GBT440 is an oral, once-daily drug being developed to specifically inhibit hemoglobin polymerization and the consequent sickling of red blood cells, the hallmark of SCD. This new therapy has the potential to increase hemoglobin’s affinity for oxygen, thereby restoring normal red blood cell function and oxygen distribution.
“Access to the EMA’s PRIME program is important not just for GBT, but for the entire SCD community, as this is the first eligibility granted to PRIME for a SCD therapy,” Dr. Ted W. Love, GBT’s president and CEO, said in a press release. “It provides further recognition from a regulatory authority that SCD is a severely unmet medical need that requires attention.”
Love added that the decision offers “external validation of the potential of GBT440 to address this need and bring a major therapeutic advantage to patients,” and that San Francisco-based GBT looks forward to working with the EMA “to further accelerate the development of GBT440 for patients in Europe.”
Acceptance into the PRIME program followed early positive results of ongoing Phase 1 and 2 clinical trials (NCT02285088 and NCT03041909). Both are evaluating the safety, tolerability and drug distribution and activity in adult healthy volunteers and SCD patients. Preclinical data on the drug’s mechanism of action provided further scientific support for potentially significant improvements in hemolysis and hemoglobin function.
The U.S. Food and Drug Administration (FDA) has granted Fast Track and Orphan Drug designations to GBT440 to treat SCD. In addition, the European Commission (EC) has designated orphan medicinal product status to GBT440 for this disease.
To further demonstrate GBT440 safety and capacity to modify SCD course, the drug is now undergoing Phase 2 HOPE-KIDS 1 Study (NCT02850406) in adolescents with SCD, and the pivotal Phase 3 HOPE trial (NCT03036813) in children with SCD aged 12 or older. Both studies are now recruiting patients.