FDA Brief: Orphan Drug Designation for Two Novel Multiple Myeloma Treatments
Posted: Wednesday, August 25, 2021
The U.S. Food and Drug Administration (FDA) recently granted two Orphan Drug designations in the treatment of multiple myeloma: one for the AlloCAR T-therapy ALLO-715 and the other for a new class of small molecule called CFT7455, which reportedly destroys disease-causing proteins selectively through degradation.
The FDA’s decision for ALLO-715 was based on initial results from the phase I UNIVERSAL clinical study. The UNIVERSAL trial in relapsed or refractory multiple myeloma has ongoing enrollment to study ALLO-715 in combination with the gamma secretase inhibitor nirogacestat and consolidation therapy. The current trial data show that ALLO-715 may achieve clinical responses while eliminating the need for bridging therapy or delays in treatment associated with manufacturing. The next trial update will be in late 2021.
In June 2021, a phase I/II clinical trial began enrolling patients investigating the safety, tolerability, and antitumor activity of CFT7455. Secondary and exploratory objectives to characterize the pharmacokinetic and pharmacodynamic profiles of this novel small molecular are also being examined.