Sanofi announces results of CHMP re-examination of the New Active Substance status for avalglucosidase alfa, a potential new standard of care for the treatment of Pompe disease
NOVEMBER 12, 2021
The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) today 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.
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 Application (MAA). This is an important designation for a product, validating it as a differentiated, new, recommended treatment for patients. While somewhat technical in nature, NAS designation supports ongoing innovation in the development of potential, new treatments for patients by providing regulatory data protections.
When the CHMP concludes that (the active substance of) a product should be considered a NAS, it means that the CHMP views the product as being meaningfully different from another already-approved product at the level of molecular structure and/or with respect to clinical efficacy or safety properties.
About Pompe Disease
Pompe disease is caused by a genetic deficiency or dysfunction of the lysosomal enzyme acid alpha-glucosidase (GAA), which results in build-up of complex sugars (glycogen) in muscle cells throughout the body. The accumulation of glycogen leads to irreversible damage to the muscles, including the diaphragm muscle that supports lung function and skeletal muscles that affect mobility, as well as cardiac muscles in infantile-onset Pompe disease.
Pompe disease can present as infantile-onset Pompe disease (IOPD), the most severe form of Pompe disease with rapid onset in infancy, and late-onset Pompe disease (LOPD), which progressively damages muscles over time. LOPD symptoms may present at any age. However, due to the wide spectrum of clinical presentations and progressive nature of the disease, it can take seven to nine years before patients receive an accurate diagnosis. As the disease progresses, people with LOPD may require mechanical ventilation to help with breathing or a wheelchair to assist with mobility.
About avalglucosidase alfa
Avalglucosidase alfa is an enzyme replacement therapy designed to target the mannose-6-phosphate (M6P) receptor. With approximately 15-fold increase in M6P content compared to alglucosidase alfa, the current standard of care, avalglucosidase alfa is designed to help improve cellular enzyme uptake and thereby improve clearance of glycogen build-up in target tissues, the underlying disease mechanism in Pompe disease.
On August 6, 2021, the U.S Food and Drug Administration (FDA) approved the enzyme replacement therapy under the brand name Nexviazyme® (avalglucosidase alfa-ngpt) for the treatment of late-onset Pompe disease in patients one year of age and older. In addition, on September 27, 2021, the Japan Pharmaceuticals and Medical Devices Agency (PMDA) approved avalglucosidase alfa for the treatment of late-onset Pompe disease and infantile-onset Pompe disease. As part of Sanofi’s commitment to the Pompe disease community and to ensure broad access, Sanofi has decided to price avalglucosidase alfa at parity with alglucosidase alfa, the only currently available therapy for the treatment of Pompe disease and the comparator arm in the pivotal study.
Avalglucosidase alfa has received special designations from regulatory agencies across markets, typically reserved for medicines that are expected to represent significant improvements in safety or efficacy and help address unmet needs, underscoring its potential anticipated value. In the U.S., the medicine was granted a Priority Review, as well as Breakthrough Therapy and Fast Track designations. In South Korea, Taiwan, Australia, Switzerland, and Japan, the medicine was granted Orphan Designation. In the UK, the Medicines and Healthcare Products Regulatory Agency previously granted Promising Innovative Medicine (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 dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.
With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.
Media Relations Contact
Tel: +1 (781) 264-1091
Investor Relations Contacts Paris
Investor Relations Contacts North America
Tel.: +33 (0)1 53 77 45 45
Sanofi Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the fact that product candidates if approved may not be commercially successful, the future approval and commercial success of therapeutic alternatives, Sanofi’s ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation, trends in exchange rates and prevailing interest rates, volatile economic and market conditions, cost containment initiatives and subsequent changes thereto, and the impact that COVID-19 will have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. Any material effect of COVID-19 on any of the foregoing could also adversely impact us. This situation is changing rapidly and additional impacts may arise of which we are not currently aware and may exacerbate other previously identified risks. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2020. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.