FDA has approved the medicine for spinal muscular atrophy, the medicine is a gene therapy and it targets a defective gene that weakens a child's muscle so dramatically that they become unable to move, and eventually unable to swallow or breathe. It strikes 400 babies in the U.S. every year.
The new medicine, from Swiss drugmaker Novartis, is said to help infants that usually die of the disease by age 2.
The disease is caused by a faulty gene, which prevents the body to produce a protein necessary for healthy nerve cells. The drug works by supplying a healthy copy of the faulty gene, to allow the nerver cells to produce the protein.
However, this is the most expensive medicine ever. The treatment is priced at $2.125 million. Out-of-pocket costs for patients will vary based on insurance coverage.
Novartis said it will let insurers make payments over five years, at $425,000 per year, and will give partial rebates if the treatment doesn't work.