Second death in Novartis gene therapy trials at the study

(Reuters) – Novartis AG, which this week announced the results of a positive tentative trial for its experimental gene therapy for vertebral muscle atrophy, announced Friday that an investigation was underway to determine if a second death could be related to the treatment.

Novartis has filed an application for Zolgensma gene therapy approval with the US Food and Drug Administration, and a decision is expected in a few weeks. The FDA submission was based on the results of a clinical trial of 15 babies treated with Zolgensma.

But Novartis has expanded its clinical trial program – presenting Tuesday in Orlando, Florida, the provisional results of the Muscle Dystrophy Association for 22 babies with type 1 ADS, the most severe form of the disease. The data showed that treatment with Zolgensma resulted in encouraging progress in motor skills such as the ability to sit down. One patient died from respiratory failure, which the investigator and an independent monitor found to be unrelated to gene therapy.

SMA, which can lead to paralysis, breathing difficulties and death, is the leading genetic cause of death in infants.

Novartis officials also revealed that in addition to this death, a 6-month-old patient with type 1 AMS had recently died from Zolgensma treatment as part of the company's European trial.

"Preliminary results indicate that this occurred in the context of a severe respiratory infection followed by neurologic complications in a symptomatic symptomatic SMA patient, and that the investigator felt that it could be related to the treatment, "Novartis spokesman Eric Althoff said Friday.

He added that an autopsy had been performed and that the results were pending. In the meantime, investigators and regulators have been informed.

Gene therapies use viruses designed to transport healthy genetic material into a person's cells to replace defective or mutated genes that cause disease or disease. Zolgensma

"As we know more, we will provide more updates," said Althoff.

Novartis estimates that in the absence of treatment, 50% of babies with type 1 ADM will not survive or will require permanent respiratory support before the age of 10 and a half months .

The company announced that its price for Zolgensma would be determined during negotiations with health plans, but estimated that gene therapy would be cost effective at a cost of $ 4 to $ 5 million as a one-time treatment.