Will this Pfizer orphan drug emerge as the Duchenne victor?
- Pfizer is currently enrolling patients for phase II clinical trials for treatment of Duchenne muscular dystrophy (DMD) in boys. BioMarin and Sarepta also working to bring drugs to market for the treatment of DMD.
- DMD is a form of muscular dystrophy caused by a genetic abnormality in exon 51. Without intervention, patients are in wheelchairs by adolescence. It impact 1/3500 male births worldwide.
- The safety, efficacy, and tolerability of Pfizer's PF-06252616 is being tested in boys between the ages of 6 and 10. One thing researchers are keeping an eye on is whether or not Pfizer's candidate for DMD can increase muscle mass and function in boys with DMD who have lost muscle and are becoming weaker.
Pfizer's DMD candidate has been granted Orphan Drug designation by the FDA, as well as Fast Track designation. In addition, the European Medicines Association (EMA) granted Pfizer Orphan Medical Product designation for PF-06252616.
But Pfizer is not the only company pursuing an approval for a DMD therapeutic. Both BioMarin and Sarepta Pharma (which has had several challenging setbacks with its DMD development efforts) are working on therapies to address this devastating disease, although Sarepta has hit some recent roadblocks with the FDA over the sample size of its trials.