ICYMI: Infant Death in Trial Unrelated to Spinal Muscular Atrophy Gene Therapy

The gene therapy Zolgensma recently won approval last summer in 2019 to be used for infants who have spinal muscular atrophy.

The company AveXis, the makers of the Zolgensma gene therapy, have recently presented interim data from recent studies. A big part of their announcement had to do with the news that one infant in the clinical trial died during the study.

When the infant died back in April of last year, the investigator for the clinical trial stated that there was a possibility the therapy might have been a cause, so this update was big news for the company and potential consumers.

Based on the coroner’s report, the infant in the clinical trial died from brain damage caused by a respiratory tract infection. This respiratory infection resulted from spinal muscular atrophy type 1 and did not have to do with the Zolgensma treatment.

Zolgensma has officially been approved by the FDA as of May 2019. Another update is that a new clinical trial will soon be launched to determine if higher doses of Zolgensma might provide better effectiveness for more patients.

What is Spinal Muscular Atrophy (SMA)?

Spinal muscular atrophy is a rare hereditary disease. People born with the disease will have gradual muscle weakness and wasting away of muscles that gets worse and worse as they age. Type 1 is the most common and most severe. Babies born with Type 1 SMA usually do not live past early childhood because of respiratory failure. There is also type 2 that is not as severe but can still be fatal; people born with this type may live into their 30s. Type 0 is the most deadly form of SMA where most patients die in infancy.

Zolgensma has also found itself in murky water recently because the FDA revealed last month that some of the preclinical data for Zolgensma had been adjusted and tampered with and this information was not revealed to the FDA until after the medicine had already been approved. Zolgensma makers claimed that they needed the time to do a thorough investigation before telling the FDA; nevertheless, two scientists involved were fired from the company following the scandal.

Read more about the company and Zolgensma here.


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