U.S. flag An official website of the United States government
  1. Home
  2. Drugs
  3. Development & Approval Process | Drugs
  4. Drug Approvals and Databases
  5. Resources for Information on Approved Drugs
  6. FDA approves crizanlizumab-tmca for sickle cell disease
  1. Resources for Information on Approved Drugs

FDA approves crizanlizumab-tmca for sickle cell disease

On November 15, 2019, Food and Drug Administration approved crizanlizumab-tmca (ADAKVEO, Novartis) to reduce the frequency of vaso-occlusive crises (VOCs) in adults and pediatric patients aged 16 years and older with sickle cell disease.

Efficacy was evaluated in 198 patients with sickle cell disease in SUSTAIN (NCT01895361), a 52-week, randomized, multicenter, placebo-controlled, double-blind trial. Patients were randomized (1:1:1) to crizanlizumab-tmca 5 mg/kg (N = 67), crizanlizumab-tmca 2.5 mg/kg (N = 66), or placebo (N = 65) administered intravenously over 30 minutes on week 0, 2, and every 4 weeks thereafter. Randomization was stratified by prior hydroxyurea (Y/N) and by the number of VOCs in the prior 12 months.

The primary efficacy outcome measure was the annual rate of VOCs leading to a healthcare visit, defined as an acute episode of pain with no cause other than a vaso-occlusive event requiring a medical facility visit and oral or parenteral opioids, or parenteral NSAIDs. Patients receiving crizanlizumab-tmca, 5 mg/kg, had a lower median annual rate of VOC compared to those receiving placebo (1.63 vs. 2.98, p=0.010). Reductions in the frequency of VOCs were observed among patients regardless of sickle cell disease genotype and/or hydroxyurea use. Thirty-six percent of patients treated with crizanlizumab-tmca 5 mg/kg did not experience a VOC compared to 17% in the placebo arm. The median time to first VOC from randomization was 4.1 vs. 1.4 months in the crizanlizumab-tmca 5mg/kg and placebo arm, respectively.

The most common adverse reactions (>10%) in patients receiving crizanlizumab-tmca were nausea, arthralgia, back pain, and pyrexia.

The recommended dose is 5 mg/kg intravenously over a period of 30 minutes on week 0, 2, and every 4 weeks thereafter.

View full prescribing information for ADAKVEO.

FDA granted this application priority review and orphan product and Breakthrough Therapy designations. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.

Follow the Oncology Center of Excellence on Twitter @FDAOncology.