Sanofi Faces Pompe Drug Shortage After FDA Warning; Jazz Buys Actio — SkimNews
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- Sanofi is experiencing shortages of Myozyme and Nexviazyme, two medicines used to treat Pompe disease, after the FDA warned the company over manufacturing problems at its Waterford, Ireland facility
- Sanofi alerted patient groups and physicians in both the U.S. and Europe that supplies are dwindling due to a bottleneck in the final manufacturing phase and quality control issues at the Waterford plant
- Jazz Pharmaceuticals agreed to buy Actio Biosciences in a deal valued at up to $1.32 billion, adding an experimental treatment for KCNT1-related epilepsy, a severe inherited form of epilepsy with no approved therapies
- ABS-1230, the drug Jazz is acquiring, is being developed for KCNT1-related epilepsy, which affects about 2,500 patients in the U.S. and causes frequent seizures and developmental problems in children
- ABS-1230 reduced seizures in an early study of children and is now being evaluated in a study intended to support a U.S. approval application
Why it matters: Two rare-disease stories with opposite trajectories: Sanofi's supply disruption hits patients with no easy substitute for a serious genetic disorder, while Jazz's $1.32B bet signals that small rare-disease pipelines are commanding premium prices even in early-stage development. For Pompe patients, the shortage lands right after an FDA warning, suggesting the manufacturing fix isn't imminent; for Jazz, acquiring a drug that showed seizure reduction in a tiny early study sets up a high-stakes regulatory path.
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