[FDA News Release, November 15, 2019] – FDA has approved Adakveo (crizanlizumab), previously known as SEG101, to reduce the frequency of vaso-occlusive crises (VOCs), or pain crises, in adult and pediatric patients aged 16 years and older with sickle cell 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.”
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. The disease occurs most often in African-Americans, where 1 out of every 365 babies born have the disease.
The FDA’s decision to approve Adakveo 5 mg/kg is based on results of the 52-week, randomized, placebo-controlled SUSTAIN trial, which showed that Adakveo significantly lowered the median annual rate of VOCs to 1.63 vs 2.98 compared to placebo (P=.010), which is equivalent to a 45% reduction. Reductions in the frequency of VOCs were observed among patients regardless of sickle cell disease genotype and/or hydroxyurea use.
“We know this drug can decrease the frequency of sickle cell pain crises in a significant and clinically meaningful way,” said Kenneth Ataga, MD, Director, Center for Sickle Cell Disease, University of Tennessee Health Science Center at Memphis, and Principal Investigator of the SUSTAIN trial. “The approval of crizanlizumab is an important advancement for people living with this very difficult condition.”
The most common adverse reactions among crizanlizumab-treated patients (incidence > 10%) were nausea (18%), arthralgia (18%), back pain (15%) and pyrexia (11%).
Novartis has priced Adakveo, which is dosed by weight, at a wholesale acquisition cost (WAC) of $2,357 per vial. Based on dosing recommendation, most patients would take between 3 and 4 vials per month. This translates to WACs of $7,000 to $9,500 per month. The medicine is expected to be available to patients in the coming weeks.
Sources:
FDA News Release, November 15, 2019
Novartis Media Release: New Novartis medicine Adakveo® (crizanlizumab) approved by FDA to reduce frequency of pain crises in individuals living with sickle cell disease
FDA-approved Prescribing Information for Adakveo available here: https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/761128s000lbl.pdf