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The United States approves new drug to treat sickle cell disease



U.S. regulators on Friday approved a new drug that can help reduce extremely painful sickle cell disease.

The Food and Drug Administration approved Novartis AG's Adakveo for patients 16 years and older. The monthly infusion, which halves the incidence of sickle cell pain episodes, will carry a list price of about $ 85,000 to $ 113,000 per year, depending on dosage. Insured patients generally pay less.

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Sickle cell disease is one of the most common hereditary blood diseases, affecting about 1

00,000 Americans, most of them black, and about 300 million people over worldwide.

Its characteristics are periodic episodes where red blood cells stick together, preventing blood from reaching organs and small blood vessels. It causes intense pain and cumulative organ damage that shortens the lives of people with the disease.

“The duration and severity of these pain crises worsen with aging. Often patients die during one of these crises, "says Dr. Biree Andemariam, medical director of the Sickle Cell Disease Association of America.

Novartis Adakveo (Novartis via AP)

Andemariam, a former Novartis advisory board member, said the drug is watching

The Swiss drugmaker continues patient testing to determine if Adakveo, also known as crizanlizumab, prolongs patients' lives, said Ameet Mallik, the company's chief of US oncology and blood disorders. [19659003] He said that severe pain episodes send US patients to emergency rooms around 200,000 times a year, with about 85% being hospitalized for days to a week, and running big bills.

The debilitating condition also causes anemia, delayed growth, visual impairment. and painful swelling of the hands and feet, making it difficult for some people to maintain a job or attend school.

Current Treatment r includes a 21 year old cancer drug called hydroxyurea and Endari, approved in 2017.

In patient studies, Endari reduced the incidence of pain episodes by about 25% and hydroxyurea reduced them by half. Hydroxyurea can have serious side effects and requires weekly blood tests. Both medications have complicated dosing and do not work – or stop working – in some patients.

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In a one-year study of 198 patients, having the highest of two Adakveo doses averaged 1.6 pain episodes during that year, and 36% had none. A placebo comparison group averaged three pain episodes that year and 17% had none. Adakveo's side effects included flu and high fever.

Danielle Jamison, of Islandton, South Carolina, has been suffering from sickle cell pain since shortly after birth. The 35-year-old previously had half a dozen pain crises that require hospital trips each year. They were reduced by about half when she started taking hydroxyurea nine years ago.

She has not been to the hospital since she started taking crizanlizumab two years ago as part of a patient study. She still has mild pain every day, but she said she can now take care of her home and drive her 9-year-old daughter to activities.

"It has made a big difference in how much I am able to do," Jamison said.

All three drugs work through different mechanisms, allowing doctors to switch patients to Adakveo or to add it to their current treatment, said Andemariam, head of the University of Connecticut's sickle cell treatment and research program.

Meanwhile, many medications are being tested to treat sickle cell disease and gene therapies to possibly cure it.


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