Is Adzynma approved by the FDA?

Adzynma was approved by the U.S. Food and Drug Administration on November 9, 2023. 

What is Adzynma?

Adzynma (ADAMTS13, recombinant-krhn) is a human recombinant protein used to prevent or treat blood clots in adults and children with congenital thrombotic thrombocytopenic purpura (cTTP). This condition is a rare, chronic blood clotting disorder associated with low levels of the ADAMTS13 enzyme, leading to severe symptoms and high mortality rates without treatment.

How Does Adzynma Work?

Adzynma is a synthetic version of the ADAMTS13 protein, which works by replacing the missing or defective enzyme. In clinical studies, a single 40 IU/kg intravenous infusion of Adzynma significantly increased ADAMTS13 activity compared to traditional plasma-based treatments.

Usage and Administration

Adzynma is administered by a healthcare provider as a slow intravenous infusion at a rate of 2 to 4 mL per minute. The dose is calculated based on body weight and the actual potency of the drug printed on the vial.

Dosage Information

• Prophylactic Dosage: 40 IU/kg body weight every other week, adjustable to once weekly based on clinical response.
• On-Demand Dosage:
  • Day 1: 40 IU/kg body weight
  • Day 2: 20 IU/kg body weight
  • Day 3 and beyond: 15 IU/kg body weight until two days after the acute event is resolved.

Side Effects

Common side effects affecting more than 5% of patients include headache, diarrhea, migraine, abdominal pain, nausea, upper respiratory tract infection, dizziness, and vomiting. Serious side effects can include hypersensitivity reactions such as rash, hives, itching, throat tightness, chest pain, difficulty breathing, lightheadedness, dizziness, nausea, or fainting. It is crucial to inform your healthcare provider about any bothersome or persistent side effects.

Warnings and Precautions

• Hypersensitivity Reactions: Adzynma may cause severe allergic reactions, including anaphylaxis. It should not be used in individuals who have had life-threatening hypersensitivity reactions to the drug or its components.
• Inhibitor Formation: The body can form inhibitors to ADAMTS13, which may reduce the effectiveness of Adzynma. Regular monitoring with blood tests is recommended.
• Pregnancy and Breastfeeding: The effects of Adzynma on unborn babies and whether it passes into breast milk are unknown. Pregnant or breastfeeding women should consult their healthcare provider.

Storage

Adzynma should be stored in the refrigerator at 2°C to 8°C (36°F to 46°F) for up to 36 months from the date of manufacture. It can also be stored at room temperature (up to 30°C/86°F) for up to 6 months. Once stored at room temperature, it should not be returned to the refrigerator. Do not use Adzynma beyond the expiration date or if it has not been stored properly. The reconstituted product should be used immediately or within 3 hours when stored at room temperature.

Conclusion

Adzynma was FDA approved on November 9, 2023, as the first recombinant ADAMTS13 treatment for cTTP, addressing the root cause of this severe condition. This approval marks a significant advancement in the management and treatment of cTTP, providing a synthetic option to replace the missing or defective enzyme in affected individuals.

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