The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) moment reaffirmed its opinion that avalglucosidase alfa doesn’t qualify as a New Active Substance (NAS). Sanofi doesn’t agree with the CHMP’s conclusion on NAS status and is assessing implicit 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 complaint, a progressive and enervating muscle complaint that impairs a person’s capability to move and breathe and a negative opinion on NAS designation. As a result, Sanofi requested a reappraisal of the CHMP opinion in relation to the NAS conclusion.
“ We’re 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 isn’t applicable for natural curatives and doesn’t regard for the innovative structural changes to enzyme relief curatives, similar as avalglucosidase alfa. Also, it fails to consider the challenges of conducting clinical exploration in rare conditions and doesn’t take into account the summation of the data, the nature of the complaint, or eventually the patient experience.”
Sibold added “ Moment’s opinion fails to meetly fete and award invention and undermines the willed spirit of the NAS designation. This disincentivizes being holders of marketing authorizations from pursuing farther invention in natural curatives with continued, high, unmet need, which eventually has negative counteraccusations for cases and the future of scientific invention in Europe. We explosively endorse that the EMA readdress and review the methodology used to assess the NAS status of natural products within the European Union, especially in the environment of rare conditions.”
The July CHMP positive opinion recommending marketing authorization for avalglucosidase alfa for the treatment of people with Pompe complaint reflects the robust data from the avalglucosidase alfa development program and the clinically meaningful advancements observed in respiratory function and movement abidance measures in people with Pompe complaint compared to alglucosidase alfa, the current standard of care in Europe.
New Active Substance (NAS) status is a designation considered by the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) as part of the review of a European Union (EU) Marketing Authorization Operation (MAA). This is an important designation for a product, validating it as a discerned, new, recommended treatment for cases. While kindly specialized in nature, NAS designation supports ongoing invention in the development of implicit, new treatments for cases by furnishing nonsupervisory data protections.
About Pompe Disease
Pompe complaint is caused by a inheritable insufficiency or dysfunction of the lysosomal enzyme acid nascence-glucosidase (GAA), which results in figure-up of complex sugars (glycogen) in muscle cells throughout the body. The accumulation of glycogen leads to unrecoverable damage to the muscles, including the diaphragm muscle that supports lung function and cadaverous muscles that affect mobility, as well as cardiac muscles in immature- onset Pompe complaint.
Pompe complaint can present as immature- onset Pompe complaint (IOPD), the most severe form of Pompe complaint with rapid-fire onset in immaturity, and late- onset Pompe complaint (LOPD), which precipitously damages muscles over time. LOPD symptoms may present at any age. Still, due to the wide diapason of clinical donations and progressive nature of the complaint, it can take seven to nine times before cases admit an accurate opinion. As the complaint progresses, people with LOPD may bear mechanical ventilation to help with breathing or a wheelchair to help with mobility.
About avalglucosidase alfa
Avalglucosidase alfa is an enzyme relief remedy designed to target the mannose-6-phosphate (M6P) receptor. With roughly15-fold increase in M6P content compared to alglucosidase alfa, the current standard of care, avalglucosidase alfa is designed to help ameliorate cellular enzyme uptake and thereby ameliorate concurrence of glycogen figure-up in target apkins, the underpinning complaint medium in Pompe complaint.
On August 6, 2021, theU.S Food and Drug Administration (FDA) approved the enzyme relief remedy under the brand name Nexviazyme ® (avalglucosidase alfa-ngpt) for the treatment of late- onset Pompe complaint in cases one time of age and aged. In addition, on September 27, 2021, the Japan Medicinals and Medical Bias Agency (PMDA) approved avalglucosidase alfa for the treatment of late- onset Pompe complaint and immature- onset Pompe complaint. As part of Sanofi’s commitment to the Pompe complaint community and to insure broad access, Sanofi has decided to price avalglucosidase alfa at equality with alglucosidase alfa, the only presently available remedy for the treatment of Pompe complaint and the comparator arm in the vital study.
Avalglucosidase alfa has entered special designations from nonsupervisory agencies across requests, generally reserved for drugs that are anticipated to represent significant advancements in safety or efficacity and help address unmet requirements, emphasizing its eventuality anticipated value. In theU.S., the drug was granted a Priority Review, as well as Advance Remedy and Fast Track designations. In South Korea, Taiwan, Australia, Switzerland, and Japan, the drug was granted Orphan Designation. In the UK, the Medicines and Healthcare Products Regulatory Agency preliminarily granted Promising Innovative Drug (PIM) designation for avalglucosidase alfa. Avalglucosidase alfa was also included in early access programs in the UK via the Early Access to Medicines Scheme (EAMS) and in France via the Temporary Authorization for Use (ATU) program, with cohort ATU designation.
Sanofi is devoted to supporting people through their health challenges. We’re a global biopharmaceutical company concentrated on mortal health. We help illness with vaccines, give innovative treatments to fight pain and ease suffering. We stand by the many who suffer from rare conditions and the millions with long- term habitual conditions.