Sanofi experiencing shortage of two Pompe disease drugs

Ed’s stories explore prescription drug pricing, affordability and access, as well issues surrounding patents, litigation, and legislation. He is also the author of the morning Pharmalittle newsletter and the afternoon Pharmalot newsletter.

Sanofi is experiencing shortages of two medicines used to treat Pompe disease, and the problem emerged shortly after the Food and Drug Administration warned the company over manufacturing issues at the Irish facility where the drugs are made.

In recent days, the drugmaker has alerted patient groups and physicians in both the U.S. and Europe that supplies of Myozyme, an older standard of care, and Nexviazyme, a newer treatment, are dwindling due to what has been described as a bottleneck in the final manufacturing phase, as well as quality control issues at its Waterford plant.

The site is responsible for what is known as batch release, which refers to the final quality assurance and regulatory process where a manufactured amount of medicine is formally certified, approved, and cleared for distribution and use by patients. This step is supposed to verify the batch of medicine meets safety and potency requirements.