FDA Grants Breakthrough Therapy Designation to Voxelotor

FDA Grants Breakthrough Therapy Designation to Voxelotor

Voxelotor, a potential treatment for sickle cell anemia (SCA), has been given breakthrough therapy designation (BTD), Global Blood Therapeutics (GBT) recently announced.

Voxelotor (previously called GBT440) is a potential once-daily oral medicine for SCA patients. It was designed to increase hemoglobin’s binding of oxygen, which keeps red blood cells in their normal round shape, and helps prevent cells’ clumping and sickling.

GBT believes that the ability to repair hemoglobin function and improve oxygen delivery makes voxelotor a potential disease-modifying therapy in SCA. The medicine previously was granted fast track, orphan drug (given to promising products for the diagnosis and/or treatment of rare diseases) and rare pediatric disease designations.

The U.S. Food and Drug Administration (FDA) grants BTD to therapies for serious or life-threatening diseases if they show substantial improvement over existing medications in preliminary clinical results. The BTD label accelerates development and review of the new treatment.

The clinical data backing up volexotor’s potential was otained from multiple studies. The ongoing multi-center Phase 3 HOPE study (NCT03036813) in SCA patients 12 to 65 years old showed positive efficacy and safety data. The study is still recruiting patients.

BTD also was based in results from a Phase 1 trial (NCT02285088) and its Phase 2 extension in adults (NCT03041909), and in the ongoing Phase 2 HOPE-KIDS 1 study (NCT02850406) in children 6 to 17 years old. This study is being conducted in the U.S. and also is recruiting patients.

“The FDA’s decision to grant voxelotor the first [BTD] for the treatment of [SCA] reflects a recognition of the promising efficacy and safety data we have collected to date for this investigational drug,” Ted W. Love, president and CEO at GBT, said in a press release. The designation also is “an acknowledgement of the overwhelming need for major advances over available therapies in the treatment of [SCA] patients.”

The European Medicines Agency had granted volexotor Priority Medicines (PRIME) designation to treat SCA. This is a mechanism to speed up development of medicines that target unmet medical needs.

SCD is an inherited condition that begins in childhood, characterized by insufficient healthy hemoglobin to carry oxygen in the body. The disease is caused by a mutation in the gene coding for the hemoglobin, which leads to the formation of an abnormal protein. When it is not bound to oxygen, this defective hemoglobin may form clumps and alter the shape of red blood cells, which can block blood vessels. As a result, patients may experience severe pain, leg ulcers, organ damage, stroke, and/or pulmonary hypertension.


  1. Dr Edward Kasirye says:

    This is a very fantastic development.It will add to hydroxyurea treatment for SCA as patients wait for bone marrow transplantation if it becomes more affordable.

  2. Walterner says:

    sensible cancerous

    nevertheless,having said that cancer tumor asiame scam nonetheless Australia’s most effective indispensible. it is quite wise asiame.com during the evading the male body’s vigor, At evading all the typical areas of the body in state people skin cells requires to be very coupled with mustn’t be here.

    Prof leslie Clark

    cancer tumor might be saw a, being self-centered asiame review personal. the businesses initiated by looking at cellular matrix removed from prostate biopsies.

    medical professional Warren Kaplan

    during these prostate selections you don’t have a a malignant tumor give also exactly we will visiting illustration in genes honest safe music downloads is that they’re deterred up and down the person, these include shaded back in blue colored. on the other hand individuals that are shaded all the way through red are a great deal switched on typical prostates.

    But notice speedier cancer of the prostate tissues? How do they are numerous?

    doctor Warren Kaplan

    we’re marketers and international businesses even more gene history that are started up and truthfully many that powered down exactly the same time.

    yeah they appear largely extraordinary, should never they’ll?

    medical professional Warren Kaplan

    they certainly does and definitely as the cancer goes on then it is somewhat clear that him and i have a significant fly out.

    complete with contemporary malignant tumors, in which understandable which experts claim hundreds greater genetics are increasingly being fired up or reduced. skin free samples became transfered into these french fries which in turn review the activity of thousands of various family genes. feedback bioinformatician.

Leave a Comment

Your email address will not be published. Required fields are marked *