What is the controversy over the spinal muscular atrophy drug in Keralam? Explained

The story so farAn initiative launched by Keralam government to make the treatment for Spinal Muscular Atrophy (SMA) accessible to more patients is now courting controversy.SMA is a rare, life-threatening genetic disorder that progressively weakens muscles, severely impairing both mobility and respiratory function. While a complete cure remains elusive, the launch of Risdiplam, marketed as Evrysdi by Roche Pharmaceuticals, the first at-home, oral treatment for SMA, gave hope to thousands of SMA patients. Risdiplam was found to have a significant effect on the progression of the disease, by slowing down motor degeneration, improving physical milestones and stabilising respiratory function.Risdiplam was launched in India in July 2021, making it the first and only approved drug in the country to manage SMA. But the drug’s astronomical cost made it inaccessible to most families.Keralam, which has been giving Risdiplam free of cost to eligible children since 2022, made a switch to the less expensive generic version of the drug in October this year . But since then, a section of parents of children living with SMA children have urged the government to switch back to the expensive, branded version.Last week, when the generic drug was dispensed for the first time at SAT hospital, a Centre of Excellence for Rare Diseases in Thiruvananthapuram, a sticker on the bottle which said “Physicians’ sample/for clinical trials” reopened the branded versus generic debate and a new allegation, that drugs meant for clinical trials were being used on children How Keralam began SMA treatmentKeralam was the first State to give Risdiplam free of cost through government hospitals in 2022 and an SMA clinic was subsequently opened at SAT hospital. Though initially Risdiplam was given free only to children under 12, in 2026 the government announced an extension of coverage to genetically confirmed SMA Type 2 patients up to age 25, with ₹30 crore allocated in the State budget for this. However, as of now, 109 Type 1 and 2 children with SMA below the age of 12 are being given Risdiplam, free.Risdiplam, under its trade name Evrysdi, used to cost ₹6.3 lakh for a bottle (current price ₹4.44 lakhs), which the State was getting at a negotiated price of ₹5.4 lakh per bottle. Under the patient assistance programme, Roche used to give 11 bottles free of cost to the State for every four bottles it purchased. The State’s annual requirement was estimated at around 2,500 bottles and the annual financial commitment was around ₹23 crore. The State, therefore, was up against enormous cumulative expenditure as more children became eligible and existing patients required continued treatment.The decision to switch to genericA technical experts committee in Keralam met several times to evaluate if the generic alternative, Natsmart, which has all regulatory approvals, and which was ready for commercial distribution in October 2025, could replace Roche’s Evrysdi. Natco Pharma, through a historic Delhi High Court verdict, had won the right to marker the generic alternative of Risdiplam at a dramatically reduced price of ₹15,900 per bottle, against Roche’s Rs. 6.3 lakhs per bottle.A bottle of Evrysdi on which Roche holds a patent, typically lasts 12 days, meaning the annual maintenance cost for a patient would amount to a crushing ₹60-70 lakh. With Natsmart, the generic alternative, the cost would be under ₹5 lakh. In February 2026, the Keralam government gave the go ahead for Natsmart, and purchase processes were initiated.What are parents’ concerns?Parents and advocacy groups including the Cure SMA Foundation of India claim that the branded medication, Evrysdi, has a proven, long-term track record of safety and efficacy, while the new generic version lacks dedicated human bioequivalence or long-term clinical studies specific to paediatric patients. They claim that a sudden switch to an unverified or less effective generic formula could reverse the critical motor and survival progress already achieved by children who are currently stable on the original drug. They have been continuously petitioning the government asking that their children be allowed to continue on Evrysdi.What do doctors say?Public health experts have hailed the government’s decision to switch to the generic drug. They say that arguments challenging the efficacy of the generic formulation and alleging a lack of ‘bioavailability’ are entirely unscientific and baseless.Bioavailability denotes the rate and extent to which an administered pharmaceutical substance enters systemic circulation through the digestive tract. A bioequivalence study normally compares the rate and extent of absorption of the generic with the reference product. The World Health Organization (WHO) recognises this as an important way to establish interchangeability. However, bioequivalence studies are not required for every generic medicine. The WHO’s guidance on generic medicines explicitly recognises circumstances in which equivalence studies can be waived. One such circumstance is when the generic and reference products are pharmaceutically equivalent oral solutions containing the same active ingredient at the same molar concentration, subject to the applicable requirements for excipients.In this case, both products contain Risdiplam. The Central Drugs Standard Control Organisation (CDSCO) approved Natsmart and the regulator’s position is that a bioequivalence study is not required for this particular oral solution. Public health experts also say that the absence of a conventional bioequivalence study does not necessarily mean the regulator approved the drug without adequate evidence. A regulator may accept a scientifically-justified waiver.Doctors also point out that Natsmart is being prescribed by many premier teaching hospitals in the country and several patients are already using it in Keralam too, for the last five months, without any complaints.Public health is about maximising access to people, and unless credible scientific evidence demonstrates that the approved generic is less safe or less effective than the original molecule, regulatory approval should remain the primary basis for procurement decisions in publicly-funded health programmes, experts have argued.The current controversyThe current controversy centres around an alarm raised by parents of children with SMA, after they pointed out that the bottle of Natsmart dispensed to them was labelled, ‘Physicians’ sample/for clinical trials’. Though the authorities clarified later that these were drug sample bottles they were forced to source directly from Natco and not part of the government supply (which was delayed), the issue has become something for Opposition parties, particularly the CPI(M), to latch on to. It has also served to perpetuate fears and misgivings about the generic drug among SMA families.What is happening at present?SAT Hospital authorities, who are responsible for the SMA clinic, have now decided to hold a meeting of families to assuage their concerns. They say that several meetings had been held earlier too, to reassure parents about the switch from Evrysdi to Natsmart. However, the new controversy over ‘clinical trial’ drugs may have undone some of the previous work. The labelling issue is clearly separate from the concerns about the switch to generic medicine and the families’ demand for scientific evidence and transparent communication is valid, authorities admit.