HQ Team
May 8, 2024: A young boy, treated for a rare genetic disease, died during a mid-stage clinical trial conducted by Pfizer, according to a statement posted by a non-profit group on its website quoting a company statement.
Duchenne muscular dystrophy, a genetic disease, leads to progressive muscle degeneration and weakness.
It affects about one in 3,500 male births worldwide. It is usually diagnosed between three and six years of age, according to the National Organization of Rare Disorders.
“The loss of any member of our community is deeply felt, and our hearts ache with the family and loved ones of this courageous young child, whose bravery and commitment to advancing research for Duchenne will forever be remembered and honoured,” according to a statement from the Parent Project Muscular Dystrophy.
Community letter
The advocacy group stated the study for evaluating the safety and dystrophin expression of the fordadistrogene movaparvovec was conducted in boys aged between at least two years and less than four years old.
Pfizer has issued a community letter expressing sympathies to the boy’s family, friends and those who were closest to his care.
“We are deeply saddened to share that a patient participating in our Phase 2 DAYLIGHT study for Duchenne muscular dystrophy has passed away suddenly.
“We do not yet have complete information and are actively working with the trial site investigator to understand what happened,” according to the letter published on the non-profit group’s website.
Early 2023
“The patient received the investigational gene therapy, fordadistrogene movaparvovec, in early 2023.”
Initial dosing for the Phase III trial for boys between four and eight years, which utilises a crossover trial design, was also completed in 2023, Pfizer stated.
“We have decided to pause dosing associated with the crossover portion of CIFFREO, and we are working with regulators and the independent external Data Monitoring Committee as we learn more about this event.”
Pfizer stated that the safety and well-being of the patients in clinical trials remained the company’s top priority, and “we are committed to sharing more information with the medical and patient community as soon as we can.
Adverse events
“We are also aware that many in the patient community are hopeful about the potential benefit of fordadistrogene movaparvovec for the treatment of DMD, and we will continue to collect data from our trials to evaluate its ability to address this disease.”
In December 2021, Pfizer reported the death of a young male patient in a phase 1 trial. The company paused screening and dosing in the study and the Food and Drug Administration placed a clinical hold on the study.
The adverse events forced Pfizer to make changes in the study protocol to exclude patients with certain gene mutations.
