Europe OKs Nexviadyme for Infantile- and Late-onset Disease

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The European Commission has approved the next-generation enzyme replacement therapy Nexviadyme (avalglucosidase alfa) to treat both late-onset and infantile-onset Pompe disease.

This is the first time a new treatment for Pompe has been approved in Europe since 2006, according to Nexviadyme’s developer Sanofi Genzyme.

“For more than two decades, we’ve been working with the community and leveraging our scientific expertise to improve care for people living with Pompe disease. We strongly believe in the meaningful clinical benefits of this medicine as a new standard of care,” Bill Sibold, executive vice president of specialty care at Sanofi, said in a press release.

Nexviadyme is already approved to treat forms of Pompe disease in the U.S., Japan, Canada, Switzerland, Australia, Brazil, Taiwan and the United Arab Emirates. The treatment is marketed under the brand name Nexviazyme outside of Europe.

Pompe disease is caused by mutations that impair the production of an enzyme needed to break down a complex sugar molecule called glycogen. An enzyme replacement therapy (ERT), Nexviadyme is designed to deliver a version of the deficient enzyme to the body’s cells.

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The next-generation therapy was designed to improve enzyme delivery to muscles and boost glycogen clearance compared to the first ERT approved for Pompe, Sanofi’s Myozyme (alglucosidase alfa), marketed as Lumizyme in the U.S.

Approval of Nexviadyme was supported by data from an ongoing clinical trial called COMET (NCT02782741), which is testing Nexviadyme against Lumizyme in 100 children and adults with late-onset Pompe disease who have not been treated before. A recent analysis covering nearly two years of data from the trial showed that Nexviadyme treatment improved walking ability and lung function.

A separate trial, Mini-COMET (NCT03019406), is testing Nexviadyme in 22 children with infantile-onset disease. Two-year data showed the therapy to be well-tolerated, and suggested that it improved or stabilized motor function.

The most frequent side effects associated with Nexviadyme in clinical trials were itching (9.4%), rash (8%), headache (7.2%), welts (6.5%), fatigue (6.5%), nausea (5.8%), and chills (5.1%).

“The approval of Nexviadyme in Europe to treat Pompe disease is backed by a robust body of evidence showing clinically meaningful improvements that can impact quality of life. The totality and rigor of the data is particularly noteworthy given the complexities of research and development for such a rare and progressive condition,” said Benedikt Schoser, MD, a professor at the Ludwig-Maximilians-University Munich.

“Nexviadyme’s demonstrated clinical benefit and molecular innovation bring a new treatment option to people living with Pompe disease who continue to face unmet needs,” Schoser added.

Sanofi has expressed disappointment with European regulatory authorities that Nexviadyme does not qualify as a new active substance (NAS), basically meaning it is not meaningfully different from available therapies. Authorities also have recommended removing Nexviadyme from the register of orphan medicinal products (OMP).

According to Sanofi, these decisions may undermine structures in place to promote the development of treatments for rare diseases in Europe.

“[Sanofi] will work hard to ensure the broadest possible access in Europe despite the European Commission’s failure to recognize Nexviadyme’s NAS and OMP designations. We call on patient advocacy groups, policymakers, clinicians and patients to join us in our efforts to ensure innovative treatments are appropriately recognized and made available to patients in Europe and beyond,” Sibold said.