FDA Approves Novel Sickle Cell Disease Treatment Voxelotor
- par Laura Grandis
- dans Médecine
- — Nov 28, 2019
"Today's approval provides additional hope to the 100,000 people in the USA, and the more than 20 million globally, who live with this debilitating blood disorder", the FDA said in a statement.
Dr. Elliott Vichinsky, the director of hematology/oncology at UCSF Benioff Children's Hosptial, said that many sickle cell disease patients experience hemoglobin polymerization. We remain committed to raising the profile of this disease as a public health priority and to approving new therapies that are proven to be safe and effective.
The regulator had cleared Oxbryta (voxelotor) for the treatment of sickle cell disease in patients aged 12 and over, and according to the SCD Association of America is a therapy that promises to "change the course" of the disease.
"With Oxbryta, sickle cells are less likely to bind together and form the sickle shape, which can cause low hemoglobin levels due to red blood cell destruction", Pazdur said in a statement.
GBT's drug, known chemically as voxelotor, works by preventing red blood cells from sickling, a deformation that restricts flow of oxygen in blood vessels, leading to severe pain and organ damage.
The approval was based on the results of a double-blind, multicenter clinical trial that included 274 patients with SCD: 90 patients received voxelotor 1,500 mg, 92 received voxelotor 900 mg, and 92 received placebo.
Today is a major milestone not only for GBT but, most importantly, for people living with SCD, their families and those who care for them. Medical officials say the new drugs have the potential to ease the complications of sickle cell disease, which afflicts approximately 100,000 Americans. At the start of the study, two-thirds (65%) of patients were receiving hydroxyurea. In the case of Oxbryta, the randomized trial full result.
While that degree of improvement is associated with significantly decreased rates of multiorgan failure and death in natural history studies, and considered a "response", the FDA cautioned that further trials are needed to verify the drug's clinical benefit. The most common side effects reported in Oxbryta and placebo patients were headache (26% vs. 22%), diarrhea (20% vs. 10%), abdominal pain (19% vs. 13%), nausea (17% vs. 10%), fatigue (14% vs. 10%), rash (14% vs. 10%) and pyrexia or fever (12% vs. 7%).
The approval, granted under the FDA's Accelerated Approval pathway, proceeds the Global Blood Therapeutic drug's Fast Track and Orphan Drug designations. In a Global Blood Therapeutics press release on 25 November, he said that Oxbryta improves hemoglobin levels and reduces anemia and hemolysis. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
