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The Subject Expert Committee (SEC), which advises the Central drug regulator on various matters of approval, has recommended grant of permission to Sanofi Healthcare India to import and market the drug olipudase alfa powder for use in paediatric and adult patients with waiver of local phase III and IV clinical trial in the country.
The firm presented the proposal to the regulator seeking approval for import and marketing of 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 with a request for waiver of phase III & phase IV clinical trial in the country.
The committee noted that the drug falls under the orphan drug category and proposed indication is a rare disease.
The SEC also noted that the drug has been granted ‘orphan drug status’ in United States of America (USA), European Union (EU), United Kingdom (UK), Australia, Japan, Brazil and Malaysia and approved in 36 countries including USA, EU, UK, Japan, Brazil and United Arab Emirates (UAE).
"After detailed deliberation, the committee recommended grant of permission to import and market the drug with waiver of local phase III & IV clinical trials in the country," said the SEC.
According to the Central Drugs Standard Control Organisation (CDSCO), it has provisions to relax the requirement of local phase IV clinical trial, where the new drug is indicated in life threatening or serious diseases or diseases of special relevance to Indian health scenario or for a condition which is unmet need in India such as XDR tuberculosis, hepatitis C, H1N1, dengue, malaria, HIV, or for the rare diseases for which drugs are not available or available at a high cost or if it is an orphan drug.
Sanofi sells olipudase alfa, branded as Xenpozyme, in various countries at present. It received approval from the US Food and Drug Administration (FDA) for the drug, for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and paediatric patients, in August, 2022. Xenpozyme is the first therapy indicated specifically for the treatment of ASMD, and is currently the only approved treatment for this disease, claimed the company during the time.
ASMD, historically known as Niemann-Pick disease types A, A/B, and B, is an extremely rare, progressive genetic disease with significant morbidity and mortality. ASMD represents a spectrum of disease, with two types that may represent opposite ends of a continuum referred to as ASMD type A and ASMD type B. ASMD type A/B is an intermediate form that includes varying degrees of central nervous system (CNS) involvement.
Xenpozyme is administered intravenously every two weeks, and its administration requires a dose escalation phase followed by a maintenance phase, it added.
The company said that it has received approval for olipudase alfa in Japan with a 'pioneer' designation in March, 2022, the first approval for the drug anywhere in the world.
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