January 10, 2018
1 min read

FDA grants breakthrough therapy designation to voxelotor for sickle cell disease

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The FDA granted breakthrough therapy designation to voxelotor for the treatment of patients with sickle cell disease, according to a press release from the therapy’s manufacturer.

Voxelotor (GBT440, Global Blood Therapeutics) — a novel hemoglobin S polymerization inhibitor administered orally once daily — previously received fast track, orphan drug and rare pediatric disease designations. Voxelotor increases hemoglobin's affinity for oxygen, thus blocking polymerization and the resultant sickling of red blood cells.

The FDA based the breakthrough therapy designation on safety and efficacy data from the phase 3 HOPE study, a phase 1/phase 2 study and open-label extension in adults, and data from the ongoing phase 2 HOPE-KIDS 1 study in children aged 6 to 17 years. Researchers also assessed voxelotor — previously known as GBT440 — for adults with severe sickle cell disease not eligible for the HOPE trials as part of a compassionate access experience.

“The FDA’s decision to grant voxelotor the first breakthrough therapy designation for the treatment of sickle cell disease reflects a recognition of the promising efficacy and safety data we have collected to date for this investigational drug, as well as an acknowledgement of the overwhelming need for major advances over available therapies in the treatment of [sickle cell disease] patients,” Ted W. Love, president and CEO of Global Blood Therapeutics, said in the release. “This designation is another significant milestone for [Global Blood Therapeutics] as we work to expedite the development of voxelotor.”

The phase 3 HOPE study and the HOPE-KIDS 1 study are ongoing.