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Three disease-modifying SMA therapies exist globally, each working on the SMN1/SMN2 biology differently. Their regulatory and price status in India differs sharply — read carefully before assuming any of these are simply “available”:
1. Zolgensma (onasemnogene abeparvovec), Novartis — one-time intravenous infusion of a self-complementary AAV9 vector delivering a functional SMN1 gene copy directly into motor neurons; it is a single-dose gene replacement, not a repeated therapy. Indicated for SMA Type 1 patients (per the global label, generally under 2 years of age / under a body-weight threshold, since dosing is weight-based and cost scales with body weight). CDSCO approved it for the Indian market in late August 2025. Listed US price is about Rs 17.7 crore ($2.1 million); Novartis has not published a separate Indian price, and one reported case involved an initial payment-plan installment of roughly Rs 9 crore. Pivotal trial data showed 95% survival (event-free, without permanent ventilation) in babies treated before symptom onset, versus roughly 31% survival without treatment — the efficacy gap is real, but requires very early genetic diagnosis (see Topic 6) since the benefit is largest pre-symptomatically. Requires liver function monitoring (ALT/AST, bilirubin) for weeks after infusion and a course of corticosteroids to manage the risk of hepatotoxicity; rare but serious complement-mediated complications (thrombotic microangiopathy) have also been reported.
2. Risdiplam (brand: Evrysdi), Roche — a small-molecule, oral splicing modifier taken once daily as a liquid, at home, for life. It works by shifting SMN2 pre-mRNA splicing to include exon 7 more often, increasing the amount of full-length, functional SMN protein produced from the patient’s existing SMN2 copies. Suitable across ages and across SMA types (1, 2, and 3), unlike Zolgensma’s narrower age/weight-based label. Branded Evrysdi costs roughly Rs 6 lakh per bottle in India (annual cost historically Rs 60–72 lakh depending on weight-based dose). In 2025 the Delhi High Court dismissed Roche’s appeal and cleared Natco Pharma (Hyderabad) to launch a generic version at approximately Rs 15,900 per 60mg bottle — about a 97% price reduction, bringing annual treatment cost to under Rs 5 lakh. This generic approval is currently the single biggest access improvement for Indian SMA families.
3. Nusinersen (brand: Spinraza), Biogen — an antisense oligonucleotide (ASO) delivered by intrathecal injection (lumbar puncture), also a splicing modifier of SMN2 pre-mRNA, mechanistically similar in goal to Risdiplam but delivered locally into the CSF rather than taken orally. Dosing is 4 loading doses in the first ~2 months, then a maintenance dose every 4 months, for life — meaning repeated hospital visits and repeated lumbar punctures indefinitely. As of now it is not officially CDSCO-approved for the Indian market; reported pricing is around Rs 87 lakh per dose, with first-year cost (loading + maintenance) estimated at Rs 4–6 crore and roughly Rs 2–3 crore annually thereafter. Available in India only through a small number of specialist hospitals via special import / compassionate-use approval routes.
Bottom line for families: Risdiplam — especially the Natco generic — is currently the most accessible disease-modifying option in India by a wide margin, both in approval status and cost. Zolgensma is regulatory-approved and offers the strongest evidence for pre-symptomatic Type 1 infants, but remains unaffordable for most families without major fundraising. Spinraza remains the hardest to access on both fronts.
References:
• India approves Zolgensma — https://theprint.in/health/india-approves-zolgensma-one-of-worlds-costliest-drugs-why-its-sparked-hope-but-also-concern/2730423/
• Generic Risdiplam approval — https://www.ocacademy.in/blogs/risdiplam-generic-approval-sma-india/
• Spinraza price and access in India — https://www.impactguru.com/info/spinraza-price-in-india/
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