Sanofi's Nexviazyme® Receives FDA Breakthrough Therapy Designation for Pompe Disease

Recently, China-Sanofi announced that its next-generation enzyme replacement therapy (ERT) drug, Nexviazyme® (Avalglucosidase Alfa for Injection), has been approved by the National Medical Products Administration (NMPA) of China for the long-term treatment of patients with Pompe disease. This is the second ERT drug from Sanofi approved in China for the treatment of Pompe disease, following Myozyme® (Alglucosidase Alfa for Injection), aiming to become the new standard treatment for Pompe disease.

Nexviazyme® is a new generation enzyme replacement therapy (ERT) drug for the treatment of Pompe disease, which has been granted the "Breakthrough Therapy" designation by the FDA. It can enhance cellular uptake of the enzyme, better improve patients' respiratory and motor functions, and has a good safety profile, aiming to become the new standard treatment for Pompe disease.

Compared with the first-generation enzyme replacement therapy drugs, the mannose-6-phosphate (M6P) level of Myozyme® is increased by approximately 15 times. A higher M6P level can enhance the uptake of the drug by muscle cells, thereby breaking down excess glycogen and reducing damage to the patient's muscle cells.

Pompe disease is an ultra-rare, autosomal recessive genetic disorder with very few patients. It is divided into Infantile-Onset Pompe Disease (IOPD) and Late-Onset Pompe Disease (LOPD). IOPD progresses rapidly, and without effective treatment, patients often die of heart failure and respiratory failure around the age of one. If LOPD is not treated in time, it leads to irreversible progressive damage to multiple organs and systems, resulting in long-term dependence on wheelchairs and ventilators, as well as premature death. In May 2018, the "First List of Rare Diseases" issued jointly by the National Health Commission and five other departments included Pompe disease.

ERT is currently the only proven effective specific treatment for Pompe disease, and is recommended as a long-term treatment option by multiple authoritative global guidelines and consensus statements. As a next-generation ERT drug for Pompe disease, Nexviazyme® has 15 times the level of mannose-6-phosphate (M6P) compared to first-generation enzyme replacement therapy drugs. Higher levels of M6P can improve enzyme delivery to muscle cells, increase cellular uptake of the enzyme, thereby promoting the breakdown of excess glycogen and reducing damage to patients' muscle cells.

The COMET study of Nylezan® is a randomized, double-blind, multinational, multicenter Phase III study, involving 100 patients across 55 research centers (20 countries). Results from the 97-week extension study showed that, compared to baseline:

• Patients who received Nulazid® treatment during both the initial analysis period (49 weeks) and the extended study period; the percentage of predicted Forced Vital Capacity (FVC) increased by 2.65 (1.05) percentage points.

• Patients who switched from first-generation enzyme replacement therapy to Nereda® treatment during the extended study period showed an increase of 0.36 (1.12) percentage points in the predicted FVC percentage.

• Patients who received Nulazid® treatment during both the initial analysis period (49 weeks) and the extended study period showed an increase of 18.60 (12.01) meters in the 6-minute walk test (6MWT) distance.

• Patients who switched from first-generation enzyme replacement therapy to Nerezan treatment during the extended study period experienced an increase of 4.56 (1244) meters in 6MWT distance.

• During the initial analysis period (49 weeks), Nulazid® demonstrated good safety, and no new safety events were observed during the extended study period (97 weeks).

Currently, Naylzan® has been approved in many countries and regions around the world, including the United States, the European Union, Japan, Canada, Australia, and Brazil, and has been granted the title of "Breakthrough Therapy" by the FDA.

Editor: Mu Mian

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