a successful transition to a commercial stage with MyPIFA's launch, despite financial ...

Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, ...

ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, ...

The most common adverse reactions in Trial 1 (≥15%) in MIPLYFFA-treated patients who also received miglustat were upper respiratory tract infection, diarrhea, and decreased weight. Three (6% ...

Zevra has sold its US Food and Drug Administration (FDA) priority review voucher (PRV) for $150m to an undisclosed party.

Rare disease-focused biopharma Zevra Therapeutics has agreed to sell its Rare Pediatric Disease Priority Review Voucher (PRV) to an undisclosed buyer for $150 million.

The PRV was granted to Zevra in September 2024 following approval by the U.S. Food and Drug Administration of MIPLYFFA (arimoclomol), which is indicated for use in combination with miglustat for ...

Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced that it has entered ...

Miplyffa is taken orally three times a day in conjunction with the enzyme inhibitor miglustat. Miglustat, which is sold by Johnson & Johnson as Zavesca, has long been used as the primary ...