It has been several months since the United States Food and Drug Administration (FDA) approved Novartis’ gene therapy drug Zolgensma. Zolgensma is meant to be a one-time curative treatment for spinal muscular atrophy (SMA), a rare neuromuscular condition that can lead to death before the age of two. We are now starting to see how insurers will treat such an expensive therapy, priced at $2.125 million per individual.
While Zolgensma isn’t the first gene therapy approved by the FDA, it is held out to be an early test case for the curative promise, and is also one of the most expensive drugs on the market today.
With its high price tag, insurers are acting a bit more cautious than the FDA did when it comes to paying for the pricey drug. The FDA approved the drug for infants less than two years of age with SMA. In the main study Novartis used to support its application for FDA approval, the trial enrolled babies with disease onset before 6 months with the most severe form of SMA, Type 1.
Insurer Treatment
Out of the thirty major insurers tracked by Bernstein, eleven have published policies specific for Zolgensma. While all eleven cover the gene therapy, some are placing limitations, such as age limits and whether patients must be symptomatic.
For example, one of the nation’s largest private insurers, Anthem, sets out six months as the maximum age at treatment and stipulates covered infants must either show symptoms before then or have at most two copies of a related gene called SMN2, which produces a less effective, “back-up” form of SMN protein. Neither of those additional criteria are included in the FDA’s label.
Two other insurers, Horizon Blue Cross Blue Shield of New Jersey and Blue Cross Blue Shield Nebraska, also require infants be younger than the two year mark described in the FDA-approved labeling.
According to Bernstein, Horizon Blue Cross of New Jersey is not covering the gene therapy for patients previously treated with Spinraza (another prescription for SMA), and nine of the companies won’t allow concurrent treatment with Spinraza. Health Net is requiring any patient who’d used Spinraza to prove their disease progressed while on treatment before it will cover Zolgensma.
Some insurers only allow for coverage when patients are symptomatic, a criteria that can raise questions as SMA is also diagnosed via genetic mutations, when symptoms may not always be present at a young age. Bernstein analysts believe that “by making coverage choices that may be legally defensible but deny Zolgensma to patients who could clearly benefit, the payers are sending a clear signal that they are very unhappy with the price of Zolgensma.”
It’s a long road ahead to see what the entry of Zolgensma will bring to the market, but it seems as if there is a lower-priced competition on the market, there is likely to be at least some pushback on price.