2022-06-29| Trials & Approvals

Sanofi’s New Pompe Disease Treatment Wins Approval in Europe

by Joy Lin
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Sanofi’s Nexviadyme has been approved by the European Commission as a treatment for Pompe Disease, a rare progressive disease that attacks the heart and skeletal muscles. The marketing greenlight is a boost to Sanofi’s ambitions of making Nexviadyme the new standard of care for Pompe community.

The enzyme replacement therapy, also known as avaglucosidase alfa, is the first approved medicine for Pompe disease in Europe since 2006, following the marketing authorization of Myozyme (alglucosidase alfa), also developed by Sanofi.


How Nexviadyme Treats Pompe Disease


Pompe disease is caused by a deficiency of alpha-glucosidase, an enzyme required to break down glycogen. Without the enzyme, glycogen accumulates to toxic levels, damaging muscle cells. The disease presents itself in two forms: a severe form that rapidly takes hold in infancy and a late-onset form that progressively damages the muscles over time. 

Nexviadyme is a recombinant form of alpha-glucosidase that binds the mannose-6-phosphate (M6P) receptor, part of the key pathway for glycogen breakdown. Its affinity for M6P is said to be higher than its predecessor Myozyme. By taking on the function of the deficient alpha-glucosidase, Nexviadyme aims to improve uptake and enhance glycogen clearance in target tissues. 

In the Comet study comparing Nexviadyme to Myozyme in late-onset Pompe disease at 49 weeks, Nexviadyme met the primary endpoint of improving respiratory function after patients treated with Nexviadyme showed a 2.9% improvement from baseline in forced vital capacity. The improvement was 2.4% greater than that of the Myozyme group.

On muscle function, patients treated with Nexviadyme walked 32.2m farther compared to baseline in a walking test, the trial’s secondary endpoint. This was 30m farther than the change observed with Myozyme. 

In another study evaluating Nexviadyme in infantile-onset Pompe disease, patients experienced improvements in motor functions and left ventricle mass z-score (LVMZ). 

Common side effects of Nexviadyme include headache, dizziness, tiredness, nausea, and vomiting. 

Related article: America’s Ongoing Battle For Lower Insulin Prices 


A Contested Standard of Care 


While approved for marketing in Europe, Nexviadyme has been rejected by the EU’s drug regulator as a new active substance (NAS), either due to its similarity in molecular structure, clinical efficacy or safety to Myozyme. Furthermore, Nexviadyme was also recommended to be removed from the Community Register of Orphan Medical products. 

 The rejection is a blow to Sanofi as NAS products could benefit from up to 10 years of market exclusivity, shielding it against generic competition. The French drug giant has challenged the ruling and requested the re-examination of the CHMP opinion on Nexviadyme’s NAS status. 

The Pompe disease treatment has been widely accepted in multiple markets around the world, such as the EU, the US, Japan, and Canada among others. Sales of Nexviadyme reached $31.5 million in Q1 2022. Sales of Myozyme decreased 3% to $247 million as Sanofi switched the treatment of Myozyme to Nexviadyme in the US. 

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