Sanofi warns of dwindling supplies of two Pompe disease drugs
Sanofi has notified patients and doctors in the United States and Europe that its Pompe disease treatments Myozyme and Nexviazyme are in short supply due to manufacturing bottlenecks and quality issues at its Irish plant.
Sanofi disclosed that its two medicines for Pompe disease, Myozyme and Nexviazyme, are facing supply constraints. The announcement came after the Food and Drug Administration issued a notice concerning manufacturing deficiencies at the company's Waterford, Ireland site. The facility is responsible for the batch-release process, which confirms that each production lot meets safety and potency standards before it can be shipped.
Problems in this final stage, combined with quality-control failures, have created a bottleneck that is shrinking the amount of drug available. Sanofi has reached out to patient advocacy groups and healthcare providers in both the United States and Europe to warn of the shortage. The reduced stock threatens treatment continuity for individuals relying on these therapies.
Why it matters
Patients with Pompe disease may lose access to essential treatments because of the manufacturing problems.
In this story