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

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 US regulator has cleared Miplyffa (arimoclomol), in combination with the enzyme inhibitor miglustat ... drug originally developed by Actelion as Zavesca for other diseases that is now ...

to file treatment protocols, which would allow physicians to use their drugs ahead of approval. The situation could also boost sales of Allschwil-based Actelion's Zavesca (miglustat).

Miglustat, which is sold by Johnson & Johnson as Zavesca, has long been used as the primary, albeit off-label, treatment for NPC patients. Zevra’s first-comer advantage was quickly challenged by ...