High Prevalence of Fabry Disease and Approval and Launch of New Products to Augment Growth of Enzyme Replacement Therapy Market

 

Enzyme replacement therapy is an alternative medical treatment that substitutes the missing enzyme in the human body. Typically, this is performed by administering an intravenous (IV) injection of a commercially available solution containing the necessary enzyme to replace one or more low-producing enzymes. Enzymes are critical for the normal function of many bodily systems. In the human body, they are responsible for the regulation of acid-alkaline balance, protein synthesis, metabolism, and other processes. These functions are regulated by numerous enzymes including those in the digestive tract, immune and the reproductive systems, the endocrine system and the neurotransmission system.

Market Dynamics

High prevalence of Fabry disease is expected to propel growth of the enzyme replacement therapy market. For instance, according to a report updated by National Center for Biotechnology Information, in January 2020, the prevalence of Fabry disease in white, male populations was 1:17,000 to 1:117,000. Moreover, approval and launch of new products is also expected to aid in growth of the 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).

Efficacy of enzyme replacement therapy in treatment of various diseases is expected to offer lucrative growth opportunities for players in the global enzyme replacement therapy market. 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.

Among regions, North America is expected to witness significant growth in the global enzyme replacement therapy market, owing to approval and launch of new products. For instance, in August 2020, the U.S. FDA accepted for review Protalix BioTherapeutics' marketing application seeking approval of enzyme replacement therapy pegunigalsidase alfa for the treatment of adults with Fabry disease.

Major players operating in the global enzyme replacement therapy market include, Shire plc, Amicus Therapeutics, Genzyme Corporation, Pfizer Inc., Protalix BioTherapeutics, AzurRx BioPharma, Inc., BioMarin Pharmaceutical, Inc., Sanofi, Sigma-Tau Pharmaceuticals, Inc., Essential Pharmaceuticals Limited, Merck KGa, and AbbVie Inc.

Major players operating in the global enzyme replacement therapy market are focused on R&D to expand their product portfolio. For instance, in January 2021, AzurRx BioPharma, Inc. announced the first two patients have been dosed in the Phase 2b OPTION 2 extension study of MS1819 using immediate release capsules for the treatment of exocrine pancreatic insufficiency in patients with cystic fibrosis.