Sanofi Faces Shortage of Two Pompe Disease Drugs
Get the Health newsletter
Daily health & science — research, biotech, public health, the studies worth knowing. Free.
- Sanofi is experiencing shortages of two medicines used to treat Pompe disease, alerting patient groups and physicians in both the U.S. and Europe that supplies are dwindling
- Myozyme, an older standard of care, and Nexviazyme, a newer treatment, are both affected — leaving patients without a clear alternative source for either drug
- The shortage emerged shortly after the FDA warned Sanofi over manufacturing issues at the Irish facility where both drugs are made
- Sanofi's Waterford plant handles batch release — the final quality assurance and regulatory step where a manufactured amount of medicine is certified for distribution
- The dwindling supply has been attributed to a bottleneck in the final manufacturing phase combined with quality control issues at the Waterford facility
Why it matters: Pompe disease is a rare, progressive neuromuscular disorder, and patients now face dwindling supplies of their two available treatments. Because both Myozyme and Nexviazyme rely on the same Waterford batch-release facility, a single regulatory bottleneck can simultaneously threaten an entire disease's therapeutic toolkit, forcing physicians and patient advocacy groups into urgent contingency planning.
Ask SkimNews
