Sanofi is experiencing supply shortages of Myozyme and Nexviazyme, two medicines used to treat Pompe disease, following manufacturing and quality-control problems at its facility in Waterford, Ireland, according to a report by STAT.
The pharmaceutical company has reportedly notified patient organizations and healthcare professionals in the United States and Europe that supplies of both treatments are declining.
It remains unclear when normal availability will resume. Sanofi has indicated that production could take several months to recover and stabilize sufficiently, STAT reporter Ed Silverman wrote.
Myozyme and Nexviazyme Supplies Under Pressure
Myozyme is an established treatment for Pompe disease, while Nexviazyme is a newer therapy developed for patients with the rare genetic disorder.
According to STAT, supply disruptions emerged after a bottleneck in the final stage of manufacturing and quality-control issues at Sanofi’s Waterford plant.
The extent of the shortage reportedly differs across countries and between the two medicines. Existing inventory, local demand and scheduled shipment dates are among the factors affecting availability.
“The impact varies by country and by product, depending on available inventory, local demand and the timing of upcoming shipments,” a Sanofi spokesperson told STAT.
“In some markets, delays are limited. In others, intermittent or recurring supply interruptions have reached patients or may do so as the situation evolves.”
What Caused the Sanofi Drug Shortages?
The reported shortages are connected to problems in the final manufacturing phase and the batch-release process at the Waterford facility.
Batch release is the final quality-assurance and regulatory step before a medicine can be distributed for patient use. During this process, each manufactured batch is reviewed and formally certified as meeting applicable safety, quality and potency requirements.
A delay or failure during batch release can prevent medicines from entering the supply chain even if earlier production stages have been completed.
Sanofi has reportedly described the current problem as involving both a bottleneck in this final phase and quality-control concerns at the site.
FDA Previously Cited Sanofi’s Waterford Facility
The supply problems emerged shortly after the U.S. Food and Drug Administration raised concerns about manufacturing practices at the Waterford plant.
According to STAT, the FDA cited the facility in June for violations involving the production of other medicines. The reported Myozyme and Nexviazyme shortages do not necessarily mean the two Pompe disease treatments were directly involved in every issue identified by the regulator.
The timing, however, has increased attention on manufacturing and quality operations at the Irish facility.
When Will Myozyme and Nexviazyme Supplies Recover?
Sanofi has not provided a firm date for the full restoration of normal supplies.
The company has indicated that it may take months for production to resume at a sufficient level and become stable. Availability may therefore continue to differ by market as remaining inventory is distributed and new batches complete the required release process.
Some countries may experience only short shipment delays, while others could face recurring or intermittent shortages that affect patients.
Healthcare providers and patients may need to consult local Sanofi representatives, pharmacies or national medicines regulators for the latest information on availability in their markets.
What Is Pompe Disease?
Pompe disease is a rare inherited condition caused by a deficiency of the enzyme acid alpha-glucosidase. Without sufficient levels of this enzyme, glycogen accumulates in the body’s cells, particularly in skeletal and heart muscles.
The disease can cause progressive muscle weakness, breathing difficulties and other serious complications. Its severity and age of onset vary among patients.
Myozyme and Nexviazyme are enzyme-replacement therapies designed to address the underlying enzyme deficiency. Because Pompe disease requires ongoing management, interruptions in access to treatment may be particularly concerning for patients, caregivers and physicians.
Sanofi Shortages Raise Concerns for Pompe Disease Patients
The duration and severity of the Myozyme and Nexviazyme shortages will depend on Sanofi’s ability to resolve the manufacturing bottleneck, complete quality-control requirements and stabilize production at the Waterford facility.
The company has acknowledged that interruptions have already affected some markets and could reach additional patients as the situation develops.
Until normal production and distribution resume, the effect is expected to vary by country, product availability and local demand.
