Novartis investigating second patient death in gene therapy trial

April 22, 2019

Novartis has launched an investigation into the death of an infant participating in a trial for an experimental gene therapy.

The drug, Zolgensma, was developed to treat spinal muscular atrophy (SMA), a rare disease that impacts the part of the nerve system involved in muscular movement. The 22 babies involved in the study were all diagnosed with Type 1 SMA — a more serious form of the disease that can cut life expectancy to less than one year in 50 percent of patients. 

The most recent death, involving a 6-month-old patient, was the second in the Novartis study. An independent investigation found that the first death, caused by respiratory failure, was not related to the treatment. But Novartis reported that second death, linked to respiratory infection followed by neurological complications that can be symptomatic in Type 1 SMA patients, was deemed by preliminary findings to possibly be related to Zolgensma.

Novartis has already filed for FDA approval for Zolgensma. If approved, it will be the second gene therapy to enter the SMA market, with more possibly on the way. Biogen’s SMA therapy, Spinraza, was approved in 2016, and Roche and PTC Therapuetics have also reported positive trial results for their therapy that’s in development.  

Read the full Reuters report.