The Food and Drug Administration (FDA) has granted Fast Track designation to the investigational gene therapy candidate, PF-06939926 (Pfizer), for the treatment of Duchenne muscular dystrophy (DMD).<\/p>\n
PF-06939926 is a recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of human dystrophin gene under the control of a human muscle-specific promoter. The Company has chosen the rAAV9 capsid due to its potential to target muscle tissue.<\/p>\n
The designation was based on data from an ongoing phase 1b study evaluating the safety and tolerability of a single intravenous infusion of PF-06939926 in 9 ambulatory boys with DMD aged 6 to 12 years. Preliminary results showed that PF-06939926 was well tolerated during the infusion period and dystrophin expression levels were sustained over a 12-month period.<\/p>\n
The Company plans to launch a double-blind, placebo-controlled phase 3 study to evaluate the efficacy and safety of PF-06939926 in boys with DMD. The study will include patients who are at least 4 years old and less than 8 years old; all participants will need to be on a daily dose of glucocorticoids for at least 3 months prior to enrolling and to stay on daily glucocorticoids for the first 2 years of the study. The primary outcome of the study (change from baseline in North Star Ambulatory Assessment) will be assessed at 52 weeks; patients will be followed for 5 years after treatment.<\/p>\n
The FDAs decision to grant our investigational gene therapy PF-06939926 Fast Track designation underscores the urgency to address a significant unmet treatment need for Duchenne muscular dystrophy, said Brenda Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer Global Product Development. We are working to advance our planned phase 3 program as quickly as possible.<\/p>\n
The FDAs Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to drugs that are expected to have an impact on factors such as survival and daily functioning.<\/p>\n
For more information visit pfizer.com.<\/p>\n
Pfizer receives FDA Fast Track designation for Duchenne muscular dystrophy investigational gene therapy. https:\/\/www.businesswire.com\/news\/home\/20201001005382\/en\/Pfizer-Receives-FDA-Fast-Track-Designation-for-Duchenne-Muscular-Dystrophy-Investigational-Gene-Therapy<\/a>. Accessed October 2, 2020.<\/p>\n <\/p>\n Read more:<\/p>\n Investigational Gene Therapy Fast Tracked for Duchenne Muscular Dystrophy - Monthly Prescribing Reference<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" The Food and Drug Administration (FDA) has granted Fast Track designation to the investigational gene therapy candidate, PF-06939926 (Pfizer), for the treatment of Duchenne muscular dystrophy (DMD). PF-06939926 is a recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of human dystrophin gene under the control of a human muscle-specific promoter. The Company has chosen the rAAV9 capsid due to its potential to target muscle tissue. Continue reading