Sanofi announces results of CHMP re-examination of the New Active Substance status for avalglucosidase alfa.

Sanofi does not agree with the CHMP’s conclusion on NAS status and is evaluating potential options for avalglucosidase alfa in the European Union.

On July 23, 2021, the CHMP issued a positive opinion recommending marketing authorization for avalglucosidase alfa for the treatment of people with Pompe disease, a progressive and debilitating muscle disorder that impairs a person’s ability to move and breathe and a negative opinion on NAS designation. As a result, Sanofi requested a re-examination of the CHMP opinion in relation to the NAS conclusion.

“We are extremely disappointed with the CHMP’s opinion to deny designating avalglucosidase alfa as a new active substance,” said Bill Sibold, Executive Vice President of Sanofi Genzyme. “We believe the CHMP NAS process applies a narrow interpretation of the NAS principles which is not appropriate for biological therapies and does not account for the innovative structural changes to enzyme replacement therapies, such as avalglucosidase alfa. Moreover, it fails to consider the challenges of conducting clinical research in rare diseases and does not take into account the totality of the data, the nature of the disease, or ultimately the patient experience.”