New Sickle Cell Drug, ADAKVEO, Approved!

The U.S. Food and Drug Administration (FDA) approved Novartis’ Adakveo (crizanlizumab) to decrease the frequency of pain crises, in adults and children age 16 years and older with sickle cell disease. Novartis indicates it expects to make the therapy available in the coming weeks.

Sickle cell disease is a genetic condition that affects the body’s red blood cells. It occurs when a child receives two sickle cell genes—one from each parent. In someone living with this disease, the red blood cells become hard and sticky and look like a C-shaped farm tool called a “sickle”. This can lead to acute pain episodes called VOCs, in addition to life-threatening complications. Those life-threatening conditions include stroke, vision loss, blood clots in the lungs, and infections.

It is estimated that:

  • SCD affects approximately 100,000 Americans.
  • SCD occurs among about 1 out of every 365 Black or African-American births.
  • About 1 in 13 Black or African-American babies is born with sickle cell trait (SCT)

Adakveo is a monoclonal antibody that binds to P-selectin on the surface of the activated endothelium—the inside walls of blood vessels—and platelets. The approval was based on the results of the 52-week SUSTAIN trial. The trial showed that Adakveo lowered the median annual rate of VOCs to 1.63 compared to 2.98 for the placebo group, a reduction of 45%.

“The approval of Adakveo marks a new era in the treatment of sickle cell disease, a genetic condition that places an extraordinary burden of unpredictable pain crises on patients and their families,” said Susanne Schaffert, president of Novartis Oncology. “The stories we have heard from patients about their sickle cell pain crises are devastating. We are pleased to help reimagine medicine together with the sickle cell community and offer new hope for fewer VOCs.”

Sickle cell disease affects about 100,000 people in the U.S., according to the U.S. Centers for Disease Control and Prevention (CDC). A 2009 study published in the journal Pediatric Blood and Cancer noted that in 2005, sickle disease cost an average of $11,702 for children under Medicaid and $14,772 for patients with employer-sponsored insurance.

The projected cost of the drug is $2,357 per vial, which the expected dosage of three or four vials per month coming to $7,071 or $9,428 per month. It is prescribed based on weight. Payers are likely to be more than willing to pay it, because in addition to decreasing the pain crises, the drug decreased the median days a patient spent in the hospital each year by 42%.

ADAKVEO is used

  • in people 16 years of age and older who have sickle cell disease
  • to help reduce how often certain episodes (crises) happen

It is not known if ADAKVEO is safe and effective in children under 16 years of age.

IMPORTANT SAFETY INFORMATION

What is the most important information I should know about ADAKVEO?

ADAKVEO may cause serious side effects, including infusion reactions.

Infusion reactions. Infusion reactions may happen within 24 hours of receiving an infusion of ADAKVEO. Tell your health care provider right away if you get any of the following signs and symptoms of an infusion reaction:

  • fever
  • chills or shivering
  • nausea
  • vomiting
  • tiredness
  • dizziness
  • sweating
  • hives
  • itching
  • shortness of breath or wheezing

Your doctor or health care provider may monitor you for signs and symptoms of infusion reactions.

ADAKVEO may interfere with a blood test.

Tell your health care provider that you are receiving ADAKVEO before having any blood tests. ADAKVEO may interfere with a laboratory test to measure your platelet counts.

Before receiving ADAKVEO, tell your health care provider about all of your medical conditions, including if you are
are pregnant or plan to become pregnant. It is not known if ADAKVEO may harm your unborn baby
are breastfeeding or plan to breastfeed. It is not known if ADAKVEO passes into your breast milk. You and your health care provider should decide the best way to feed your baby during treatment with ADAKVEO
Tell your health care provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of ADAKVEO include:

  • nausea
  • back pain
  • joint pain
  • fever

These are not all of the possible side effects of ADAKVEO. For more information, ask your health care provider or pharmacist.

Call your doctor for medical advice about side effects.