Priced at $ 2.125 million, US regulators have approved the most expensive medicine ever for a rare disorder that destroys a baby’s muscle control. Out-of-pocket expenditure will vary for patients based on their insurance coverage. Swiss drug maker, Novartis, has developed this treatment which is a gene therapy that treats the inherited condition called spinal muscular atrophy.

Every year, the disease is estimated to affect 400 babies born in the US. The treatment is intended to treat a defective gene that weakens a child’s muscles to such an extent that they become unable to move, and eventually unable to swallow or even breathe. The drug works by supplying a healthy copy of the faulty gene, which allows nerve cells to then start producing the required protein. This ceases the deterioration of the nerve cells and allows the baby to develop normally.

On 24th May 2019, the US FDA approved the treatment, called Zolgensma, for all children under age 2 who are confirmed by a genetic test to have any of the four types of the disease. The therapy is a one-time infusion that takes about an hour.