The US Food and Drug Administration approved Novartis
) as new medicine that can help reduce extremely painful sickle cell disease
flare-ups for patients 16 and older. The monthly infusion, which halves occurrences of sickle cell pain episodes, will carry a list price of roughly US$82,000 to US$114,000 per year, depending on dosing.
Unfortunately, sickle cell disease
is one of the most common inherited blood disorders, affecting about 118,000 Americans, most of them black, and about 276 million people worldwide.
Its typical hallmark is periodic episodes in which red blood cells stick together, blocking blood from reaching organs and small blood vessels. That causes intense pain and cumulative organ damage that shortens the lives of people with the disease.
Dr. Biree Andemariam, chief medical officer of the Sickle Cell Disease
Association of America told Thailand Medical
News, "The duration and severity of these pain crises worsens with aging. Often patients die during one of these crises."
Dr Andemariam, a former Novartis
advisory board member, said the drug appears to work better the longer patients receive it.
, the swiss drugmaker is continuing patient testing to determine whether Adakveo
, also known as crizanlizumab
, lengthens patients' lives, said Ameet Mallik, the company's head of U.S. oncology
and blood disorders. He said severe pain episodes send U.S. patients to emergency departments about 200,000 times per year. About 85% are hospitalized for days to a week, running up big bills.
The disease’s debilitating condition also causes anemia, delayed growth, vision damage and painful swelling in hands and feet, making it hard for some people to maintain jobs or attend school.
At present conventional treatments include a 21-year-old cancer drug called hydroxyurea and Endari, approved in 2017. In patient studies, Endari reduced frequency of pain episodes about 25% and hydroxyurea reduced them by half. Hydroxyurea can have serious side effects and requires weekly blood tests. Both drugs have complicated dosing and don't work or stop working in some patients.
In a clinical study spanning one-year involving 198 patients, those getting the higher of two Adakveo
doses averaged 1.6 pain episodes over that year and 36% had none. A comparison group on placebo averaged three pain episodes that year and 17% had none. Adakveo
's side effects included influenza and high fever.
One patient, Danielle Jamison, of Islandton, South Carolina, has suffered with sickle cell disease
pain episodes since shortly after birth. The 35-year-old previously had a half-dozen pain crises requiring hospital trips each year. Those lessened by about half when she began taking hydroxyurea nine years ago. She hasn't been in the hospital since she started taking crizanlizumab
rong> two years ago as part of a patient study. She still has mild daily pain, but she said she can now take care of her home and drive her 9-year-old daughter to activities. "It's made a huge difference in how much I'm able to do," Jamison said.
The three drugs work through different mechanisms, so doctors may switch patients to Adakveo or to add it to their current treatment, said Andemariam, head of University of Connecticut's sickle cell disease treatment and research program.
Patients wanting to go on the new drug should consult their physicians before making any changes.