Recently, officials with the FDA granted orphan drug designation (ODD) to alemtuzumab (CLLS52; Cellectis), which is an investigational product used as part of the lymphodepletion regimen administered before UCART22, evaluated in the BALLI-01 clinical trial in relapsed/refractory B-cell acute lymphoblastic leukemia.
According to a statement from the company, the impact of adding alemtuzumab to the lymphodepletion regimen was demonstrated in Cellectis’ BALLI-01 study, in which the addition of this lymphodepletion agent to the fludarabine and cyclophosphamide regimen was associated with sustained lymphodepletion and significantly higher UCART22 cell expansion.
Cellectis is the inventor of the combination of CD52 knockout UCART cells with a lymphodepleting regimen containing an anti-CD52 antibody such as alemtuzumab.
The CD52 knockout aims to render the UCART product candidates resistant to alemtuzumab as part of the lymphodepleting regimen.
The FDA grants ODD status to medicines intended for the treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. Receiving ODD status may help to expedite and reduce the cost of development, approval, and commercialization of a therapeutic agent.1