- Pfizer pauses trial post-child’s death; cause unclear
- Study for Duchenne muscular dystrophy drug delayed by fatality
- Pfizer investigates fatality; competing therapies pose high costs
Pfizer has suspended its drug trial for a rare muscle-wasting disease in response to the untimely death of a child.
A year ago, the child, ranging in age from two to four years, experienced cardiac arrest after undergoing the unique gene therapy.
The precise cause of the death and whether or not it was a result of the treatment, fordadistrogene movaparvovec, remain unknown to Pfizer.
The New York-based pharmaceutical giant stated that the delay would enable it to investigate the fatality “while maintaining the participants’ safety, which is our utmost concern.”
The victim was a participant in a mid-stage study that enrolled children with Duchenne muscular dystrophy between the ages of two and four.
A rare disease that affects boys at a rate of approximately one in every 3,500 male births, the average lifespan of a patient is 25 years.
Although the study was accompanied by three severe adverse events, all of the children fully recovered within two weeks.
Pfizer has discontinued the administration of the identical gene therapy to boys aged up to eight who were enrolled in a separate final-stage study.
In that study, the drug’s potential to impede the progression of the disease was compared to that of a placebo.
If the trials passed, it was positioned to become one of the most expensive medications ever.
The FDA granted accelerated approval to a competing gene therapy developed by Sarepta Therapeutics Inc. last year, making it the only treatment approved for children with DMD.
One dose costs approximately $3 million.
DMD is brought on by a genetic mutation that prevents the body from producing adequate protein dystrophin, which is responsible for maintaining muscle strength.
In its absence, muscles progressively deteriorate, resulting in difficulties with locomotion, sitting, and speech and ultimately impacting the muscles encompassing the lungs and heart.
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By age 11, the majority of patients will typically require a wheelchair.
Pfizer’s medication inhibits their degradation by delivering a truncated yet functional dystrophin protein directly to patients’ muscle cells.
A single administration via infusion is required.
Historically, steroids like prednisone and deflazacort were the only medications used to treat DMD.
They mitigate inflammation, a significant issue in Duchenne, thereby delaying the onset of heart and pulmonary complications and slowing the decline in muscle strength and mobility.
However, these can lead to weight gain, stunted growth, delayed puberty, and a decrease in bone density—all of which increase the risk of fractures—among other severe adverse effects.
