FDA Approves New Sickle Cell Drug As Part Of Flood Of New Treatments For The Disease
“Everybody’s been waiting for this moment where the flood gate of new treatments is opening,” said Dr. Biree Andemariam, chief medical officer of the Sickle Cell Disease Association of America. But the new drug is expensive: Global Blood Therapeutics priced Oxbryta at $125,000 a year.
The Wall Street Journal:
New Drugs Emerge To Treat Sickle Cell Disease
The U.S. Food and Drug Administration approved a new drug for sickle cell disease Monday, adding to a new wave of treatments that promise relief from the life-threatening blood disorder that largely afflicts African-Americans. Oxbryta, a once-daily pill from Global Blood Therapeutics Inc., GBT 6.79% blocks a process in blood cells that can lead to anemia and organ damage, hallmarks of sickle cell disease. It is the second treatment to get FDA approval in recent weeks, after the agency approved Novartis AG ’s Adakveo to reduce the frequency of bouts of pain that sickle cell patients can suffer. (Loftus, 11/25)
Stat:
Global Blood Secures FDA Approval For New Pill To Treat Sickle Cell Disease
Oxbryta will cost $125,000 per year before discounts, Global Blood said. Oxbryta was approved based on results from a clinical trial that enrolled 274 patients with sickle cell disease and treated them with one of two doses of Oxbryta or a placebo. The results: 51% patients on the higher dose of Oxbryta achieved a hemoglobin response, defined as an increase in hemoglobin of at least one gram per deciliter after 24 weeks. Just over 6% of the placebo patients had the same hemoglobin response. The difference between the two groups was statistically significant. (Feuerstein, 11/25)
FiercePharma:
Global Blood Therapeutics Scores FDA Nod For Oxbryta, Forecasts 'Paradigm Shift' In Sickle Cell Disease
The nod comes months ahead of the drug’s February 2020 action date at the FDA. Oxbryta scored an FDA breakthrough therapy designation, a priority review and accelerated approval, indicating how the agency viewed the new option compared with existing treatments. In recent months, the FDA has approved meds that significantly advance patient care ahead of their decision dates. (Sagonowsky, 11/25)
