Sanofi Gets CDSCO panel nod to market Olipudase alfa for rare disease
New Delhi: Granting the local Phase III and phase IV clinical trial waiver in the country, the Subject Expert Committee (SEC) functional under the Central Drug Standard Control Organisation (CDSCO) has given its nod to the drug major Sanofi for importing and marketing Olipudase alfa powder for concentrate for solution for infusion 20 mg vial indicated as enzyme replacement therapy for long-term treatment of noncentral nervous system (CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in paediatric and adult patients.
This came after the firm presented the proposal to import and market Olipudasealfa powder for concentrate for solution for infusion 20 mg vial indicated as enzyme replacement therapy for long-term treatment of non-central nervous system (CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in paediatric and adult patients with a request for waiver of Phase III & Phase IV clinical trial in the country.
Olipudase alfa is a recombinant human acid sphingomyelinase. It is the first and only enzyme replacement therapy in the world for the treatment of Acid Sphingomyelinase Deficiency (ASMD), also known as Niemann- Pick disease.
ASMD is a rare lysosomal storage disease caused by mutations in the SMPD1 gene, leading to a deficiency in acid sphingomyelinase and the abnormal accumulation of the primary ASM substrate, sphingomyelin. Olipudase alfa works to hydrolyze sphingomyelin accumulated in body tissues, such as the lungs, liver, spleen, kidneys, and bone marrow.
Olipudase alfa is used to treat Acid Sphingomyelinase Deficiency (ASMD) in children and adults. It is an enzyme replacement therapy that works to ameliorate the signs and symptoms of ASMD by reducing the amount of sphingomyelin that accumulates in organs and causes tissue damage in patients with ASMD. Acid sphingomyelinase typically breaks down metabolically-related lipids such as sphingomyelin in various cell types, such as monocytes, macrophages, and hepatocytes. The deficiency of acid sphingomyelinase thus leads to the accumulation of these lipids in body tissues, causing progressive cell and tissue damage and impairing organ functioning. Thus, it works to reduce the extent of non-neurological manifestations of ASMD, such as splenomegaly and hepatomegaly.
At the recent SEC meeting for Endocrinology & Metabolism held on the 19th and 20th of January 2023, the expert panel reviewed the proposal presented by the drug major Sanofi for importing and marketing the Olipudasealfa powder for concentrate for solution for infusion 20mg vial along with a request for waiver of Phase III & Phase IV clinical trial in the country.
The committee noted that the drug fall under the orphan drug category and the proposed indication is a rare disease.
The committee also noted that the drug has been granted ‘orphan drug status’ in the US, EU, UK, Australia, Japan, Brazil & Malaysia and approved in 36 countries including USA, EU, UK, Japan, Brazil and UAE.
After detailed deliberation, the committee recommended the grant of permission to import and market the drug with a waiver of local Phase III & IV clinical trials in the country.
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