Zolgensma approved in India: What is the ₹17cr drug
India just approved Zolgensma—a one-time gene therapy for Spinal Muscular Atrophy (SMA) in children under two.
This is a big step for families dealing with this rare disease that weakens muscles and movement.
With a US price tag of about ₹17.7 crore, it's now the costliest medicine ever cleared in India.
Zolgensma replaces faulty gene
Zolgensma works by swapping out the faulty SMN1 gene with a working version through a single IV dose.
If given early, it can stop severe SMA type 1 from progressing.
The local approval means Indian kids can get treated faster—no more long waits or relying on overseas charity programs.
Around 4,000 babies are born with SMA in India every year.
One dose vs. lifelong treatment
Unlike other treatments like Spinraza or risdiplam that require lifelong use, Zolgensma is just one dose.
Trials show 95% of infants with SMA type 1 treated before the onset of symptoms and before age two survive, compared to 31% without the drug.
Hospitals have already helped some patients using crowdfunding and special access programs, but doctors will need to monitor kids closely for possible liver side effects after treatment.