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FDA approves Global Blood Therapeutics’ drug targeting root cause of sickle cell disease

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The Food and Drug Administration has approved a new drug for treating sickle cell disease in adults and children.

The agency said Monday that it had given accelerated approval to South San Francisco, California-based Global Blood Therapeutics’ Oxbryta (voxelotor) for SCD in patients aged 12 and older. GBT noted that Oxbryta is the first drug to win FDA approval that targets the root cause of the disease, namely by inhibiting sickle hemoglobin polymerization. But it’s the second drug overall to be approved for SCD in less than two weeks, following the Nov. 15 approval of Novartis’ Adakveo (crizanlizumab), for reducing the frequency of pain crises in SCD patients.

Shares of GBT closed 6.8% higher on the Nasdaq Monday afternoon following the news. SCD is estimated to affect 100,000 people in the U.S., predominantly of African descent, according to the Centers for Disease Control and Prevention.

In a conference call with investors Monday, GBT Chief Commercial Officer David Johnson said the drug’s list price would be $10,417 per month, with 65 percent of payers paying a net price of about $8,000 per month after discounts – particularly those the company must provide to participate in Medicaid – and broad coverage expected by the end of 2020. Executives noted that about 50% of SCD patients are covered under Medicaid, while commercial insurers cover 30%, and 10-15% are covered under Medicare or dual-eligible, with a smaller percentage uninsured. The company plans to not increase the list price for three years and thereafter tie increases to inflation. It also plans to offer a patient support program, GBT Source, while financial assistance programs mean eligible patients won’t have to pay co-pays.

“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,” FDA Office of Oncologic Diseases acting Director Richard Pazdur said in a statement. “This therapy provides a new treatment option for patients with this serious and life-threatening condition.”

The basis of the accelerated approval is the Phase III HOPE study, which enrolled 274 patients who were randomized to receive Oxbryta at 1,500mg, 900mg or placebo. Results of the study, which were published in The New England Journal of Medicine in August, showed that 51% of patients in the 1,500mg group had a hemoglobin response, compared with 33% of those in the 900mg group and 7% in the placebo group.

The company plans to continue studying the drug in the HOPE-KIDS 2 study, a post-approval confirmatory trial that will use transcranial doppler flow velocity to show whether there is a decrease in stroke risk among children aged 2-15 and that the company plans to start by the end of the year.

Photo: Michail_Petrov-96, Getty Images

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