Sanofi’s neuromuscular disorder drug now TGA registered

Pharma News: Sanofi’s enzyme replacement therapy, which improves respiratory function and walking distance in patients with a debilitating and potentially fatal neuromuscular disorder, has been registered in Australia.

Nexviazyme (avalglucosidase alfa) is included on the Australia Register of Therapeutic Goods for long term enzyme replacement therapy for the treatment of patients one year and older with Pompe disease (acid α-glucosidase deficiency), a progressive and debilitating muscle disorder that impairs a person’s ability to move and breathe.

Neurologist Associate Professor Robert Henderson from the Wesley Hospital, and Royal Brisbane and Women’s Hospital, said that while enzyme replacement therapy has been on Australia’s Life Saving Drugs Program since 2015 for patients with Pompe disease, more treatment options are needed.

“Until now, there has only been one available treatment for Pompe disease and people often experience a decline in muscle function over time. Nexviazyme offers meaningful improvements in respiratory function and walking distance compared to the existing treatment and it appears to be a new standard of care for Pompe disease,” he said.

Nexviazyme is an enzyme replacement therapy (ERT) designed to target the mannose-6-phosphate (M6P) receptor to improve cellular enzyme uptake and enhance glycogen clearance in target tissues. In the pivotal Phase 3 trial (COMET), Nexviazyme demonstrated clinically meaningful improvements in respiratory function and walking distance in late-onset Pompe disease patients, as well as improvements in key disease burden measures in infantile-onset Pompe disease patients.

Australia and New Zealand Head of Medical, Dr Kasia Siwek, said “This registration reflects our longstanding commitment to harness cutting edge science to address the unmet medical needs of the Pompe Community. We are committed to working with the Federal Government to ensure that Nexviazyme is available as soon as possible for Australians aged one or more with Pompe disease.”

Nexviazyme was considered by the Pharmaceutical Benefits Advisory Committee in November.