Clinical Hold Lifted on Sickle Cell Drug

FDA has lifted a clinical hold against a Fulcrum Therapeutics IND for FTX-6058 for the potential treatment of sickle-cell disease (SCD). The hold was ordered 2/23 based on preclinical data previously submitted in 4/2022, 10/2022 and 12/2022, Fulcrum says, adding that non-clinical and clinical evidence of hematological malignancies observed with other inhibitors of polycomb repressive complex 2 was also an FDA concern.

“Based on the initial data from the Phase 1b trial, which showed increasing levels of HbF [fetal hemoglobin] with each dose escalation, we believe in the potential of FTX-6058 to not only shift the current standard of care but importantly, offer these patients a differentiated oral option,” the company says. Earlier data in SCD demonstrated that HbF increases were associated with potential overall patient benefit, it adds.

FDA has granted FTX-6058 a fast track designation and an orphan drug designation for treating SCD.