NVS Novartis AG

By Peter Loftus 

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., 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.

Doctors say the new drugs have the potential to ease the complications of sickle cell disease, which afflicts about 100,000 Americans.

More new treatments are on the horizon. Drugmakers including Bluebird Bio Inc., Vertex Pharmaceuticals Inc. and Crispr Therapeutics Inc., along with government researchers, are developing experimental therapies that may have potential to cure or provide long periods of relief.

"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 and a sickle cell specialist at UConn Health, in Farmington, Conn.

But the therapies are expensive, part of a growing trend of high prices for drugs that treat rare diseases. Global Blood Therapeutics priced Oxbryta at $125,000 a year, though the company said the drug would cost most health plans $96,000 a year after discounts. Novartis's drug lists for as much as $113,100 a year, depending on the weight of the patient.

Drugmakers say the treatments can help cut other costs, such as hospitalization.

Sickle cell disease is caused by an inherited mutation in an oxygen-carrying protein known as hemoglobin. Decades ago, most children diagnosed with it didn't live into adulthood. Life expectancy has risen significantly since the 1980s, due to newborn screening and treatment advances like blood transfusions and penicillin to prevent severe infections.

But most patients don't live longer than 50 years, doctors say, and many face debilitating complications including strokes and kidney failure. Few drugs have been approved in recent decades.

Global Blood Therapeutics, of South San Francisco, Calif., designed Oxbryta to stop a molecular process that results in sickle-shaped red blood cells, the feature that gives the disease its name.

After 24 weeks of treatment, about 51% of the clinical-trial subjects who had received a high dose of the drug had higher levels of hemoglobin, the protein that carries oxygen in the blood, compared with 33% among those getting a lower dose and 7% in the placebo group. Those taking the drug also experienced reduced anemia.

"We think what this drug is doing is fundamental in terms of reversing disease," said Global Blood Therapeutics Chief Executive Ted Love.

Reported side effects include headaches and diarrhea.

The FDA approved the drug's use in patients aged 12 years and older.

"Our scientific investments have brought us to a point where we have many more tools available in the battle against sickle cell disease, which presents daily challenges for those living with it," said Acting FDA Commissioner Adm. Brett P. Giroir said.

Patricia Rose, 52, of Berwyn, Ill., said she took Oxbryta in a clinical trial along with another drug called hydroxyurea she has been taking since around 2000, and the combination helped alleviate the painful episodes resulting from the disease more than the older drug alone.

Novartis's Adakveo, infused intravenously once a month, was approved in patients age 16 and older to reduce the frequency of a painful complication of sickle cell that occurs when blood circulation becomes obstructed. The pain often sends patients to the hospital.

In a nearly 200-patient study, people taking Adakveo had a median of 1.63 such episodes a year, versus 2.98 in the placebo group.

Bluebird Bio is developing a gene therapy that would deliver functional copies of the faulty gene that causes sickle cell disease and restore a patient's ability to make healthy red-blood cells. The therapy is in mid-stage patient studies, and the company expects to file for regulatory approval in 2022, a spokeswoman said.

Meantime, Crispr Therapeutics and partner Vertex Pharmaceuticals are working on a treatment that would edit genes taken from patients' blood samples, and then infuse them back into patients as part of a stem-cell transplant.

The National Institutes of Health said last month it plans to invest at least $100 million in the next four years toward finding affordable, gene-based cures for sickle cell disease and HIV. The Bill & Melinda Gates Foundation said it would invest an equal amount toward the goal.

"The hope is that sickle cell may be cured in some cases but in other cases managed as a chronic condition," said Dr. Lewis Hsu, a pediatric sickle-cell specialist at Children's Hospital University of Illinois.

Write to Peter Loftus at peter.loftus@wsj.com

(END) Dow Jones Newswires

November 25, 2019 17:28 ET (22:28 GMT)

Novartis (NYSE:NVS)
Historical Stock Chart
From Mar 2024 to Apr 2024

Novartis (NYSE:NVS)
Historical Stock Chart
From Apr 2023 to Apr 2024