Spinal muscular atrophy drug: regulatory approval should follow Kerala procurement policy, experts say

Treatment of spinal muscular atrophy (SMA), a rare progressive genetic disorder that destroys motor neurons and weakens muscles, leading to severe disability and sometimes premature death, is emerging as a contentious issue in Kerala.

From 2021, Kerala is procuring a high-cost proprietary SMA drug, Risdiplam from Roche pharma – which costs ₹5.4 million per bottle and was procured by the state government at a negotiated price of about ₹1-1.25 lakh to be provided free to SMA patients in the state.

But now that Natco pharma’s generic version of the drug, approved by drug regulators, is available in the country – at a price 97% lower than its patented version – the state government is considering procuring the generic version, as it will allow the state to expand access to the drug to more SMA patients and use the savings to treat other rare diseases that are equally expensive.

Demand from advocacy groups

However, patient advocacy groups such as Cure SMA have been vocal in Kerala arguing that the efficacy of the generic drug has yet to be tested in clinical trials and that the state government should continue to supply the patented molecule until the generic is proven to be effective.

Public health experts and doctors are now urging state government that public health is all about maximizing access for people, and that unless credible scientific evidence shows that an approved generic is less safe or less effective than the original molecule, regulatory approval should remain the primary basis for purchasing decisions in publicly funded health programs.

Ironically, the patent-versus-generic debate over the SMA drug is something unique to Kerala — the only state that uses public funds to provide free SMA treatment — while in the rest of the country, complaints are about SMA patients not having enough access to the generic drug, which costs ₹15,900 a bottle.

“Generic drugs do not need to repeat clinical trials, they just need to demonstrate bioequivalence – that is, the generic drug should be shown to deliver the same amount of active ingredients into the bloodstream at the same rate as the original. I understand that the mother of an SMA patient is concerned about quality issues, but a generic drug made in India alone can make SMA treatment available to patients in the capital,” said a prominent neurologist.

Nationally approved

“There is no room for debate here as the generic version of Risdiplam has been approved for launch by the national drug regulatory authorities. Generics are what make expensive drugs available to all sections of society. The government would be able to procure the generic SMA drug – Risdinat – at even lower prices so that we can maintain treatment for many SMA patients,” said a senior health official.

He pointed out that there are several other rare diseases and that even the government has to take care of all these patients with the limited resources at its disposal.

“Kerala cannot continue to get the original molecule at high prices when an approved generic formulation is available at a fraction of the cost. An adult SMA patient weighing more than 20 kg needs roughly 2.5 vials every month. Using the money required to purchase one vial of the patented drug, nearly three months of treatment can be provided for an adult patient with a generic,” said B. Ekbal urgently needs a state health policy to buy the generic.

Newly approved generic drugs often collect long-term post-marketing data only after they enter widespread clinical use. Public procurement policy cannot reasonably be based on speculative fears, he added. Since there is no evidence to demonstrate any clinically significant deficiency in the generic formulation, further procurement of a product at nearly eight times the price cannot be justified on speculative grounds alone.

Ultimately, the success of a publicly funded health program should not be measured by how much is spent on drugs, but by how many patients receive timely and effective treatment with available resources, Dr. Ekbal.

Published – 26 Jun 2026 20:44 IST