Two medicines used to treat Pompe disease, a rare inherited muscle disorder caused by the abnormal buildup of glycogen in the body's cells, are running short across the United States and Europe. Sanofi, which manufactures both drugs, has alerted patient groups and physicians that supplies of Myozyme and Nexviazyme are dwindling. The disruption emerged shortly after the Food and Drug Administration warned the company about manufacturing problems at its facility in Waterford, Ireland, where both medicines are produced.
The FDA warning and what followed
The FDA flagged manufacturing issues at the Waterford plant, and the supply shortfall followed. Sanofi has told patient groups and physicians in both the United States and Europe that supply is tightening. The company attributes the problem to two things happening at the same site: a bottleneck in the final phase of manufacturing, and quality control issues. Those two problems at the same location create a compounding effect. Slower final-stage throughput means less medicine moves through the system, and quality control concerns can halt movement further still.
Batch release: the step nothing can skip
Batch release is the final quality assurance and regulatory stage of drug manufacturing. Here is what that process involves: a specific manufactured quantity of medicine is formally reviewed, tested against safety and potency standards, certified, and legally cleared for distribution and patient use. Regulators require this step before any dose leaves the facility. The Waterford plant is responsible for batch release across Sanofi's Pompe disease medicines. When that step slows or stalls, finished product cannot ship. It does not matter how much was manufactured earlier in the process. Nothing moves until batch release is complete.
Two drugs, one plant, one supply chain
Myozyme is the older standard of care for Pompe disease. Nexviazyme is the newer treatment that has since come to market. Both are made at the Waterford plant in Ireland. That single point of origin means the quality control problems and manufacturing bottleneck there affect the entire range of what Sanofi currently makes available for this disease. Patient advocacy groups and physicians across the United States and Europe have received direct notification from the company that supplies of both medicines are running low.