Shionogi’s IntraBio acquisition brings Aqneursa, a drug approved for treating two rare neurological disorders and the potential to expand to additional indications, both rare and common. It’s Shionogi’s second big rare disease move this year, following the acquisition of global rights to an amyotrophic lateral sclerosis drug from Tanabe Pharma.
Zevra Therapeutics received a priority review voucher for Miplyffa, the first FDA-approved treatment for the rare metabolic disorder Niemann Pick disease type C. Though Zevra has other drug candidates that could benefit from the voucher, the biotech is selling it to an undisclosed buyer.
In an interview, Kyan Health Co-Founder and Chief Commercial Officer Konstantin Struck discussed how Kyan gives mid-market and enterprise employers access to premium workforce mental healthcare, at a price point that is affordable.
Zevra Therapeutics drug Miplyffa is now FDA approved for Niemann-Pick disease type C, a lysosomal storage disorder. The affirmative regulatory decision took into account additional clinical data generated since the FDA turned down a submission for the drug in 2021.