USA. On Friday, regulators approved a new drug that can help reduce the 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 appearance of episodes of sickle cell pain, will cost about $ 85,000 to $ 113,000 a year, depending on the dosage. Insured patients will usually pay less.
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Its distinguishing feature is the periodic episodes in which red blood cells clump together, blocking blood from reaching the organs and small blood vessels. This causes severe pain and cumulative organ damage, which shortens the lives of people with the disease.
'The duration and severity of these pain crises deteriorates with aging. Often patients die during one of these crises, "says Dr. Biri Andemarari, Chief Medical Officer of the Association for sickle cell disease of America.
Andemariam, Former Member to the Novartis Advisory Board, said the drug seemed to work better the longer the patients took it.
The Swiss drugmaker continues to test patients to determine if Adakweo, also known as crizlanizumab, prolongs patients' lives, said Ameet M lick, head of the American Cancer and Blood Company
He said episodes of severe pain send American patients to emergency rooms about 200,000 times a year, about 85% hospitalized for days to a week, receiving large bills.
The debilitating condition also causes anemia, stunted growth, visual impairment and painful swelling of the hands and feet, making it difficult for some people to maintain or attend
Current treatments include a 21-year cancer medicine called x idroxyurea and Endari approved in 2017
In patient studies, Endari reduced the incidence of pain episodes by about 25% and halved their hydroxyurea. Hydroxyurea can have serious side effects and requires weekly blood tests. Both drugs have complex dosing and do not – or stop – in some patients.
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In a one-year study of 198 patients, these receiving the higher than two doses of Adakveo averaged 1.6 episodes of pain this year, and 36 % had none. The placebo comparison group averaged three episodes of pain this year, and 17% had none. Adakweo side effects include flu and fever.
Daniel Jamison, of Island, South Carolina, has been suffering from episodes of sickle cell pain shortly after birth. The 35-year-old previously had half a dozen pain crises requiring hospital travel each year. These decreased by about half when she started taking hydroxyurea nine years ago.
She has not been to the hospital since she began taking crisisalizumab 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 take her 9-year-old daughter to work.
"It made a huge difference in how much I was able to do,"
All three drugs work through different mechanisms so that doctors can switch patients to Adakweo or add it to their current treatment, says Andemarari, head of the sickle treatment and sickle research program at the University of Connecticut.
In the meantime, there are numerous medicines for the treatment of sickle cell disease and gene therapies to cure eventually.