This week, the United States Food and Drug Administration approved a new treatment for a genetic disease called spinal muscular atrophy. This somewhat rare condition causes muscle deterioration in infants, which could claim their live by age 2.
FDA Acting Commission Dr. Ned Sharpless comments that this approval signifies yet another milestone in harnessing the transformational power of gene and cell therapy for treating a variety of diseases. He notes, “The potential for gene therapy products to change the lives of these patients who may have faced a terminal condition, or worse, death, provides hope for the future.”
This gene therapy treatment is called Zolgensma, which is marketed by AveXis (a chapter of Novartis).
While this announcement is certainly excellent news for those who might be looking for a treatment, it should also be noted that the price tag for the one-time treatment is $2.125 million. It was expected that the price would be in the low millions, but the announcement of the drug’s price immediately reignited the standing controversy about the skyrocketing cost of drugs (and how we are supposed to be able to pay for them).
OF course, Novartis argues that this price definitely met their price targets for cost-effectiveness since this approval puts a drug on the market that is nearly half the price of a competing treatment that is already on the market, Spiranza, could cost over ten years. Novartis also also promised that they company will provide patient support through payment programs aimed at keeping out-of-pocket expenses as low as possible for as many patients as possible.
The United States National Institute of Neurological Disorders and Stroke explains that spinal muscular atrophy is a genetic disorder (inherited) resulting from a genetic defect which brings about the death of specific nerve cells responsible for muscle movement. Those with this condition find it difficult to walk and talk or even to swallow or breathe. The active component of Zolgensma replaces this defective gene with a working copy that can restore life to these cells before symptoms develop.