GBT Granted Approval for New Treatment for Sickle Cell Disease

 

Global Blood Therapeutics was recently granted priority review by the FDA for their new drug Oxbryta. This drug is meant to treat sickle cell disease, and it is the first of its kind. It is meant for adults and children who are twelve or older, and its unique characteristics have fostered a lot of attention. After the approval from the FDA, GBT saw their stock increase by 11%. Beyond the financial aspect, healthcare professionals and people who have sickle cell disease are very excited for the innovations that Oxybryta brings for treatment.

About Sickle Cell Disease

Sickle cell disease is a group of disorders, with the most common and well known form being sickle cell anemia. It affects the shape of red blood cells, changing them so that they resemble a sickle. These deformed blood cells get caught along the walls of blood vessels, which creates blockage and restricts the flow of blood. This malformation is caused by an issue with the protein hemoglobin. Hemoglobin is responsible for carrying oxygen throughout the body. It is a recessive condition, meaning both parents must pass down the gene. While it is a rare disease, it is more common within certain demographics, such as people of African descent and people of Hispanic descent.

Symptoms of this disease include pain crisis, swelling in the hands and feet, symptoms of anemia like fatigue, jaundice, and delayed growth. Children who have sickle cell disease often experience these symptoms only during pain crisis, whereas adults constantly feel the effects. Regardless of age, damage often occurs to the organs that experience a lack of blood flow due to blockages. Common organs that are damaged are the spleen, liver, brain, eyes, kidneys, lungs, heart, joints, skin, and bones.

Currently the only cure that exists for this disease is a bone marrow and blood transplant, but it is not a complete cure as not everybody with sickle cell disease qualifies for a transplant. Treatments are often symptomatic and are meant to prolong life.

About Oxbryta and GBT

Oxbryta is meant to inhibit sickle hemoglobin polymerization, which is the root of sickle cell disease. It is an oral treatment that is taken once a day, and it increases hemoglobin’s affinity for oxygen. It blocks polymerization, which is what makes the red blood cells deformed. Studies performed with this drug found that about half of participants, 51.1%, achieved an increase in hemoglobin, whereas only 6.5% of those taking the placebo saw this outcome. Oxbryta is expected to be available to patients within the next two weeks, and is listed at $125,000 annually.

Global Blood Therapeutics is a biopharmaceutical company whose goal is to find treatments and hope for “underserved patient communities.” It is currently developing two drugs meant to treat sickle cell disease. Along with Oxbryta, Voxelotor is in the late stages of development. Voxelotor is an oral drug as well, taken once a day for the treatment of the disease.

Find the original article here.


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