Sanofi expects to bring two of its rare disease therapies to India later this year, or early next year.

The company has received a recommendation from the Subject Expert Committee to import and market two new products in India -- Nexviazyme (Avalglucosidase alfa powder) and Xenpozyme (Olipudase alfa powder) for Pompe disease and Niemann-Pick disease (ASMD) respectively, a note from the company said. “We are hoping to launch these therapies in India by end of 2023 or beginning of next year, subject to completion of all further regulatory steps,” said Anil Raina, General Manager, Sanofi Speciality Care (India).   

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Both these products have been granted orphan drug status and approval in several countries, including the US, Australia, the EU, UK and Japan, according to a note from the company. In fact, the company had received waivers on late stage clinical trials, Phase III and IV, a person familiar with the development said. The products would be imported, but details on its pricing are not yet available. 

Dr Shalini Menon, Sanofi Country Medical Lead, said: “Xenpozyme is the first therapy indicated specifically for the treatment of ASMD, and is currently the only approved treatment for this disease. This is a devastating and extremely rare disease that affects both children and adults. The approval of Xenpozyme represents the culmination of bold work done in research and development, and Sanofi’s unwavering commitment to this historically overlooked community.” She said Nexviazyme is for both infantile and late-onset Pompe disease, and showed improvements in respiratory function and walking distance measures.” 

“Over the past 40 years, Sanofi has launched different therapies for the treatment of many rare diseases, namely Gaucher disease, Fabry disease, Pompe disease, Mucopolysaccharidosis I (MPS I) and Acid Sphingomyelinase deficiency (ASMD),” Raina said.  

Since its incorporation in India (2007), he added, the company has been working towards enhancing awareness on rare diseases, providing free diagnostic support, and building capacity of the clinicians, among other things. Outlining other global humanitarian programmes and charitable access programmes that the company runs, he said, “More than 1000 patients in over 70 countries are in the company’s global Humanitarian Program today, out of which over 170 patients are from India.”