Sanofi Gets CDSCO Panel Nod To Market Avalglucosidase Alfa Powder for Pompe Disease

This came after Sanofi presented a proposal to import and market Avalglucosidase alfa powder for concentrate for solution for infusion (10 mg/ml) indicated for the treatment of long-term enzyme replacement therapy for patients with Pompe disease, along with a request to waive Phase III and Phase IV clinical trials in the country.

Pompe disease (also known as acid-maltase disease and glycogen storage disease II) is a rare genetic disorder that causes progressive weakness to the heart and skeletal muscles. It is caused by mutations in a gene that makes an enzyme called acid alpha-glucosidase (GAA), which the body uses to break down glycogen, a stored form of sugar used for energy.

Enzyme replacement therapy can help improve muscle tone and reduce glycogen storage in individuals with Pompe disease.

Avalglucosidase alfa is a lysosomal glycogen-specific enzyme that is used to treat patients one year of age and older with late-onset Pompe disease, or lysosomal acid alpha-glucosidase (GAA) deficiency.

Enzyme replacement therapy using avalglucosidase alfa aims to restore the missing GAA enzyme. Avalglucosidase alfa is a hydrolytic lysosomal glycogen-specific recombinant human GAA enzyme that is conjugated with multiple synthetic bis-mannose-6-phosphate (bis-M6P)­ tetra-mannose glycans for enhanced targeting to skeletal muscles. The M6P of avalglucosidase alfa binds to cation-independent mannose-6-phosphate receptor (CI-MPR) on the cell surface with high affinity, which allows drug uptake into cells. Avalglucosidase alfa is internalized and transported into lysosomes to undergo proteolytic cleavage. It then exerts GAA enzymatic activity to cleave glycogen.

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