US FDA has approved the most expensive medicine named Zolgensma sold by the Novartis at $2.125 million (around Rs. 14cr) to treat spinal muscular atrophy (SMA), a fatal spinal disease that kills nearly everyone within few years, for children aged below two after doing a genetic test.
The drug targets a defective gene that weakens a child's muscles that they become unable to move, and finally unable to swallow or breathe. It occurs in about 400 babies born in the US every year. The therapy is a one-time infusion that takes about an hour.
‘Babies with the rare genetic fatal disease spinal muscular atrophy could now have a one time treatment option in the form of Zolgensma infusion.’
Zolgensma acts by supplying a fresh copy of the faulty gene, allowing the nerve cells to start producing the needed protein that allows the baby to develop normally.
Babies with the most severe form of the disease who got Zolgensma within six months of birth had limited muscle problems. They stopped losing muscle control, but the medicine can't reverse the damage already done.
Early diagnosis and early treatment is crucial, so Novartis has been working with states to get genetic testing for newborns required at birth. Most states were expected to have that requirement by next year.
Side effects of this new drug includes vomiting and potential liver damage. Patients must be monitored thoroughly for the first few months after treatment.
One more medicine for the disease approved is Spinraza to be given once every four months, sold by Biogen which charges $750,000 for the first year and then $350,000 per year after that.