Sanofi's 'Nexviazyme' for Pompe disease in shortage

Administrative measures under review

Essential medicines that patients require, a regular monthly supply is needed

Has reported to the Ministry of Food and Drug Safety (MFDS) about the supply shortage…supportive measures will be implemented, including granting exemptions from foreign-labeled packaging

Product photo of Nexviazyme

Following concerns about a supply shortage of Sanofi-Aventis Korea’s orphan drug 'Nexviazyme (avalglucosidase alfa)', South Korean health authorities have initiated a review to support it. Given that this drug is an essential medicine with no alternative, health authorities aim to preemptively address clinical gaps for patients.

Sanofi-Aventis Korea officially announced the supply deficit of Nexviazyme to the Ministry of Food and Drug Safety (MFDS) on June 12.

The company attributed supply shortage to "global manufacturing capacity deficits relative to rising international demand, as well as by shipping and logistics delays stemming from geopolitical conflicts in transit regions through the Middle East."

Nexviazyme is an enzyme replacement therapy (ERT) indicated for patients diagnosed with Pompe disease, a rare hereditary metabolic disorder. Pompe disease is a condition caused by a deficiency of the enzyme required to break down glycogen, leading to abnormal intracellular accumulation of glycogen in muscle tissue. This condition triggers progressive systemic skeletal muscle atrophy, loss of ambulation, and respiratory insufficiency, which can lead to mortality in severe clinical cases. 

Notably, Nexviazyme is the latest medicine with enhanced cellular drug uptake compared to existing standard-of-care agents, thereby demonstrating clinically meaningful improvements in patients' respiratory parameters and ambulatory functions. In South Korea, it is categorized as an irreplaceable essential medication with no therapeutic alternatives available in the market.  

Sanofi reported concerns through its shortage notification, stating that "Regular monthly supply and strict adherence to the dosing regimen are ncessary for this product. Any supply disruption could expose patients to a rapid, quantitative decline in their six-minute walk test (6MWT) scores, a prognostic indicator," and added, "Enzyme deficiencies in active myocytes trigger irreversible skeletal muscle damage and atrophy, which directly correlate with deterioration in overall clinical status and the loss of autonomy in activities of daily living."

Currently, Sanofi Korea has activated an emergency import protocol. Although the company expedited an import of 1,200 vials on June 12, this volume remains insufficient to satisfy regional demand, leaving the drug vulnerable to an early out-of-stock (OOS) scenario. Consequently, while Sanofi Korea plans to import an additional 1,700 vials by July 20, it is concurrently exploring regulatory channels to directly introduce foreign-labeled packaging (global packs) to bridge the temporary inventory gap.

The MFDS has begun assessing the current situation and implement administrative support measures to protect patient access. An MFDS official stated, "While the Middle Eastern war may not seem a direct contributing factor, avoiding a therapeutic gap remains the MFDS's top priority," and added, "Based on feedback from the manufacturer, the MFDS is actively considering administrative fast-tracking, including granting exemptions from localized Korean labeling requirements for imported product lots originally destined for other countries to facilitate expedited customs clearance.

Nexviazyme has been covered under the National Health Insurance system since September 1, 2023. It requires an intravenous infusion of 20 mg per kilogram of body weight every two weeks; a patient weighing 60 kg requires a 1,200 mg dose. With the insurance  price set at KRW 1,436,600 per 100-mg vial, bi-weekly treatment costs reach KRW 17.16 million. However, because eligible individuals qualify for the rare disease copayment exemption program, patients are responsible for only 10% of the total treatment cost out of pocket.