Parents of children with Spinal Muscular Atrophy (SMA) in Kerala are worried about the State government’s move to buy a generic version of a life-saving medicine to be distributed among them for free, as it has not undergone clinical trials so far.

The drug now being given is innovator risdiplam, a precision SMN2 exon-splicing modifier that acts at the genomic level. Over 120 children have benefited after the government launched the free drug distribution scheme since July 2021. Clinical trials of innovator risdiplam have demonstrated a one-year survival benchmark of approximately 92%.

According to doctors, SMA is a fatal, progressive neurodegenerative disease in which inadequate treatment exposure may result in irreversible motor neuron loss. Unlike many conventional therapies, lost motor function cannot simply be recovered later through switching products or adjusting doses after disease progression has occurred, they say.

K. Razeena, director (Patient Empowerment), CureSMA Foundation of India, a parents-led community initiative, says that though the currently available generic version is reported to be more affordable and has the marketing approval from the Drugs Controller General of India, human bioequivalence studies, clinical safety data, or long-term efficacy outcomes in SMA patients are not available as yet.

She points out that recent judicial verdicts concerning risdiplam relate exclusively to patent litigation. “These proceedings do not evaluate the safety, efficacy, therapeutic equivalence, or clinical performance of generic formulations. Decisions about treatment in a life-threatening disease must be guided by scientific and medical evidence rather than legal determinations,” Dr. Razeena says.

According to parents such as Manju Rajesh from Thrissur and Siddhi Vijesh from Kochi (names changed), their children are currently clinically stable on innovator risdiplam, with documented survival benefits and continued motor milestone gains. They say that any non-medically justified substitution in these children has raised concerns about potential clinical deterioration.

A senior Health department official, meanwhile, points out that the using the affordable generic version will lead to expansion of the scheme to more patients.

Dr. Razeena claims that the parents are not opposed to affordable treatment. “Rather, we believe that the government and procurement agencies should await publication of substantial human exposure data, bioequivalence studies, safety findings, and real-world clinical outcomes from large numbers of SMA patients before considering any procurement change,” she says.

At present, none of the major centres of excellence in the country using Central government funding for SMA care has adopted generic risdiplam for routine procurement. Also, there is no precedent for routine use of a genomic or exon-splicing therapy in the country without published human data demonstrating safety, efficacy, and meaningful patient benefit.

The patients’ families believe that introducing such therapies into routine clinical practice without supporting evidence will represent a significant departure from established scientific standards. Even small variations in drug exposure or product performance could have serious consequences in a disease where neurological damage is irreversible.

While the market price of risdiplam is approximately ₹5.2 lakh per bottle, the previous Left Democratic Front government was able to negotiate directly with the manufacturer and procure the medicine for less than ₹1 lakh per bottle.

According to sources, children in the 0–12 age group who previously faced progressive disability and early mortality have achieved new developmental milestones after they were administered the drug. Even severely affected Type 1 SMA children have shown meaningful improvements, including the ability to take baby steps with hand support.