The 6-month-old patient died during a Phase 3 European trial testing the gene therapy for spinal muscular atrophy, a muscle-wasting disease. Because the patient died of a respiratory infection followed by neurological complications, an investigator believed the event could be related to the treatment.
The death, which brought up safety concerns about the treatment, also prompted an investigation by the drugmaker.
The investigation and coroner report revealed that the cause of death was brain damage from oxygen deprivation, rather than brain damage from a toxic drug agent. This revealed that the spinal muscular atrophy caused the infection, which lead to oxygen deprivation and caused the death.
The report helps allay concerns about the risks of the $2.1 million-per-patient treatment.
Read the full report here.
More articles on pharmacy:
Carcinogen concerns prompt Sandoz to halt global distribution of heartburn drug
DEA raids some West Coast Kaiser Permanente pharmacies
California biotech files for bankruptcy
Becker’s 3rd Annual Fall Chief Pharmacy Officer Summit is the top tier event that the people leading the future of pharmacy convene. This 3-day event is built specifically for Chief Pharmacy Officers and senior pharmacy leaders. The sessions examine the workforce, financial and clinical priorities shaping pharmacy today. Becker’s is where candid discussions flourish and new ideas thrive. Becker's 3rd Annual Fall Chief Pharmacy Officer Summit
