Adzynma (adamts13, recombinant-krhn)
An approved treatment for Thrombotic Thrombocytopenic Purpura.
The same compound appears under different names depending on the context. Here is how to identify ADAMTS13, recombinant-krhn wherever you encounter it, plus the key facts at a glance.
- Generic name
- ADAMTS13, recombinant-krhn
- Brand name
- Adzynma
- Development code
- TAK-755
- Drug class
- Enzyme replacement therapy
- Manufacturer
- Takeda
- How it's taken
- An infusion by vein.
An enzyme replacement infusion, approved November 9, 2023, for adults and children with congenital TTP, given to prevent episodes or to treat one.
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Where ADAMTS13, recombinant-krhn fits
Enzyme replacement for congenital TTP, used instead of plasma infusions to prevent or treat episodes.
How ADAMTS13, recombinant-krhn works
People with congenital TTP inherit 2 changed copies of the ADAMTS13 gene and make too little of the enzyme that trims von Willebrand factor. Adzynma replaces the enzyme, so the sticky strands are cut down before they can trap platelets.
Mechanism: Lab-made copy of the ADAMTS13 enzyme that people with congenital TTP are missing
Side effects and safety
Adzynma has a boxed warning that some patients make antibodies that block the enzyme, with serious outcomes including death, so ADAMTS13 activity and antibodies are monitored; the FDA has investigated a death from these antibodies after treatment. In the main study of 48 patients, the most common side effects were headache (31.3%), diarrhea (16.7%), migraine (14.6%), stomach pain, nausea and upper respiratory infection (12.5% each), and dizziness and vomiting (10.4% each).
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 ADAMTS13, recombinant-krhn
An infusion by vein. To prevent episodes, 40 IU/kg every other week or weekly. To treat an episode, 40 IU/kg on day 1, 20 IU/kg on day 2 and then 15 IU/kg daily until 2 days after the episode ends.
Availability and cost
Only available as the brand-name product.
Help paying for Adzynma
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
Co-pay assistance of up to $20,000 a year for eligible patients with commercial insurance.
For: private insurance · source - Insurance and case manager help
Help with coverage, and help connecting uninsured patients to other programs. You do not have to enroll to get the medicine.
For: private insurance, Medicare, Medicaid, TRICARE, no insurance, underinsured · source
Access and eligibility
Approved for adults and children with congenital TTP. It is not approved for immune (acquired) TTP.
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
In a Phase 3 crossover study (NCT03393975, published in NEJM in 2024), no acute TTP events occurred among 37 patients aged 12 and older on Adzynma, against 1 among 38 on plasma-based therapy, and low-platelet events fell from 4.44 to 2.0 a year.
Development history
Developed by Takeda as TAK-755 and approved by the FDA on November 9, 2023. The FDA later added a boxed warning about neutralizing antibodies.
Explore Thrombotic Thrombocytopenic Purpura trials
Other Thrombotic Thrombocytopenic Purpura treatments
Common questions about ADAMTS13, recombinant-krhn
▸What is ADAMTS13, recombinant-krhn (Adzynma)?
An enzyme replacement infusion, approved November 9, 2023, for adults and children with congenital TTP, given to prevent episodes or to treat one.
▸How does ADAMTS13, recombinant-krhn work?
People with congenital TTP inherit 2 changed copies of the ADAMTS13 gene and make too little of the enzyme that trims von Willebrand factor. Adzynma replaces the enzyme, so the sticky strands are cut down before they can trap platelets.
▸What are the side effects of ADAMTS13, recombinant-krhn?
Adzynma has a boxed warning that some patients make antibodies that block the enzyme, with serious outcomes including death, so ADAMTS13 activity and antibodies are monitored; the FDA has investigated a death from these antibodies after treatment. In the main study of 48 patients, the most common side effects were headache (31.3%), diarrhea (16.7%), migraine (14.6%), stomach pain, nausea and upper respiratory infection (12.5% each), and dizziness and vomiting (10.4% each).
▸How is ADAMTS13, recombinant-krhn taken?
An infusion by vein. To prevent episodes, 40 IU/kg every other week or weekly. To treat an episode, 40 IU/kg on day 1, 20 IU/kg on day 2 and then 15 IU/kg daily until 2 days after the episode ends.
▸Is ADAMTS13, recombinant-krhn FDA approved?
Yes, ADAMTS13, recombinant-krhn (Adzynma) is FDA approved (2023) for the treatment of Thrombotic Thrombocytopenic Purpura.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- U.S. Food and Drug Administration. ADZYNMA. https://www.fda.gov/vaccines-blood-biologics/adzynma
- U.S. Food and Drug Administration. FDA investigating death due to neutralizing antibodies to ADAMTS13 following Adzynma treatment for congenital thrombotic thrombocytopenic purpura. https://www.fda.gov/vaccines-blood-biologics/safety-availability-biologics/fda-investigating-death-due-neutralizing-antibodies-adamts13-following-adzynma-treatment-congenital
- New England Journal of Medicine · 2024. Recombinant ADAMTS13 in congenital thrombotic thrombocytopenic purpura. https://pubmed.ncbi.nlm.nih.gov/38692292/