Shares of Global Blood were jumping over 10% this week on news that the company has secured FDA approval for a new pill to treat sickle cell disease.
Sickle cell disease is a group of disorders that affects hemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body.
It was on Monday that the FDA approved the once a day pill to treat the disease. It will be sold under the name brand Oxbryta. The approval was three months earlier than had been expected.
Oxbryta works by making oxygen attach more tightly to hemoglobin. With hemoglobin in a highly oxygenated state, the mutation that causes sickle cell disease is covered up. This aids the red blood cells to keep a normal shape and prevents them from clumping together inside blood vessels.
“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. This therapy provides a new treatment option for patients with this serious and life-threatening condition,” said Dr. Richard Pazdur, the FDA’s top official in charge of reviewing drugs for blood-related disease and cancer.
This is the first time that the FDA has approved a drug to treat sickle cell disease based solely on data showing an increase in hemoglobin.
“Today is a major milestone not only for Global Blood, but, most importantly, for people living with sickle cell disease, their families and those who care for them,” Global Blood’s chief executive, Ted Love stated.
Disclaimer: We have no position in Global Blood Therapeutics Inc. (NASDAQ: GBT) and have not been compensated for this article.