The FDA clearance allows the company to start expanding its clinical investigation of PROHEMA into rare, non-malignant disorders.
The company intends to start enrollment of the Phase Ib PROVIDE trial (PROHEMA eValuation for the treatment of Inherited metabolic DisordErs) in pediatric patients with IMDs in the fourth quarter of 2014.
Fate Therapeutics president and chief executive officer Christian Weyer said for many severe inherited metabolic disorders, including those with central nervous system involvement, cellular enzyme replacement through unrelated donor cord blood transplantation has emerged as a potentially transformative therapeutic intervention.
"We believe pharmacologically-optimized HSC therapeutics, such as PROHEMA, hold significant promise for improving outcomes in patients with inherited metabolic and other rare genetic disorders," Weyer said.
"FDA clearance of the PROVIDE study marks an important step towards our goal of clinically evaluating this novel treatment paradigm in the non-malignant disease setting."
About 12 patients with various forms of IMDs, between the ages of one and 18 will be enrolled in the PROVIDE trial at up to three US pediatric HSC transplant centers.
According to the company, the trial’s inclusion criteria allows for the enrollment of patients with different types of lysosomal and peroxisomal storage diseases such as Hurler and Hunter syndromes, Krabbe disease and various other leukodystrophies, among others.
Primary endpoint of the PROVIDE trial is safety as evaluated by neutrophil engraftment.