#I have altered the deal full

There are only four sickle cell drugs cleared in the US, including hydroxyurea and Global Blood’s Oxbyrta, which stops blood cells from changing shape. Some patients undergo regular transfusions to minimize the proportion of sickled cells.

Treatment options remain scant for the blood disorder. A limited number of patients are eligible to receive a transplant of a sibling’s healthy bone marrow, where blood cells are made, that can cure them.

Cystic fibrosis affects about 100,000 in developed countries compared to about 150,000 sickle cell patients in the US and Europe, said Cantor Fitzgerald analyst Li Watsek, and there are millions more people with sickle cell in sub-Saharan Africa, India, the Middle East and the Mediterranean. Between 20, the US government spent less than one-third as much money on sickle cell as it did on cystic fibrosis, a rare disease that most often afflicts White people, according to a 2020 study published in JAMA Network Open, and cystic fibrosis advocacy groups spent 75 times as much as sickle cell groups over the same period. “A lot of companies said, ‘Huh, there’s a whole open field here where nothing works we should find something that works,’” said Julie Kanter, director of the adult sickle cell clinic at the University of Alabama at Birmingham.īut the fact remains that historical investment in sickle cell disease has been comparatively low. Before its purchase of Global Blood, Pfizer was pursuing its own drugs for sickle cell but one failed and the other wasn't any better than Global Blood's Oxbryta. The late bloom of therapies is in part due to scientists gaining a better understanding of the disease and new technologies to tackle it, said Seng Cheng, chief scientific officer of rare diseases at Pfizer. Some of the emerging treatments use cutting-edge science like Crispr, a tool for editing genes. Today, roughly 30 drugs are in human trials with the same number in the early phases of research, according to a Bloomberg analysis of SpringerNature data. In about a decade, sickle cell has gone from an overlooked affliction to one with so many drugs in development it’s hard to keep track. Just a few years ago, the only drug was hydroxyurea, a cancer therapy that also prevents blood cells from deforming.

#I have altered the deal full

The drug giant’s purchase of Global Blood Therapeutics Inc. is the latest lap in a race to take on the inherited disorder in which a genetic mutation causes red blood cells to contort into crescents instead of full moons, preventing them from functioning properly. Global Blood makes Oxbryta, one of the few drugs approved to treat the disease, and is developing others. Finally big drugmakers are taking notice, culminating with Pfizer Inc.’s $5.4 billion acquisition of one of the more promising drugs to treat sickle cell disease. (Bloomberg) - It’s been more than seventy years since a renowned scientist first described the cause of a painful, sometimes lethal disorder that in the US afflicts mainly Black people.