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Will Pfizer’s muscular dystrophy gene therapy cross the finish line?
Others have tried and failed — could this one yield a happy ending for patients?
In early 2021, a young boy in Barcelona was the first to receive Pfizer’s investigational gene therapy for Duchenne muscular dystrophy (DMD). This was part of a global Phase 3 human trial to test Pfizer’s lead candidate, PF-06939926, in a cohort of 99 patients with DMD-a debilitating genetic condition that causes progressive muscle wasting mostly in males-for which there are currently no approved treatment options.
This is the last lap for Pfizer in the onerous race towards an approved gene therapy for DMD, but the pharma giant isn’t the only competitor. Others with similar gene therapy offerings have sprinted forward with bursts of apparent success, only to end up stumbling and losing their lead.
Take Sarepta Therapeutics, for instance, whose experimental gene therapy SRP-9001 was reported to dramatically improve DMD patients’ conditions in early trials, cutting in half the time it took for one patient in the trial to rise up from the ground and climb a set of stairs. However, these triumphs were short-lived, with no significant differences observed between the placebo and treated groups in mid-stage trials, causing the company’s stock prices to plummet.
