The FDA granted Mustang Bio’s (NASDAQ:MBIO) MB-107 rare pediatric disease designation for the treatment of X-linked severe combined immunodeficiency.
Patients with X-linked severe combined immunodeficiency produce very few T-cells and natural killer cells. MB-107 is a lentiviral gene therapy that is currently being assessed in two Phase 1/2 clinical trials.
The company plans to initiate two Phase 2 trials of MB-107, one in newly diagnosed infants between two months and two years of age, and one in patients over the age of two years, in the fourth quarter of 2020.
“We anticipate that our pivotal clinical program will begin shortly, and we look forward to continuing to work efficiently with the FDA, in order to bring MB-107 to children suffering from this devastating disease as quickly and safely as possible,” Dr. Manuel Litchman, president and CEO of Mustang, said in a statement.
MB-107 received FDA regenerative medicine advanced therapy designation and European Medicines Agency advanced therapy medicinal product classification for X-linked severe combined immunodeficiency in August 2019 and April 2020, respectively.
