Pfizer’s gene therapy clinical trial, which was halted late last year due to patient deaths, is now apparently resuming. FDA has lift Clinical pause on the pivotal Duchenne muscular dystrophy study, which will now be conducted in a more limited manner with new safety measures.
Going forward, Pfizer’s gene therapy foradistrogene movaparvovec will be tested only in Duchenne patients who still have the ability to walk.this The deaths reported last December were from Phase 1b of the program in a group of patients in more advanced stages of muscle-wasting disease. These patients, who had lost the ability to walk, received higher doses of gene therapy.
Pfizer said on Thursday that it had resolved the FDA’s requirement for gene therapy efficacy testing. The drugmaker is also revising the clinical trial protocol and now requires patients to be hospitalized for a week after dosing so they can be monitored.
Pfizer’s contribution to the Duchenne community and Post online Muscular Dystrophy by group parent project.
Duchenne results from a genetic mutation that affects the body’s ability to make dystrophin, an important muscle protein. The disease causes progressively worsening weakness of the skeletal and cardiac muscles. Patients usually only live into their 20s. Sarepta Therapeutics gets FDA-approved treatment and NS Pharma can slow the progression of the disease, but these drugs are only available for patients with certain genetic traits. Pfizer Gene Therapies provides a gene for the production of dystrophin. It’s designed to restore some muscle protein production and offers a potential one-time treatment.
Pfizer isn’t the only company taking a gene-drug approach to Duchenne. Solid Biosciences’ lead gene therapy candidate, SGT-001, is in Phase 1/2 testing. Solid encountered setbacks with this gene therapy, Two independent clinical trials withstood the need for change to patient safety trial design. Solid’s next-generation Duchenne gene therapy candidate, SGT-003, is preparing for clinical trials.On Wednesday, the Cambridge, Massachusetts-based biotechnology company announced a reorganization This will reduce headcount by 35% so that resources can be devoted to both projects.
Meanwhile, Sarepta, also in Cambridge, reach critical test Its Duchenne gene therapy SRP-9001. To date, the program has not reported any deaths or adverse events to warrant a clinical hold.
In addition to receiving FDA approval to resume gene therapy clinical trials, Pfizer said regulators in the U.K., Canada, Taiwan, Spain and Belgium have also given approval to restart Phase 3 trials. The company expects nearly all sites to be reopened by the end of June.
Pfizer also said it recognized the need for new treatment options for non-outpatient Duchenne patients. The company said it will continue to work with the trial’s external data monitoring committee, as well as gene therapy experts, to determine how gene therapy can be administered to this group of patients with more advanced disease.
Photo: Dominica Reuters/AFP, via Getty Images
