Global Blood Therapeutics scores blockbuster FDA nod for Oxbryta, forecasts 'paradigm shift' in sickle cell disease

Another day, another early FDA nod.

Global Blood Therapeutics has its first drug approval, and it’s one that’ll bring a “paradigm shift” in how patients with sickle cell disease are treated, execs say. Monday, the FDA approved Oxbryta, formerly known as voxelotor, to treat sickle cell disease patients aged 12 and older. 

A once-daily oral tablet, Oxbryta marks an advance for patients who suffer from the lifelong, inherited blood condition that eventually affects “virtually every organ” in patients’ bodies, GBT’s head of medical affairs Jonathan Sorof told FiercePharma. In the U.S., the disease mostly affects African Americans. The med will be available within two weeks and will bear a list price of $10,417 per month, or $125,000 per year, the company said.

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.

RELATED: GBT sickle cell trial hits endpoint ahead of NDA filing 

For years, doctors in the field have been focused on managing patients' symptoms, which include pain throughout the body, fatigue, delayed development and more, Sorof said. There’s been a “passionate dissatisfaction” with the status quo, but Oxbryta represents an advance because it can treat the root cause of the disease, he added.

GBT has already hired and deployed its commercial team, chief commercial officer David Johnson said, and now it’ll get to work educating doctors and payers about the new option. Over the first six months of next year, the company will work to get its contracts in place and secure Medicare and Medicaid reimbursement. The company hopes to have “broad coverage” by the middle to end of next year, he said.

The drug is pricier than Wall Street expected or on the higher end of pricing expectations, according to analysts. The med has a $2.2 billion opportunity in the U.S. alone, Cantor Fitzgerald's Alethia Young wrote. Still, analysts aren't expecting much in 2020 revenues. Young predicts $60 million in sales next year, while J.P. Morgan's Cory Kasimov expects $30 million.

Overall, Wall Street predicts $2.5 billion in peak global sales.

The FDA approved the med based on a trial of 274 patients with sickle cell disease. Ninety patients in the study received 1500 mg of the drug, while 92 received 900 mg and 92 received placebo. Investigators measured efficacy based on hemoglobin response rates, which came in at 51% for the 1500 mg drug group compared with 6.5% for the placebo group.

There are about 100,000 sickle cell disease patients in the U.S. and millions more throughout the world, GBT said. The disease primarily affects people whose ancestors are from sub-Saharan Africa, but it also affects several other ancestries.

RELATED: Novartis eyes $1B sales with FDA nod for targeted sickle cell disease drug Adakveo

The approval follows Novartis' Adakveo nod in the middle of November. That drug scored an FDA green light to prevent painful vaso-occlusive crises associated with sickle cell disease.