Published 27 May 2019
Novartis’s Biologics License Application (BLA) for Zolgensma, designed to tackle the genetic root cause of spinal muscular atrophy (SMA) Type 1, a deadly neuromuscular disease, was accepted by the FDA. According to Pharmaceutical Daily News, SMA is caused by a defective or missing SMN1 gene, and without a functional SMN1 gene, infants with SMA Type 1 rapidly lose the motor neurons responsible for muscle functions such as breathing, swallowing, speaking and walking. If left untreated, a baby’s muscles become progressively weaker eventually leading to paralysis or death, in most cases by his or her second birthday.
This is why Novartis says that pricing the drug Zolgensma at $2.1 million is fair. According to CNBC, Novartis said it’s “working closely with insurers to create 5-year agreements based on the success of the treatment as well as other novel pay-over-time options.” It’s currently in “advanced discussions” with more than 15 insurers on payment options. Shares of Novartis were up nearly 4% late-afternoon Friday. The once-off gene therapy is said to be a milestone in the transformational power of gene and cell therapies to treat a wide range of diseases.
Many debilitating conditions like SMA Type 1 have their roots in genetics. While not as immediately life-threatening, there are hereditary foot conditions like bunions which can severely affect quality of life for patients as well. If you or anyone in your family suffers from such ailments, we recommend seeing a podiatrist early to seek treatment.