FDA approves first targeted therapy to treat patients with painful complication of sickle cell disease

FDAToday, the U.S. Food and Drug Administration approved Adakveo (crizanlizumab-tmca), a treatment to reduce the frequency of vaso-occlusive crisis - a common and painful complication of sickle cell disease that occurs when blood circulation is obstructed by sickled red blood cells - for patients age 16 years and older.

"Hope has never been higher for people living with sickle cell disease and their families and supporters, with a pipeline of new treatments on the horizon, like the one being approved today, and several initiatives underway to better utilize current tools in the battle against the painful and deadly blood disorder," said Acting FDA Commissioner Adm. Brett P. Giroir, M.D. "The opportunity before us in the coming months and years is profound and historic."

Sickle cell disease is an inherited blood disorder in which the red blood cells are abnormally shaped (in a crescent or "sickle" shape), which restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage. It is also characterized by severe chronic inflammation that results in vaso-occlusive crisis where patients experience episodes of extreme pain and organ damage. According to the Centers for Disease Control and Prevetion, sickle cell disease affects approximately 100,000 Americans. The disease occurs most often in African-Americans, where 1 out of every 365 babies born have the disease.

"Adakveo is the first targeted therapy approved for sickle cell disease, specifically inhibiting selectin, a substance that contributes to cells sticking together and leads to vaso-occlusive crisis," said Richard Pazdur, M.D., director of the FDA's Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA's Center for Drug Evaluation and Research. "Vaso-occlusive crisis can be extremely painful and is a frequent reason for emergency department visits and hospitalization for patients with sickle cell disease."

The Adakveo approval was based on the results of a randomized clinical trial enrolling 198 patients with sickle cell disease with a history of vaso-occlusive crisis. Patients either received Adakveo or a placebo. The patients treated with Adakveo experienced fewer health care visits for vaso-occlusive crisis annually (median annual rate of 1.63 visits), compared to patients who received a placebo (median annual rate of 2.98 visits). In addition, 36 percent of patients who received Adakveo did not experience vaso-occlusive crisis during the study, and it delayed the time that patients first experienced vaso-occlusive crisis after starting treatment from 1.4 months to 4.1 months.

Common side effects for patients taking Adakveo were back pain, nausea, pyrexia (fever) and arthralgia (joint pain). Health care professionals are advised to monitor patients for infusion-related reactions and to discontinue Adakveo for severe reactions. Patients who receive Adakveo should be monitored for interference with automated platelet counts or platelet clumping (platelet counts reported may be much lower than the actual count in the blood). Health care professionals are advised to run tests as soon as possible or use citrate tubes (a practice to avoid platelet activation).

The FDA granted this application Priority Review and Breakthrough Therapy designation, which expedites the development and review of drugs that are intended to treat a serious disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies. Adakveo also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The FDA granted approval of Adakveo to Novartis.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Most Popular Now

Favipiravir flu drug 'clearly effective' in treati…

According to the multiple news articles the drug favipiravir (sold under the brand name Avigan), developed by Fujifilm Toyama Chemical, had produced encouraging outcomes ...

Understanding how COVID-19 affects children vital …

Though COVID-19 so far appears to be largely sparing children, researchers are cautioning that it is critical to understand how the virus affects kids to model the pandem...

Roche initiates Phase III clinical trial of Actemr…

Roche (SIX: RO, ROG; OTCQX: RHHBY) is working with the Food & Drug Administration (FDA) to initiate a randomised, double-blind, placebo-controlled Phase III clinical tria...

Free EDC software for non-profit COVID-19 related …

Italy-based EDC provider, Nubilaria srl, offers its ACTide EDC pro-bono for European non-profit COVID-19 Coronavirus related studies. The platform is made available for t...

Novartis commits to donate up to 130 million doses…

Novartis announced its commitment to donate up to 130 million doses of generic hydroxychloroquine to support the global COVID-19 pandemic response. Hydroxychloroquine and...

AstraZeneca to donate 9 million face masks to supp…

AstraZeneca is donating nine million face masks to support healthcare workers around the world as they respond to the COVID-19 (novel coronavirus) global pandemic. AstraZ...

Vivli to launch a portal for sharing data from COV…

In a visible sign of data sharing leadership, Vivli, the Center for Clinical Research Data has committed to serving the open science community through the launch of a COV...

CAR macrophages go beyond T cells to fight solid t…

Chimeric antigen receptor (CAR) T cell therapy has been a game-changer for blood cancers but has faced challenges in targeting solid tumors. Now researchers from the Pere...

Singapore modelling study estimates impact of phys…

A new modelling study conducted in a simulated Singapore setting has estimated that a combined approach of physical distancing [2] interventions, comprising quarantine (f...

COVID-19: The immune system can fight back

Melbourne researchers have mapped immune responses from one of Australia's first novel coronavirus (COVID-19) patients, showing the body's ability to fight the virus and ...

New kind of CRISPR technology to target RNA, inclu…

CRISPR-based genetic screens have helped scientists identify genes that are key players in sickle-cell anemia, cancer immunotherapy, lung cancer metastasis, and many othe...

Roche response to COVID-19 pandemic

Roche Group (SIX: RO, ROG; OTCQX: RHHBY), provided an update on the various actions the company is taking to address the COVID-19 pandemic. On March 19, 2020, Roche co...