Adakveo (crizanlizumab)
An approved treatment for Sickle Cell Disease.
The same compound appears under different names depending on the context. Here is how to identify Crizanlizumab wherever you encounter it, plus the key facts at a glance.
- Generic name
- Crizanlizumab
- Brand name
- Adakveo
- Development code
- SEG101
- Drug class
- P-selectin inhibitor
- Manufacturer
- Novartis
- How it's taken
- Given as an intravenous infusion over 30 minutes.
An antibody treatment that prevents vaso-occlusive crises in sickle cell disease by blocking the sticky interactions between blood cells and blood vessel walls that trigger pain episodes.
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Where Crizanlizumab fits
Add-on therapy to reduce the frequency of vaso-occlusive pain crises in sickle cell disease. Can be used alongside hydroxyurea for patients who continue to have crises.
How Crizanlizumab works
In sickle cell disease, sickled red blood cells stick to the walls of blood vessels, triggering a cascade that blocks blood flow and causes severe pain crises. Crizanlizumab blocks a protein called P-selectin on the surface of blood vessel walls and platelets, preventing this sticky interaction and reducing the frequency of vaso-occlusive crises.
Mechanism: Monoclonal antibody that blocks P-selectin, preventing blood cells from sticking to vessel walls
Side effects and safety
Common side effects include headache, joint pain, nausea, back pain, tiredness, belly pain, fever, diarrhea, vomiting, and mouth or throat pain. Infusion-related reactions, most often pain, can happen during or within 24 hours of an infusion, mostly with the first 2 infusions, and some have needed a hospital stay. Adakveo can also make automated platelet counts read falsely low. The European authorization was revoked in August 2023 after the STAND trial did not confirm benefit.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Crizanlizumab
Given as an intravenous infusion over 30 minutes. Loading doses at weeks 0 and 2, then once every 4 weeks thereafter. Administered at infusion centers.
Availability and cost
Only available as the brand-name product.
Anti-P-selectin monoclonal antibody requiring monthly IV infusions. First biologic specifically approved to reduce vaso-occlusive crises in sickle cell disease.
Help paying for Adakveo
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
Universal Co-pay Program: eligible privately insured patients may pay as little as $0 per month; Novartis pays up to $15,000 per calendar year. Not for Medicare or Medicaid.
For: private insurance · source - Free medicine program
Novartis Patient Assistance Foundation provides Adakveo free to eligible patients who are uninsured or have government insurance and meet income limits.
For: no insurance, Medicare, Medicaid · source
Good to know: Co-pay program terms: Copay.NovartisOncology.com or 1-877-577-7756. Free drug is through the separate Novartis Patient Assistance Foundation (1-800-277-2254); income limits apply.
- From a charity · The Assistance FundSickle Cell Disease fundWaitlist
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
The foundation says: “WAITLIST — Accepting Waitlist Patients. TAF is currently accepting requests to join the enrollment waitlist for this program. Waitlists a…”
Clinical trial results
The SUSTAIN trial showed a 45% reduction in median annual vaso-occlusive crisis rate (1.63 vs 2.98 for placebo). However, the confirmatory STAND trial did not meet its primary endpoint, leading to EMA withdrawal in Europe. FDA approval remains in effect in the US.
Development history
Approved by FDA in November 2019 for people 16 and older. This was a regular approval, not an accelerated approval. The European Commission revoked the European authorization in August 2023 after the STAND trial did not confirm benefit. It remains FDA approved in the US, and the US label was updated in 2024 to include the STAND results.
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Common questions about Crizanlizumab
▸What is Crizanlizumab (Adakveo)?
An antibody treatment that prevents vaso-occlusive crises in sickle cell disease by blocking the sticky interactions between blood cells and blood vessel walls that trigger pain episodes.
▸How does Crizanlizumab work?
In sickle cell disease, sickled red blood cells stick to the walls of blood vessels, triggering a cascade that blocks blood flow and causes severe pain crises. Crizanlizumab blocks a protein called P-selectin on the surface of blood vessel walls and platelets, preventing this sticky interaction and reducing the frequency of vaso-occlusive crises.
▸What are the side effects of Crizanlizumab?
Common side effects include headache, joint pain, nausea, back pain, tiredness, belly pain, fever, diarrhea, vomiting, and mouth or throat pain. Infusion-related reactions, most often pain, can happen during or within 24 hours of an infusion, mostly with the first 2 infusions, and some have needed a hospital stay. Adakveo can also make automated platelet counts read falsely low. The European authorization was revoked in August 2023 after the STAND trial did not confirm benefit.
▸How is Crizanlizumab taken?
Given as an intravenous infusion over 30 minutes. Loading doses at weeks 0 and 2, then once every 4 weeks thereafter. Administered at infusion centers.
▸Is Crizanlizumab FDA approved?
Yes, Crizanlizumab (Adakveo) is FDA approved (2019) for the treatment of Sickle Cell Disease.
▸Why was crizanlizumab withdrawn in Europe but not the US?
The confirmatory STAND trial did not meet its primary endpoint, leading the European Medicines Agency to recommend revoking the European authorization in May 2023, which the European Commission did in August 2023. The FDA approval in the US was a regular approval, not an accelerated approval, and it remains in effect. In 2024 the FDA added the STAND results to the US label, which says efficacy was not established in that trial.
▸How does crizanlizumab work differently from hydroxyurea?
Hydroxyurea boosts fetal hemoglobin to prevent sickling at the molecular level, while crizanlizumab blocks P-selectin to prevent sickled cells from sticking to blood vessel walls. They target different steps in the disease process and can be used together.
▸Can crizanlizumab be used with hydroxyurea?
Yes. Crizanlizumab can be used as add-on therapy alongside hydroxyurea for patients who continue to have vaso-occlusive crises despite hydroxyurea treatment. The 2 drugs work through complementary mechanisms.
▸What did the SUSTAIN trial show?
The SUSTAIN trial demonstrated a 45% reduction in the median annual vaso-occlusive crisis rate (1.63 crises per year vs 2.98 for placebo). This led to FDA approval in November 2019.
▸How is crizanlizumab administered?
Crizanlizumab is given as an intravenous infusion over 30 minutes at an infusion center. Loading doses are given at weeks 0 and 2, followed by maintenance infusions once every 4 weeks.
▸What happened in the STAND confirmatory trial?
The STAND trial, designed to confirm the benefit seen in SUSTAIN, did not meet its primary endpoint of reducing vaso-occlusive crises. This result led to the loss of the European authorization in 2023. In the US, the FDA kept the approval and added the STAND results to the label in 2024.