This article is more than 5 years old. Information may no longer be current.
FDA grants priority review to crizanlizumab for pain crises in sickle cell disease
Click Here to Manage Email Alerts
The FDA granted priority review to crizanlizumab for prevention of vaso-occlusive crises among patients with sickle cell disease.
Crizanlizumab (SEG101, Novartis) previously received breakthrough therapy designation for the same indication.
Crizanlizumab — a humanized anti-P-selectin monoclonal antibody administered via monthly infusion — binds to P-selectin on the surface of platelets and endothelium in the blood vessels.
The agent has been shown to inhibit interactions between red blood cells, sickled red blood cells, endothelial cells, platelets and leukocytes, preventing these cells from being able to bind to P-selectin, a key driver of the vaso-occlusive process.
Vaso-occlusive crises are extremely painful and can lead to serious life-threatening complications. They are the most common cause of hospital admissions and ED visits among individuals with sickle cell disease, contributing to more than $1.1 billion in medical costs each year in the United States.
“The FDA's decision to give crizanlizumab priority review reflects the impact that this medicine could have for the many thousands of U.S. [adults with sickle cell disease] who experience painful vaso-occlusive crises,” John Tsai, MD, head of global drug development and chief medical officer for Novartis, said in the release. “We are looking forward to the opportunity, if crizanlizumab is approved, to reimagine medicine in sickle cell disease for patients who live with this condition every day of their lives.”
The new drug application Novartis submitted for crizanlizumab is based on positive results of the phase 2 SUSTAIN trial, which compared crizanlizumab with placebo among patients with sickle cell disease.
Crizanlizumab reduced the median annual rate of vaso-occlusive crises that led to health care visits by 45.3% compared with placebo (1.63 vs. 2.98; P = .01). Researchers observed the benefit among patients regardless of whether they received hydroxyurea therapy.
Crizanlizumab appeared associated with significantly longer median time to first vaso-occlusive crises (4.07 months vs. 1.38 months; P < .001).
A higher percentage of crizanlizumab-treated patients did not experience any vaso-occlusive crises during treatment (35.8% vs. 16.9%; P = .01).
Investigators reported comparable incidence of treatment-emergent adverse events (86.4% vs. 88.7%) and serious adverse events (25.8% vs. 27.4%) in the crizanlizumab and placebo groups.
The most common adverse events reported among crizanlizumab-treated patients were back pain, nausea, pyrexia and arthralgia. The majority of these reactions were grade 1 or grade 2.
No crizanlizumab-treated patients discontinued therapy due to adverse events.