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FDA grants rare pediatric disease designation to GBT440 for sickle cell disease
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The FDA granted rare pediatric disease designation to GBT440 for the treatment of sickle cell disease, according to the drug’s manufacturer.
GBT440 (Global Blood Therapeutics) — an oral, once-daily therapy for patients with sickle cell disease — increases hemoglobin’s affinity for oxygen. This, in turn, blocks polymerization and the sickling of red blood cells.
Three trials are evaluating GBT440 in pediatric patients with sickle cell disease, including:
- the phase 3 HOPE trial, which includes patients aged 12 years or older;
- the phase 1/phase 2 GBT440-001 trial; and
- the open-label, single- and multiple-dose HOPE-KIDS 1 trial, designed to evaluate the safety, tolerability, pharmacokinetics and exploratory treatment effect of GBT440 in pediatric patients aged 6 to 17 years old.
“The FDA’s rare pediatric disease designation for GBT440, in addition to the previously granted orphan drug and fast track designations, confirm the agency’s recognition that the sickle cell disease community faces a critical need for new treatments and further strengthens our GBT440 program,” Ted W. Love, MD, president and CEO of Global Blood Therapeutics, said in a company-issued press release. “We have made important progress in our pediatric program this year and look forward to sharing additional updates at the [ASH Annual Meeting and Exposition] in December.”