Potential Sickle Cell Treatment Receives Designation From FDA

Patients suffering from sickle cell disease, SCD, might just be getting news they’ve been waiting to hear for a long time. Just this week, the U.S. Food and Drug Administration granted Global Blood Therapeutics, Incorporation (GBT) with the first breakthrough Therapy Designation for their continued development of treatment called Voxelotor, reports Rare DR.

This Voxelotor treatment, also known as GBT440, is a therapy for disease modification. It’s budding success has shown designation from European Medicines Agency as well as orphan drug designation two years ago from the FDA.

When a patient has sickle cell, their red blood cells harden into a c-shape and become sticky. From this, blood vessels have a tendencies to clot resulting in extreme pain for the patient, as well as high risk for strokes, acute chest syndrome as well as infection. Global is confident that their treatment can be preventative of the disease by blocking the polymerization of hemoglobin by maintaining a strong oxygenated hemoglobin state. Read more about sickle cell disease here.

While they still have a ways to go, having the support of FDA gives us all hope that Global will be able to successful development what they aim to achieve with voxelotor. FDA made its decision from data provided from four studies, looking specifically at safety data and projected efficiency.

Voxelotor will continue to be studied at the Hemoglobin Oxygen Affinity Modulation to Inhibit Polymerization study that focuses on patients older than 12-years-old with sickle cell. It also will look specifically at children ages 6-17 years-old, which is known as the Hope-Kids 1 Study. They continue to look deep into the treatment and evaluate it’s safety, side-effects, as well as it’s direct treatment impact on the individual being tested. If the treatment continues to be successful and eventually hit the market, it will be taken orally about once a day for patients.


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