Sanofi racks up 2 approvals in Europe for enzyme replacement therapies for rare diseases

When CEO Paul Hudson took over at Sanofi in 2019, he launched a “play to win” strategy centered on first-in-class and best-in-class drugs in therapeutic areas where the company was lacking.

On Tuesday morning, Sanofi revealed two approvals from the European Commission—both for enzyme replacement treatments for rare diseases—that speak to Hudson’s mission.

One is for Xenpoxyme, the first and only enzyme replacement therapy to treat non-central nervous system symptoms of acid sphingomyelinase deficiency (ASMD). The other is for Nexviadyme, the first new treatment option for Pompe disease approved in Europe in more than 15 years.

The Xenpoxyme approval is for the A/B and B types of the genetic condition which causes a buildup of fats which can disrupt organ function and be fatal. The A/B form of ASMD affects organs, including the brain, and can be fatal in the first few years of life. The B type of ASMD also effects organs, primarily the lungs but not the brain, leading to a life expectancy of just 17 years.

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Xenpoxyme, which is infused every two weeks, replaces the defective acid sphingomyelinase enzyme, reducing the fatty build up. Two trials, upon which the approval was based, showed that Xenpoxyme improved lung function and reduced liver and spleen volumes.

There are roughly 2,000 people in Europe, Japan and the United States with the A/B and B types of the disease, according to Sanofi. Xenpoxyme is approved in Japan and is due for a decision in the U.S. in October.

“The ASMD community has waited many years for a treatment for this rare and debilitating genetic disease,” John Reed, M.D., Ph.D., and Sanofi’s global R&D chief said in a release. “The approval represents a transformational shift in what we can offer to patients.”

The approval for Nexviadyme is for late-onset and infantile-onset Pompe disease. It was approved by the FDA in August of last year for the late-onset form of the disorder and is known commercially in the U.S. as Nexviazyme.

The drug is an improvement on Sanofi’s blockbuster Myozyme, helping facilitate better lung function.