Receives Subject Expert Committee recommendation to bring new therapies of Pompe, ASMD to India
Bangalore, February 27: Sanofi Speciality Care today reaffirmed its commitment to rare diseases in India on the occasion of Rare Disease Day, stating that it will continue with its efforts in empowering the lives of patients with rare diseases by offering transformative healthcare options.
Treatment & Diagnosis
• Diagnostic Program “Disha”, a formal national level testing program offering free-of-cost testing of samples of suspected cases. Over 6000 samples have been tested under this program since its inception in 2013. The samples are tested at the genetics laboratory of Sir Gangaram Hospital, New Delhi and MedGenome, Bengaluru.
• Pratigya, Patient Support Program: Offer free of cost home infusion option for patients on treatment, initiated in 2021.
• Three month’s fellowship programmes in Genetics offered at 8 key centres since 2013, in collaboration with the Society for Indian Academy of Medical Genetics.
Rare Disease Day is the globally-coordinated movement on rare diseases, working towards equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease. Sanofi has been supporting the rare disease day event commemoration in India for 13 years now, ever since the first event was commemorated in 2010 at the Sir Gangaram Hospital, Delhi.
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). Since its incorporation in India in 2007, we have been working not only towards enhancing awareness of rare diseases, providing free diagnostic support, but also building capacity of the clinicians, and supporting Centres of Excellence (CoEs) in public and private institutions.”
According to Mr Raina, “The company also has a global humanitarian program aimed at providing free life-saving treatment to patients in India diagnosed with Lysosomal Storage Disorders (LSDs). Under our India Charitable Access Program (INCAP), we have been supporting patients for Gaucher disease, Pompe disease, Fabry disease and MPS I disease with life-saving Enzyme Replacement Therapy. Many of the children receiving therapy under this programme are now in their teens and adulthood, and are able to lead near-normal lives.”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.
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.”
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.
According to 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.”