Note for the team: Availability, prices, access programmes and government support (National Policy for Rare Diseases 2021, Centres of Excellence) in India have changed since 2021. Figures below are from the old website and must be updated and reviewed by a scientific advisor before launch.
Key points
- Three disease-modifying treatments have been approved internationally.
- Risdiplam (Evrysdi), taken by mouth, was approved in India in 2021.
- Treatment works best when started early, but it does not replace supportive care.
- Talk to your neurologist and to us about access programmes and support.
Until 2016 there was no approved treatment for SMA, beyond managing symptoms and preventing complications. Since then, three treatments have been approved that target the underlying cause — a shortage of SMN protein.
At a glance
| Risdiplam (Evrysdi) | Nusinersen (Spinraza) | Onasemnogene (Zolgensma) | |
|---|---|---|---|
| Type | Oral medicine | Injection into the spinal fluid | One-time gene therapy |
| How it works | Helps the SMN2 gene make more working SMN protein | Helps the SMN2 gene make more working SMN protein | Delivers a working copy of the SMN1 gene |
| How it is given | Liquid by mouth, daily, at home, for life | Intrathecal injection: 4 loading doses, then every 4 months, for life | A single infusion into a vein |
| Who | Children and adults, from 2 months (per 2021 approval) | All types of SMA | Young children (originally under 2 years) |
| First approved | US FDA, Aug 2020 · DCGI, 2021 | US FDA, Dec 2016 | US FDA, May 2019 |
| In India | Approved and launched (July 2021) | Not available (as of 2021) | Through managed-access programmes (as of 2021) |
Risdiplam (Evrysdi)
Evrysdi, by Roche, was the first SMA treatment approved by the Drugs Controller General of India (DCGI), launched in July 2021 — within 11 months of its US approval. Cure SMA India worked closely with Roche, doctors, the patient community and the regulator to fast-track its approval.
Evrysdi works on the SMN2 gene, helping it make more functional SMN protein throughout the nervous system and body. It is taken by mouth at home every day and must be taken for life. In 2021 the price ranged from about ₹22 lakh to ₹72 lakh per year, depending on age and weight. Cure SMA India has been advocating for GST exemption and wider access.
Nusinersen (Spinraza)
Spinraza was the first SMA drug approved by the US FDA, on 23 December 2016, and is approved for all types of SMA. It is given by intrathecal injection — directly into the fluid around the spinal cord through the lower back. Patients receive four loading doses in the first two months, then a maintenance dose every four months for life.
In 2019, Cure SMA India coordinated with the US-based NGO Direct Relief on a humanitarian access programme for Spinraza for a limited number of Indian patients.
Onasemnogene abeparvovec (Zolgensma)
Zolgensma, developed by AveXis (now part of Novartis), is a one-time gene therapy. A modified, harmless virus carries a working copy of the SMN1 gene into motor neurons, which then start producing SMN protein. It was approved in the USA on 24 May 2019 for children under two.
It is one of the most expensive medicines in the world. A few patients in India have received it through Novartis’s Global Managed Access Program (GMAP).
Supportive care is still essential
Even with medicines, people with SMA need regular care and monitoring. A combination of physiotherapy, occupational therapy, good nutrition and respiratory care can do wonders for quality of life. See our care guides and the standards of care.
Clinical trials
New treatments and combinations are being studied around the world. Ask your neurologist whether a clinical trial might be suitable, or contact us — we share information about trials open to Indian patients.
Getting help with access
Treatment access in India is changing. Our team can explain current options, access programmes and government support schemes. Contact us or register with us so we can keep you informed.