In a push for immediate government action, Haris Beeran, a member of the Rajya Sabha, has formally appealed to the Health Ministry to address the pressing issue of unaffordable access to medications for Spinal Muscular Atrophy (SMA). His letter emphasizes the necessity for local manufacturers to be permitted to produce generic versions of these crucial drugs to significantly reduce costs.
SMA, recognized as a rare disease in India, affects an estimated 8,000 to 25,000 children born each year.
“The absence of systematic screening and registration has hindered our ability to gauge the true burden of SMA in India,” Beeran stated.
Currently, three treatments are accessible for SMA patients: Onasemnogene abeparvovec-xioi (Zolgensma), a gene therapy for children under two years; Nusinersen (Spinraza), which is suitable for both adults and children; and Risdiplam (Evrysdi), a small molecule therapy also applicable to all age groups.
However, the financial burden of these treatments is immense, making them unattainable for many individuals and families. Zolgensma is priced at ₹17 crore, while Nusinersen carries a similarly high cost. The retail price of a single bottle of Risdiplam is ₹6,20,835, translating to expenses that can reach ₹72 lakhs for children and up to ₹1.86 crores for adults requiring approximately 30 bottles annually.
An industry expert has suggested that local manufacturing could reduce the cost of Risdiplam to just ₹3,024 per year for Indian patients.
Beeran underscored the urgent need for governmental intervention, noting that Risdiplam, being a small molecule, is more feasible for generic production compared to the other treatments. Leveraging the technological capabilities of India’s pharmaceutical sector, Risdiplam could be produced locally at a fraction of its current price, ensuring it is accessible to those in need.
Yet, existing patent protections on Risdiplam legally inhibit local companies from producing affordable generic alternatives. Reports indicate that Roche, the patent holder, has sought legal action to prevent Indian manufacturers from creating a cost-effective version of the drug.
Beeran highlighted that Section 100 of the Patents Act 1970 empowers the Government of India to authorize generic producers to manufacture Risdiplam in the public interest.
To assist individuals living with SMA, the government has been urged to allocate research and development funds in collaboration with relevant ministries to explore low-cost gene therapy options like Spinraza. Additionally, he emphasized the importance of implementing SMA screening initiatives, establishing a national registry for SMA and other rare diseases, and creating a national procurement pool for medicines and diagnostics related to these conditions.
Published – January 08, 2025 03:40 am IST
