A rare, severe neuromuscular disease, known as Spinal Muscular Atrophy (SMA), has a new gene therapy treatment approved by the Food and Drug Administration External Site (FDA) in May. Type 1 of the disease, caused by a gene mutation that destroys motor neurons, leads to progressive muscle weakness (including breathing and swallowing) and paralysis. SMA Type 1 impacts babies from birth — about 300 new cases in the U.S. per year — and typically results in death by age two. Zolgensma®, the new treatment from Novartis, replaces this defective gene with a one-time infusion that could cost upwards of seven figures.
But initial results are promising. In an early-stage trial, 15 of 15 (100 percent) patients were alive and without need for permanent ventilation after 24 months of follow-up.1 Additionally, 11 of 12 (92 percent) patients could sit unassisted for more than 5 seconds. Preliminary data from an ongoing late-stage trial support these findings, too.
The only other FDA-approved drug for SMA, Spinraza® External Site (nusinersen), requires a patient to take it for their entire lifetime. Without the long-term data from Zolgensma trials, providers and patients may want to use Spinraza after the one-time infusion of Zolgensma.
Three reasons Zolgensma will likely pass the million-dollar mark
High drug prices have been grabbing national headlines and the attention of state legislatures, and Zolgensma is poised to break records External Site. It's a complex balance: stratospheric prices — estimated at more than $2.1 million per patient — considered alongside clinical results showing a possible cure for this fatal disease. Three important factors come in to play with pricing of rare-disease drugs like this: expensive research and development for the gene therapy, limited number of cases for trialing (and ultimately purchasing) the drug, and in Zolgensma's case, only one dose given for the patient's lifetime.
Coverage decision coming soon
Wellmark Blue Cross and Blue Shield uses several strategies to evaluate and manage high-cost specialty drugs like this, and our independent medical and pharmacy experts will review Zolgensma coverage options in the coming months. We will update this article once a final decision has been made.
Questions? Please contact your authorized Wellmark representative.
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