FDA grants priority review for avalglucosidase alfa, a potential new therapy for Pompe disease
November 18 2020 - 1:00AM
FDA grants priority review for
avalglucosidase alfa, a potential new therapy for Pompe
disease
- The FDA decision date for avalglucosidase alfa, an
investigational enzyme replacement therapy, is set for May 18,
2021
- Regulatory submission based on positive data from two trials in
patients with late-onset and infantile-onset Pompe disease,
respectively
- Avalglucosidase alfa received FDA Breakthrough Therapy and Fast
Track designations for the treatment of people with Pompe
Disease
- Pompe disease, a rare degenerative muscle disorder, affects
approximately 3,500 people in the U.S.
- Milestone reinforces 20+year commitment to Pompe disease
community
PARIS – November 18, 2020 - The
U.S. Food and Drug Administration (FDA) has accepted for priority
review the Biologics License Application (BLA) for avalglucosidase
alfa for long-term enzyme replacement therapy for the treatment of
patients with Pompe disease (acid α-glucosidase deficiency). The
target action date for the FDA decision is May 18, 2021.
Avalglucosidase alfa is an investigational
enzyme replacement therapy designed to improve the delivery of acid
alpha-glucosidase (GAA) enzyme to muscle cells, and if approved,
would offer a potential new standard of care for patients with
Pompe disease.
In October, the European Medicines Agency
accepted for review the Marketing Authorization Application for
avalglucosidase alfa for long-term enzyme replacement therapy for
the treatment of patients with Pompe disease. The Medicines and
Healthcare Products Regulatory Agency in the UK has granted
Promising Innovative Medicine designation for avalglucosidase
alfa.
“The hallmarks of Pompe disease are the
relentless and debilitating deterioration of the muscles, which
causes decreased respiratory function and mobility,” said Karin
Knobe, Head of Development for Rare Diseases and Rare Blood
Disorders at Sanofi. “Avalglucosidase alfa is specifically designed
to deliver more GAA enzyme into the lysosomes of the muscle cells.
We have been greatly encouraged by positive clinical trial
results in patients with late-onset and infantile-onset Pompe
disease.” Pompe disease is a rare, degenerative muscle disorder
that can impact an individual’s ability to move and breathe. It
affects an estimated 3,500 people in the U.S. and can manifest at
any age from infancy to late adulthood.i The BLA is based on
positive data from two trials:
- Pivotal Phase 3, double-blind, global comparator-controlled
trial (COMET), which evaluated the safety and efficacy of
avalglucosidase alfa compared to alglucosidase alfa (standard of
care) in patients with late-onset Pompe disease. Results from this
trial were presented during a Sanofi-hosted virtual scientific
session in June 2020 and in October 2020 at World Muscle Society
and the American Association of Neuromuscular and Electrodiagnostic
Medicine.
- The Phase 2 (mini-COMET) trial evaluated the safety and
exploratory efficacy of avalglucosidase alfa in patients with
infantile-onset Pompe disease previously treated with alglucosidase
alfa. Results from this trial were presented at the WORLDSymposium,
in February 2020.
Delivery of GAA to Clear
Glycogen
Pompe disease is caused by a genetic deficiency
or dysfunction of the lysosomal enzyme 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 respiratory muscles and the diaphragm
muscle supporting lung function, and other skeletal muscles that
affect mobility.
To reduce the glycogen accumulation caused by
Pompe disease, the GAA enzyme must be delivered into the lysosomes
within muscle cells. Research led by Sanofi has focused on ways to
enhance the delivery of GAA into the lysosomes of muscle cells by
targeting the mannose-6-phosphate (M6P) receptor that plays a key
role in the transport of GAA.
Avalglucosidase alfa is designed with
approximately 15-fold increase in M6P content, compared to standard
of care alglucosidase alfa, and aims to help improve cellular
enzyme uptake and enhance glycogen clearance in target tissues.ii
The clinical relevance of this difference has not been
confirmed.
Avalglucosidase alfa is currently under clinical
investigation and its safety and efficacy have not been evaluated
by any regulatory authority worldwide.
About Sanofi 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. Sanofi, Empowering Life |
Media Relations Contact Sally Bain
Tel.: +1 781 264 1091sally.bain@sanofi.com Investor
Relations Contacts Paris Eva Schaefer-Jansen Arnaud
DelepineYvonne Naughton Investor Relations Contacts
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i https://rarediseases.org/rare-diseases/pompe-disease/
ii Zhou Q. Bioconjug Chem. 2011 Apr 20;22(4):741-51
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