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.
The FAMILY PODIATRY CENTRE is 7 years old. We have 3 branches and have a presence in Singapore and Malaysia. We are a multidisciplinary
clinic focusing on all conditions that affect the lower limbs.
Article by Clinical Director and Founder of Family Podiatry Centre, Mark Reyneker