Experts seek affordable access to medicines for treating Spinal Muscular Atrophy

Experts seek affordable access to medicines for treating Spinal Muscular Atrophy

Seeking urgent Union government intervention to overcome the lack of affordable access to medicines for treating Spinal Muscular Atrophy (SMA) and the need to allow local manufacturers to make generic versions of the drugs to cut down costs, Haris Beeran, Rajya Sabha member has written to the Health Ministry.   

In India, SMA is classified as a rare disease and there are nearly 8,000 children to 25,000 children born annually with it. 

“Without systematic screening and registration, we do not have accurate numbers of SMA disease burden in India,’’ he noted.

Currently three treatments are available for SMA patients – Onasemnogene abeparvovec-xioi (Zolgensma) a gene therapy for treating children below two years of age, Nusinersen (Spinraza) (used to treat both adults and children), and Risdiplam (Evrysdi) is a small molecule which is also used to treat both adults and children. 

However, the cost of these medicines is prohibitive and, therefore, beyond the reach of both the Central and State governments and as such most people. 

The cost of Zolgensma is ₹17 crore, Nusinersen is also expensive while the maximum retail price of one bottle of Risdiplam is ₹6,20,835. Therefore, the cost of Risdiplam can be between ₹72 lakhs for children, and the cost can go up to 1.86 crores because an adult needs nearly 30 bottles a year, he said in his letter.

According to an expert, through local production, Risdiplam can be made available to Indian patients for just ₹3,024 per year.

Mr. Beeran added that the current situation warrants urgent government intervention. Since it is a small chemical molecule, the generic version can be produced easily compared to the first two medicines. Considering the technological capacities of the Indian pharmaceutical industry Risdiplam can be produced locally at a fraction of the current price and made available to all needy people. 

However, the patent protection on the Risdiplam legally prevents the local companies from producing the affordable generic version. 

According to news reports, Roche, the patent holder of Risdiplam, approached the Delhi High Court to prevent an Indian company from producing the affordable version of Risdiplam. 

The MP has said that Section 100 of the Patents Act 1970 provides enough powers to the Government of India to authorise one or more generic manufacturers to produce Risdipalm in the public interest.

“To help persons living with SMA, the government is also requested to  provide research and development funds in coordination with relevant Ministries/departments to develop low-cost gene therapy (Spinraza) options for SMA. Also, measures needed to be taken for screening for SMA and establish a national registry for SMA along with other rare diseases. India should also establish a national pool procurement of medicines and diagnostics for SMA and all other rare diseases,’’ he said.