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SEC recommends waiver of local phase III and IV trials for Sanofi's orphan drug to treat Pompe disease

Gireesh Babu, New Delhi
Monday, January 30, 2023, 08:00 Hrs  [IST]

The Subject Expert Committee (SEC) that advises the drug regulator of the country has recommended waiver of local phase III and IV clinical trials in India for Sanofi Healthcare India's orphan drug to treat the Pompe disease. The Committee recommended grant of permission to import and market the drug without these stages of studies.

The SEC for endocrinology and metabolism, which met in the CDSCO office in New Delhi on January 19 and 20, considered the proposal of Sanofi Healthcare India to import and market avalglucosidase alfa powder for concentrate for solution for infusion (10 mg/ml). The drug is indicated for the treatment of long term enzyme replacement therapy for the treatment of patients with Pompe disease. The company requested for a waiver of phase II and phase IV clinical trials in the country, in its proposal.

The Committee noted that the drug falls under the orphan drug category and
proposed indication is a rare disease. It also noted that the drug has been granted orphan drug status in US, Australia, Switzerland, Japan and Malaysia and approved in 13 countries including the United States of America, European Union, United Kingdom, Japan, Canada, Switzerland and Australia.

"After detailed deliberation, the committee recommended for grant of permission to import and market the drug with waiver of local phase III & IV clinical trials in the country," said the SEC after the meeting.

Sanofi, which has received approval from the US Food and Drug Administration (FDA) for its avalglucosidase alfa-ngpt, branded as Nexviazyme in US, in August, 2021 and marketing authorisation from European Commission for avalglucosidase alfa for the long-term treatment of both late-onset and infantile-onset Pompe disease, a rare, progressive and debilitating muscle disorder. The brand name of the drug in Europe is Nexviadyme.

"Nexviadyme is the first and only newly approved medicine for Pompe disease in Europe since 2006, when the European Commission authorized the marketing of alglucosidase alfa, branded Myozyme," said Sanofi while announcing the approval from European Commission.

Nexviadyme is approved in multiple markets around the world for the treatment of certain people living with Pompe disease, including the European Union, the United States, Japan, Canada, Switzerland, Australia, Brazil, Taiwan and the United Arab Emirates, it added in June, 2022. Outside of Europe, the treatment is marketed under the brand name Nexviazyme. In the US and Japan, the majority of the Myozyme (alglucosidase alfa)-treated population has started, or is in the process of starting, treatment with Nexviazyme (avalglucosidase alfa).

Pompe disease is caused by a genetic deficiency or dysfunction of the lysosomal enzyme acid alpha-glucosidase (GAA), which results in build-up of complex sugars (glycogen) in muscle cells throughout the body. The accumulation of glycogen leads to irreversible damage to the muscles, including the diaphragm that supports respiratory function and skeletal muscles that affect mobility, functional endurance and breathing, added the company.


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