The week in pharma: action, reaction and insight – week to September 27

By Barbara Obstoj-Cardwell. Editor

Significant regulatory news last week included the US Food and Drug Administration (FDA) granting approval for Zevra Therapeutics’ Miplyffa, making it the first treatment for Niemann-Pick disease type C, although this was quickly followed by an FDA nod for Aqneursa, developed by privately-held rare disease specialist IntraBio, for the same condition. Additionally, Bristol Myers Squibb last week announced that the FDA has approved its schizophrenia treatment Cobenfy, previously called KarXT. On the research front, US drug developer IDEAYA Biosciences released new Phase II data on its investigational uveal melanoma drug darovasertib. And US biotech Wave Life Sciences announced positive Phase II data on its Duchenne muscular dystrophy candidate WVE-N531. Also of note, due to safety concerns, US pharma giant Pfizer announced it is withdrawing its sickle cell disease drug Oxbryta from the market.

Zevra’s arimoclomol approved for Niemann-Pick disease