Sanofi to present Phase 3 results of avalglucosidase alfa in patients with late-onset Pompe disease
08 Juin 2020 - 7:00AM
Sanofi to present Phase 3 results of avalglucosidase alfa in
patients with late-onset Pompe disease
Sanofi to present Phase 3 results of avalglucosidase
alfa in patients with late-onset Pompe disease
- Virtual scientific session June 16, 2020, 8:00-9:00am
ET/2:00-3:00pm CET
- Avalglucosidase alfa receives FDA Breakthrough Therapy
designation
PARIS – June
8, 2020 – Sanofi will host a virtual
scientific session to present data from the Phase 3 COMET trial of
investigational enzyme replacement therapy (ERT) avalglucosidase
alfa in patients with late-onset Pompe disease (LOPD).
The session, open to healthcare professionals
and members of the media, will include a data presentation by Jordi
Diaz-Manera, M.D., Ph.D., Professor of Neuromuscular Disorders,
Translational Medicine and Genetics at the John Walton Muscular
Dystrophy Research Center, Newcastle University, UK, and Professor
of Neuromuscular Diseases, Translational Medicine and Genetics in
the Neuromuscular Diseases Unit, Neurology department of Hospital
de la Santa Creu, Barcelona, Spain.
The presentation will be followed by a Q&A
session moderated by Alaa Hamed, M.D., MPH, MBA, Global Head of
Medical Affairs, Rare Diseases at Sanofi.
The scientific session, endorsed by the COMET
trial author group, is being scheduled as a result of the
postponement of the July 2020 International Congress on
Neuromuscular Diseases (ICNMD) due to the COVID-19 pandemic. Data
from the Phase 3 COMET trial would have been presented at the July
2020 ICNMD. Pre-registration is required for the June 16, 2020
scientific session. Please click here to
register.
The U.S. Food and Drug Administration (FDA) has
granted Breakthrough Therapy designation status to avalglucosidase
alfa for the treatment of patients with a confirmed diagnosis of
Pompe disease. Breakthrough Therapy designation serves to expedite
the development and review of drugs that target serious or
life-threatening conditions. Drugs qualifying for this designation
must show preliminary clinical evidence of a substantial
improvement on a clinically significant endpoint over available
therapies, or over placebo if there is no available therapy.
About Pompe disease
Pompe disease is caused by a genetic deficiency
or dysfunction of the lysosomal enzyme acid alpha-glucosidase
(GAA), resulting in build-up of glycogen in muscles, including the
proximal muscles and the diaphragm, and eventually causing
progressive and irreversible muscle damage. This rare disease
affects an estimated 50,000 people worldwide and can manifest at
any age from infancy to late adulthood.
Pompe disease is often classified as late-onset
Pompe disease (LOPD) or infantile-onset Pompe disease (IOPD).
Patients with LOPD typically present any time after the first year
of life to late adulthood. The hallmark symptoms of LOPD are
impaired respiratory function and skeletal muscle weakness, which
often leads to impaired mobility. Patients often require
wheelchairs to assist with mobility and may require mechanical
ventilation to help with breathing.i Respiratory failure is the
most common cause of death in patients with Pompe disease.ii Pompe
disease is classified as IOPD when symptoms begin prior to one year
of age. In addition to skeletal muscle weakness, heart function is
also commonly impacted.
About Avalglucosidase alfa
The goal of ERT for Pompe disease is to deliver
enzyme into the lysosomes within muscle cells to replace the
missing or deficient GAA that is needed to prevent build-up of
glycogen in the muscles. Avalglucosidase alfa is an investigational
ERT for Pompe disease designed to improve the delivery of enzyme to
the cells in the muscles, most notably into skeletal muscle. With
approximately 15 moles of mannose-6-phosphate (M6P) per mole of
GAA, avalglucosidase alfa aims to help improve cellular enzyme
uptake and enhance glycogen clearance in target tissues.iii
The FDA granted Fast Track designation to
avalglucosidase alfa for the treatment of patients with Pompe
disease. Avalglucosidase alfa has not been approved by the U.S. FDA
or any other regulatory agency worldwide for the uses under
investigation.
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 |
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Media Relations Contact Sally Bain Tel.: +1
781-264-1097 Sally.Bain@sanofi.com |
Investor
Relations Contact Felix Lauscher Tel.: +33 (0)1 53 77 45
45 ir@sanofi.com |
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suffering. We stand by the few who
suffer
i Hagemans ML, et al. Brain 2015;128:671-677.
ii Winkel LP et al. J Neurol. 2005;252(8):875–84.
iii Zhou Q. Bioconjug Chem. 2011 Apr 20;22(4):741-51
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