FDA OKs Crizanlizumab, First Targeted Therapy for Sickle Cell Crisis

Megan Brooks

November 15, 2019

The US Food and Drug Administration (FDA) has approved crizanlizumab (Adakveo, Novartis) to reduce the frequency of vaso-occlusive crisis in patients aged 16 years or older with sickle cell disease.

"Vaso-occlusive crisis can be extremely painful and is a frequent reason for emergency department visits and hospitalization for patients with sickle cell disease," Richard Pazdur, MD, 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, said in a statement.

"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," Pazdur said. 

According to the Centers for Disease Control and Prevention, sickle cell disease affects roughly 100,000 Americans. The disease occurs most often in African Americans, among whom one out of every 365 babies born has the disease.

The FDA approved crizanlizumab based on results of a randomized, placebo-controlled trial involving 198 patients with sickle cell disease and a history of vaso-occlusive crisis.

In the trial, infusions of crizanlizumab significantly lowered (by 45%) the median annual rate of vaso-occlusive crisis to 1.63 vs 2.98 compared with placebo. Reductions in the frequency of vaso-occlusive crisis were observed among patients regardless of sickle cell disease genotype and/or hydroxyurea use, Novartis said in a news release.

More than one third (36%) of patients who received crizanlizumab did not experience vaso-occlusive crisis during the study compared with 17% of placebo-treated patients. Crizanlizumab delayed the time that patients first experienced vaso-occlusive crisis after starting treatment from a median of 1.4 months to 4.1 months.

Novaritis expects crizanlizumab to be available to patients in the coming weeks.

"Patients with sickle cell disease often face unique challenges, and have long suffered silently through unimaginable pain crises," Beverley Francis-Gibson, president and chief executive officer of the Sickle Cell Disease Association of America, said in the Novartis release. "We are excited to have a new medicine that may help many of the thousands of people living with sickle cell disease by reducing the frequency of these potentially dangerous and painful episodes."

Common side effects with crizanlizumab are back pain (15%), nausea (18%), pyrexia (11%), and arthralgia (18%).

Patients should be monitored for infusion-related reactions and crizanlizumab discontinued in cases of severe reactions, the FDA said. Patients should also be monitored for interference with automated platelet counts or platelet clumping. Full prescribing information is available online.

Crizanlizumab was an orphan drug and had priority review and breakthrough designation.

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