Sanofi Faces Pompe Drug Shortage After FDA Warning
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- Sanofi is experiencing shortages of Myozyme and Nexviazyme, two medicines used to treat Pompe disease, and has alerted patient groups and physicians in both the U.S. and Europe that supplies are dwindling.
- The Sanofi shortage emerged shortly after the U.S. Food and Drug Administration warned the company over manufacturing issues at its Irish facility in Waterford, where the two drugs are made.
- The cause has been described as a bottleneck in the final manufacturing phase combined with quality control issues at Sanofi's Waterford plant.
- Jazz Pharmaceuticals agreed to buy Actio Biosciences in a deal valued at up to $1.32 billion, adding an experimental treatment for a rare inherited form of epilepsy with no approved therapies.
- The Jazz-Actio drug, called ABS-1230, targets KCNT1-related epilepsy, which causes frequent seizures and developmental problems in children and affects about 2,500 patients in the U.S.
- ABS-1230 reduced seizures in an early study of children and is being evaluated in a study intended to support a U.S. approval application.
Why it matters: Sanofi's Pompe shortage — emerging right after an FDA manufacturing warning at the same Irish plant — threatens treatment continuity for patients with a rare, progressive disease. The Jazz-Actio deal gives Jazz a candidate for KCNT1-related epilepsy, a condition with no approved therapies affecting roughly 2,500 U.S. patients.
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