Pfizer announced that they will begin clinical trials in the USA in experimental gene therapy studies for DMD, a type of muscle wasting disease. The announcement was made after the US Food and Drug Administration (FDA) lifted its decision to hold the advanced Pfizer trial.
The FDA put Pfizer’s request for the trial on hold after a patient died in another early-stage study of Duchenne muscular dystrophy (DMD) treatment. The work was stopped.
Pfizer reported that the FDA granted permission after n data reviews and the trial’s adjustment to include a seven-day hospital stay to closely monitor patients receiving gene therapy.
Advanced work was underway in 11 countries before it was stopped. Pfizer noted that so far they have received permission to restart work from countries such as the UK, Canada and Taiwan.
DMD, which is caused by a mutation in a gene, causes the protein called dystrophin, which is responsible for ensuring the integrity of muscle cells, cannot be synthesized and disappears. The disease is mostly seen in boys.
Pfizer’s treatment is based on producing a shortened version of dystrophy, one of the longest gene sequences in the human body.
The company expects all units to be operational around the world by the end of June for further research.