Sanofi Speciality Care has reiterated its commitment to addressing rare diseases in India, marking Rare Disease Day by announcing continued efforts to empower patients with transformative healthcare options. The global movement for rare diseases seeks to achieve equity in social opportunity, healthcare, and access to diagnosis and treatments for those living with rare conditions. Sanofi has supported Rare Disease Day in India for 13 years, starting with the first event in 2010 at the Sir Gangaram Hospital in Delhi.

Anil Raina, General Manager of Sanofi Speciality Care (India), noted that the company has launched therapies for various rare diseases over the past 40 years, including Gaucher disease, Fabry disease, Pompe disease, Mucopolysaccharidosis I (MPS I), and Acid Sphingomyelinase deficiency (ASMD). Since the company’s incorporation in India in 2007, Sanofi has been working to raise awareness of rare diseases, provide free diagnostic support, build clinicians’ capacity, and support Centers of Excellence (CoEs) in public and private institutions.

Raina also highlighted Sanofi’s global humanitarian program, which provides free life-saving treatment to Indian patients diagnosed with Lysosomal Storage Disorders (LSDs). The India Charitable Access Program (INCAP) has supported patients with Gaucher disease, Pompe disease, Fabry disease, and MPS I disease with life-saving Enzyme Replacement Therapy, with over 170 patients from India currently enrolled in the program.

With the largest portfolio of rare disease therapies, Sanofi has received a recommendation from the Subject Expert Committee to import and market two new products in India for Pompe disease and Niemann-Pick disease (ASMD) respectively. Both products, Nexviazyme® (Avalglucosidase alfa powder) and Xenpozyme™ (Olipudase alfa powder), have been granted orphan drug status and approval in several countries, including the US, Australia, EU, UK, and Japan. Sanofi is aiming to launch these therapies in India by the end of 2023 or early next year, subject to completion of all necessary regulatory steps.

According to Dr. Shalini Menon, Country Medical Lead at Sanofi, Xenpozyme is the first therapy indicated specifically for the treatment of ASMD, and the only approved treatment for the disease. She added that this rare and devastating condition affects both children and adults, and the approval of Xenpozyme reflects Sanofi’s unwavering commitment to this historically overlooked community. Menon also noted that Nexviazyme is for both infantile and late-onset Pompe disease, and has shown improvements in respiratory function and walking distance measures.