Early Diagnosis of and R&D in Lysosomal Storage Disorders to Augment Growth of Lysosomal Alpha Glucosidase Market

Lysosomal Alpha Glucosidase Market


 Acid alpha-glucosidase is an enzyme that helps to break down glycogen in the lysosome. Lysosomal acid alpha-glucosidase deficiency may lead to Pompe disease, glycogen storage disease II, and acid maltase deficiency.

Market Dynamics

Early diagnosis of lysosomal storage disorders is expected to propel growth of the lysosomal alpha glucosidase market. For instance, in January 2021, in the U.S., The Kansas Department of Health and Environment, added two lysosomal storage disorders to the Kansas Newborn Screening blood spot panel: Pompe Disease and Mucopolysaccharidoses I (MPS I).

R&D in lysosomal storage disorders is expected to offer lucrative growth opportunities for players in the lysosomal alpha glucosidase market. For instance, in October 2020, 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 (acid a-glucosidase deficiency).

Similarly, in February 2021, a study presented by Sanofi Genzyme during the WORLDSymposium 2021 Virtual meeting, reported that investigational recombinant human lysosomal acid α-glucosidase enzyme replacement therapy avalglucosidase alfa generated substantial clinical improvements in patients with late-onset Pompe disease versus alglucosidase alfa. In February 2021, Spark Therapeutics, a member of the Roche Group, announced the dosing of the first participant in the Phase 1/2 RESOLUTESM trial of SPK-3006, an investigational liver-directed adeno-associated viral vector gene therapy for late-onset Pompe disease.

However, clinical trial failures are expected to hinder growth of the lysosomal alpha glucosidase market. For instance, in February 2021, Amicus Therapeutics’s Phase III PROPEL pivotal trial of cipaglucosidase alfa and miglustat for late-onset Pompe disease failed to achieve primary endpoint.

The lysosomal alpha glucosidase market is witnessing increasing adoption of enzyme replacement therapy for the treatment of lysosomal storage disorders. For instance, in 2020, 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.

Competitive Analysis

Major players operating in the lysosomal alpha glucosidase market include, Genzyme Corp., a unit of Sanofi, Pharming Group N.V., Valerion Therapeutic, M6P Therapeutics, Audentes Therapeutics, Inc. and Oxyrane.

Major players operating in the lysosomal alpha glucosidase market are focused on R&D to expand their product portfolio. For instance, in June 2020, Sanofi’s investigational enzyme replacement therapy avalglucosidase alfa, demonstrated clinically meaningful improvement in critical manifestations (respiratory impairment and decreased mobility) of late-onset Pompe disease. Similarly, in February 2021, M6P Therapeutics reported preclinical efficacy and safety results for Gaucher disease and mucolipidosis II (MLII) respectively.

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