Voxelotor, sold under the brand name Oxbryta, is a medication used for the treatment of sickle cell disease. Developed by Global Blood Therapeutics, voxelotor is the first hemoglobin oxygen-affinity modulator. Voxelotor has been shown to have disease-modifying potential by increasing hemoglobin levels and decreasing hemolysis indicators in sickle cell patients. It has a safe profile in sickle cell patients and healthy volunteers, without any dose-limiting toxicity.In November 2019, voxelotor received accelerated approval in the United States for the treatment of sickle cell disease (SCD) for those twelve years of age and older. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication. In December 2021, voxelotor received accelerated approval in the United States for the treatment of sickle cell disease (SCD) for those aged four to eleven years. Side effects
Common side effects include headache, diarrhea, abdominal pain, nausea, fatigue, rash and pyrexia (fever). History
Voxelotor was granted accelerated approval by the U.S. Food and Drug Administration (FDA) in November 2019. The FDA granted the application for voxelotor fast track designation and orphan drug designation.The approval of voxelotor was based on the results of a clinical trial with 274 participants with sickle cell disease.The FDA granted the approval of Oxbryta to Global Blood Therapeutics.
The facial skin may be dry, exfoliative (flaking) or erythematous. Cervical lymphadenopathy (enlarged lymph nodes in the neck). Facial palsy (weakness and altered sensation of the face).The enlargement of the tissues of the mouth, lips and face seen in OFG is painless. Melkersson–Rosenthal syndrome is where OFG occurs with fissured tongue and paralysis of the facial nerve. The cause of the facial paralysis is thought to be caused by the formation of granulomas in the facial nerve, which supplies the muscles of facial expression. Causes
The cause of the condition is unknown. The disease is characterized by non-caseating granulomatous inflammation. That is, the granulomas do not undergo the caseating ("cheese-like") necrosis typical of the granulomas of tuberculosis. There is disagreement as to whether OFG represents an early form of Crohn disease or sarcoidosis, or whether it is a distinct, but similar clinical entity. Crohn disease can affect any part of gastrointestinal tract, from mouth to anus. When it involves the mouth alone, some authors refer to this as "oral Crohn disease", distinguishing it from OFG, and others suggest that OFG is the same condition as Crohn disease when it presents in the oral cavity. OFG may represent a delayed hypersensitivity reaction, but the causative antigen(s) is not identified or varies form one individual to the next. Suspected sources of antigens include metals, e.g.