Adakveo (Crizanlizumab-tmca) Injection

Adakveo (Crizanlizumab-tmca) is a monoclonal antibody used to reduce the frequency of vaso-occlusive crises (VOCs) or pain crises in patients with sickle cell disease (SCD). Sickle cell disease is a genetic condition in which red blood cells deform into a sickle shape, leading to blocked blood flow and causing pain, organ damage, and other serious complications. Adakveo is specifically designed to address these painful episodes by targeting the mechanisms that cause blood cells to clump together.

How It Works

Adakveo works by inhibiting P-selectin, a protein found on the surface of endothelial cells (cells that line the blood vessels) and platelets. In sickle cell disease, P-selectin plays a crucial role in promoting the adhesion of sickled red blood cells to the walls of blood vessels. This process can lead to blockages in the blood vessels, causing painful VOCs.

Mechanism of Action

1. Targeting P-Selectin:
   • Adakveo binds to P-selectin, which prevents it from interacting with red blood cells and platelets. This inhibition reduces the risk of blood cells sticking together and forming blockages in the vessels.
2. Preventing Vaso-Occlusion:
   • By blocking P-selectin activity, Adakveo helps reduce the frequency of vaso-occlusive crises, allowing better blood flow and reducing the pain associated with sickle cell crises.

Side Effects

Like all medications, Adakveo has the potential to cause side effects. While many patients tolerate the medication well, some may experience side effects ranging from mild to severe.

Common Side Effects

• Nausea:
  • Nausea is a common side effect, particularly after the injection.
• Fever:
  • Patients may develop a fever, especially after the initial doses.
• Joint Pain (Arthralgia):
  • Some individuals report joint pain after receiving Adakveo.
• Back Pain:
  • Pain in the back is another common side effect that may occur.
• Infusion-related Reactions:
  • Some patients may experience reactions such as headache, dizziness, or shortness of breath during or after the infusion.

Serious Side Effects

• Hypersensitivity Reactions:
  • Severe allergic reactions, including swelling, difficulty breathing, and rash, may occur and require immediate medical attention.
• Serious Infusion Reactions:
  • Though rare, severe reactions during the infusion may occur, including hypotension, shortness of breath, or anaphylaxis.

Indications

Adakveo is indicated for the treatment of:

• Sickle Cell Disease:
  • Adakveo is approved for the reduction of vaso-occlusive crises (VOC) in patients with sickle cell disease aged 16 years and older. VOCs are painful events caused by blocked blood flow in the vessels due to sickled red blood cells clumping together.

Contraindications

While Adakveo is generally well-tolerated, there are certain conditions where its use is contraindicated. Patients with the following conditions or situations should avoid Adakveo:

• Severe Hypersensitivity to Crizanlizumab or its Ingredients:
  • Individuals with known hypersensitivity to crizanlizumab-tmca or any of the excipients in the formulation should avoid using Adakveo, as it may cause severe allergic reactions.
• Pregnancy:
  • While there is no clear evidence of harm to unborn babies, pregnant women should use Adakveo with caution, as there is limited data on its effects during pregnancy.
• Breastfeeding:
  • It is not known whether crizanlizumab passes into breast milk, so breastfeeding mothers should use the drug with caution or consult their healthcare provider.

Pricing Information

The cost of Adakveo varies depending on location, availability, and insurance coverage. Below is a table that provides an approximate price range of Adakveo in different countries.

Country	Price (per vial)	Reference
United States	$2,500 – $3,500	GoodRx
United Kingdom	£2,000 – £2,700	NHS
Canada	CAD $3,200 – $4,000	Canada Drugs
Germany	€2,300 – €3,200	Apotheke
Australia	AUD $4,500 – $5,200	PBS

Top 5 Global Brands

Several global pharmaceutical companies are actively involved in developing and distributing treatments for blood-related conditions such as sickle cell disease. Below are the top five global brands in this domain:

1. Novartis:
   • Novartis is the manufacturer of Adakveo and a global leader in healthcare innovation, particularly in developing therapies for sickle cell disease and other hematological disorders.
2. Pfizer:
   • Pfizer is known for its robust portfolio in rare diseases, including blood disorders. Its expertise spans the development of both biologics and small-molecule treatments.
3. Roche:
   • Roche is recognized for its advanced biologics and diagnostic solutions for a wide range of conditions, including sickle cell disease and other genetic disorders.
4. GSK (GlaxoSmithKline):
   • GSK focuses on developing cutting-edge therapies for rare and genetic diseases, including blood-related conditions such as anemia and hemophilia.
5. Sanofi:
   • Sanofi’s extensive work in immunology, hematology, and rare diseases positions it as a key player in the global pharmaceutical landscape, particularly in the development of therapies for rare blood disorders.