Pfizer Paused Experimental Gene Therapy Trial Following Boy’s ‘Sudden’ Death

Pfizer Paused Experimental Gene Therapy Trial Following Boy’s ‘Sudden’ Death

According to the latest reports, Pfizer has paused experimental gene therapy following boy’s sudden death. Check out more details about the matter below.

Pfizer pauses experimental gene therapy

Pfizer Inc.,leading pharmaceutical company, has stopped a clinical trial for a new gene therapy intended to treat muscular dystrophy. This decision came after a young patient who received the treatment passed away as a result of cardiac arrest.

The drugmaker announced the sudden death of the child in a letter to the community, which was shared by Parent Project Muscular Dystrophy (PPMD), a non-profit advocacy group.

A child who participated in Pfizer’s phase 2 “DAYLIGHT” study for Duchenne muscular dystrophy (DMD) received an investigational recombinant adeno-associated virus gene therapy called Fordadistrogene Movaparvovec in early 2023.

The child’s identity was not disclosed by the company. The study enrolled children between the ages of two and four and has since concluded, according to Pfizer. Additionally, the initial dosing for a placebo-controlled, randomized Phase 3 trial evaluating Pfizer’s DMD therapy, known as CIFFREO, was also completed in 2023.

The trial aimed to investigate whether the drug fordadistrogene movaparvovec could slow down the progression of Duchenne muscular dystrophy (DMD) in boys aged four to less than eight years, compared to a placebo.

However, after one of the boys who participated in the trial died, Pfizer temporarily stopped administering the drug associated with the cross-over portion of the CIFFREO trial while it investigated the cause of the boy’s death.

The drugmaker is working closely with regulators and the independent external Data Monitoring Committee to gather more information on the potential cause of the boy’s death.

“The safety and well-being of the patients in our clinical trials remains our top priority, and we are committed to sharing more information with the medical and patient community as soon as we can,” the company continued.

They also said: “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.”

Rada Mateescu

Passionate about freedom, truth, humanity, and subjects from the science and health-related areas, Rada has been blogging for about ten years, and at Health Thoroughfare, she's covering the latest news on these niches.

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