Sanofi Speciality Care has reaffirmed its commitment to rare diseases in India on the occasion of Rare Disease Day- 28th February, stating that it will continue with its efforts in empowering the lives of patients with rare diseases by offering transformative healthcare options.

Anil Raina, General Manager, Sanofi Speciality Care (India) said, “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)."

With the largest portfolio of rare disease therapies, the company has also 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.

“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 Raina.

Both these products have been granted orphan drug status and approval in several countries including the US, Australia, EU, UK and Japan, to name a few.

Dr Shalini Menon, Country Medical Lead Sanofi, “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.