Novartis Reports Baby’s Death During Breakthrough Gene Therapy for SMA-1 

Bloomberg recently reported Novartis declared the death of a six-month-old baby, possibly the result of a Zolgensma gene therapy experimental treatment. The baby had spinal muscular atrophy,  which is a horrific and devastating genetic disease affecting the part of the nervous system controlling voluntary muscle movement.  The disease triggers the loss of nerve cells known as the motor neuron.

The Swiss pharma noted in an emailed statement  the death occurred in a late-stage trial in Europe still involved with patient enrollment. The baby experienced a severe respiratory infection followed by neurological complications. Bloomberg’s James Paton reported that autopsy findings are pending. A lot of press has focused on Zolgensma.  TrialSite News profiled  it as part of Novartis’ fundamental pivot from blockbuster to precision and targeted platforms or from “volume to value.” It noted by some estimates, the sponsor was seeking a targeted treatment price of $4 million.

This treatment represents a new “burgeoning field” focusing on gene therapies that can be used to permanently address “debilitating and sometimes lethal flaws in critical genes” as they advance through clinical trials reported Paton.  Apparently, it was the second death in the Avexis-led trials reported by the sponsor. Avexis was acquired by Novartis for  a staggering $8.7 billion.  Reuters Deena Beasley covered the second death and the investigation a whether it is correlated to the treatment.

Ms. Beasley reported Novartis filed for US FDA approval of Zolgensma based on the findings of clinical trials involving 15 babies. Novartis reported Reuters expanded on the program including results from 22 babies with Type 1 SMA (the most serious form of the disease). The results evidenced positive progress; however one baby died from respiratory failure. An investigator and independent monitor offered the opinion it was unrelated to the treatment according to Reuters.

SMA is deadly—in fact, it is the leading genetic cause of death in infants and a terrifying situation for any parent. New advanced gene therapies such as Zolgensma are based on engineered viruses that carry healthy genetic material into the patient’s cells to replace the bad or mutated genes that cause the disease in the first place.

Reuters reported Novartis estimates that 50% of babies afflicted with SMA Type 1 will die (or will require permanent breathing support by 10.5 months old) and hence the astronomically high price of $4 to $5 million. They are in ongoing discussions with payers.  BiopharmaDive reports Novartis is due an FDA decision soon.