STAT+: A shortage of two Sanofi medicines for Pompe disease rattles patients

Sanofi is facing a shortage of two medicines used to treat Pompe disease, a rare genetic disorder. The issue surfaced shortly after the Food and Drug Administration (FDA) issued a warning to the company concerning manufacturing problems at its Irish facility. Myozyme, an older standard of care for Pompe disease, and Nexviazyme, a newer treatment, are both affected.

The drugmaker has notified patient groups and physicians in the U.S. and Europe about the dwindling supplies. The shortage is believed to be due to a bottleneck in the final manufacturing phase at the Waterford plant, as well as quality control issues. The plant is responsible for batch release, the final quality assurance and regulatory process that certifies the medicine is safe and potent for distribution and patient use.