New costly 'magical' drug can cure leading cause of infant deaths

A new gene therapy drug made by Novartis is about to come on the market with a potential price tag of $2 million. The drug is called ‘Zolgensma’ and has the ability to cure spinal muscular atrophy (SMA) which is an inherited disease and often kills infants before the age of two. According to Dr. Mikhail Varshavski "we are making great progress."

“If you have a medication that can treat that illness, and not just treat it Maria, cure this illness, that’s magical, that’s amazing," Dr. Mikhail Varshavski  know as "Dr. Mike" told FOX Business' Maria Bartiromo Wednesday.

SMA is a disease that robs infants of physical strength by attacking the motor nerve cells in the spinal cord. It removes the ability to walk, eat, and even breathe. It’s the number one cause of death for infants. Babies with type 1 SMA rarely survive beyond the first few years of life, while most children with type 2 survive into adulthood. Types 3 and 4 don’t usually affect life expectancy. There are between 400 to 500 babies born in the U.S. each year with the disease.

As it awaits an FDA decision, Novartis has been working to build its case for the one-time treatment expected to carry a price tag in the millions of dollars. The Swiss drug maker, Novartis has suggested a price of $4 million to $5 million for Zolgensma, but analysts estimate it will cost around $2 million.

“When you hear that it’s at such a high cost, at $2 million for a single treatment, that’s scary. Not only to patients, but also to insurers,”  said Varshavski, “If you’re going to have a lifesaving treatment, a curing treatment, but unaffordable, what’s the use in having that treatment?”

Parents are bracing for a battle if the insurance industry balks at the price, but may be left with little options.

“If a parent has a child born with spinal muscular atrophy, they can’t choose not to purchase this medication. So whatever it costs, whatever insurance plan they have to buy they’re gonna pay for it,”  said Varshavski. “As a doctor, my thing is, we need to have access to medications, we need to have reasonable prices for medications.”


According to the FDA, The Orphan Drug Act (ODA) was passed in 1983 to address the concern that pharmaceutical manufacturers were not pursuing drug development for diseases that affect a small number of people. The concern was in part that companies viewed pursuing these therapies as undesirable because the markets for them are small in comparison with the markets for more widespread chronic diseases. In order to help and promote the development of orphan drug therapies, the ODA provided companies that engaged in research for such drugs with fewer than 200,000 patients with tax incentives, research subsidies, and extended patent protection.