FDA approves new drug to prevent sickle cell pain, organ damage

National News

This undated image provided by Novartis shows bottles of Novartis’s Adakveo. U.S. regulators have approved the new sickle cell disease medicine that can prevent extremely painful, dangerous “crises” in which misshapen blood cells clump together, reducing blood and oxygen flow. (Novartis via AP)

(AP) — U.S. regulators on Friday approved a new medicine that can prevent some extremely painful sickle cell disease flare-ups.

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

Sickle cell disease is one of the most common inherited blood disorders, affecting about 100,000 Americans, most of them black, and about 300 million people worldwide.

Its 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.

“The duration and severity of these pain crises worsens with aging. Often patients die during one of these crises,” said Dr. Biree Andemariam, chief medical officer of the Sickle Cell Disease Association of America.

Andemariam, a former Novartis adviser, 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 oncology and blood disorder research.

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 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.

Current treatments include a 21-year-old cancer drug called hydroxyurea and Endari, approved in 2017.

In patient studies, Endari reduced the 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 one-year study of 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 percent had none. Adakveo’s side effects included influenza and high fever.

Danielle Jamison, of Islandton, South Carolina, has suffered with sickle cell 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 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.

All 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 the University of Connecticut’s sickle cell treatment and research program.

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

Copyright 2020 Nexstar Broadcasting, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.

Latest Stories

More Local News

Recent Videos

Amber's Friday Morning #OneMinuteForecast 7/3

Thumbnail for the video titled "Amber's Friday Morning #OneMinuteForecast 7/3"

Friday's forecast: Severe storm chances ramp up this evening

Thumbnail for the video titled "Friday's forecast: Severe storm chances ramp up this evening"

FURRY FRIENDS JULY 3

Thumbnail for the video titled "FURRY FRIENDS JULY 3"

NDC JULY 3

Thumbnail for the video titled "NDC JULY 3"

Babe Ruth Baseball

Thumbnail for the video titled "Babe Ruth Baseball"

Mandan Rodeo Days

Thumbnail for the video titled "Mandan Rodeo Days"

Northwoods League

Thumbnail for the video titled "Northwoods League"

Thursday, July 2nd, 2020 - KX Storm Team Nightly Forecast - Dave Holder

Thumbnail for the video titled "Thursday, July 2nd, 2020 - KX Storm Team Nightly Forecast - Dave Holder"

July 4 COVID Safety Reminders

Thumbnail for the video titled "July 4 COVID Safety Reminders"

DJGA Golf

Thumbnail for the video titled "DJGA Golf"

WWII Veteran

Thumbnail for the video titled "WWII Veteran"

Travel Plans

Thumbnail for the video titled "Travel Plans"

PPP Extension

Thumbnail for the video titled "PPP Extension"

Vision Zero

Thumbnail for the video titled "Vision Zero"

Update on Grape Experiments

Thumbnail for the video titled "Update on Grape Experiments"

Fraternal Order of Police

Thumbnail for the video titled "Fraternal Order of Police"

Police Complaints

Thumbnail for the video titled "Police Complaints"

Licenses for Kids

Thumbnail for the video titled "Licenses for Kids"

Summer Theatre on FB

Thumbnail for the video titled "Summer Theatre on FB"

LGBTQ+ Same Sex Marriage Vote to Come

Thumbnail for the video titled "LGBTQ+ Same Sex Marriage Vote to Come"
More Video

KX News Trending Stories

Don't Miss