NOVEMBER 12, 2021: “The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) reaffirmed its opinion that avalglucosidase alfa does not qualify as a New Active Substance (NAS).
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.”
Sibold added: “Today’s opinion fails to appropriately recognize and reward innovation and undermines the intended spirit of the NAS designation.
This disincentivizes existing holders of marketing authorizations from pursuing further innovation in biological therapies with continued, high, unmet need, which ultimately has negative implications for patients and the future of scientific innovation in Europe.
We strongly advocate that the EMA revisit and redefine the methodology used to assess the NAS status of biological products within the European Union, especially in the context of rare diseases.”
The July CHMP positive opinion recommending marketing authorization for avalglucosidase alfa for the treatment of people with Pompe disease reflects the robust data from the avalglucosidase alfa development program and the clinically meaningful improvements observed in respiratory function and movement endurance measures in people with Pompe disease compared to alglucosidase alfa, the current standard of care in Europe.”
https://www.sanofi.com/en/media-room/press-releases/2021/2021-11-12-12-38-38-2333221
