Cuvrior Gets FDA Approval for Wilson Disease

The Food and Drug Administration (FDA) has approved Cuvrior (trientine tetrahydrochloride) for the treatment of adults with stable Wilson disease who are de-coppered and tolerant to penicillamine.

Cuvrior contains trientine, a copper chelator that eliminates absorbed copper from the body by forming a stable complex that is then eliminated through urinary excretion. Trientine also chelates copper in the intestinal tract, reducing copper absorption.

The approval was based on data from the randomized, open-label phase 3 CHELATE study (ClinicalTrials.gov Identifier: NCT03539952), which compared the efficacy and safety of Cuvrior with penicillamine in 53 adults with Wilson disease. Prior to study entry, all patients had been receiving penicillamine for at least 1 year and were adequately controlled and tolerating penicillamine.


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At the start of the study, patients entered a 12-week baseline period and continued to receive their established total daily dosage of penicillamine for 12 weeks. At week 12, patients were randomly assigned to either remain on penicillamine (n=27) or to switch to Cuvrior (n=26) for the 24-week post randomization period. The primary endpoint was the mean serum non-ceruloplasmin copper (NCC) level at 24 weeks post randomization (week 36).

Results showed that at week 36, patients treated with Cuvrior had a similar mean NCC level to those treated with penicillamine, 56mcg/L vs 46mcg/L, respectively (difference, -9; 95% CI, -24, 6). Mean 24 -hour urinary copper excretion was observed to be lower in patients receiving Cuvrior compared with penicillamine (274mcg/24hrs vs 511mcg/24hrs [difference, 236mcg/24hrs; 95% CI, 111-361]).

The most common adverse reactions reported were abdominal pain, change of bowel habits, rash, alopecia, and mood swings.

Cuvrior is supplied as a functionally scored tablet containing 300mg of trientine tetrahydrochloride. The product is expected to be available in early 2023.

References

  1. Orphalan announces FDA approval of Cuvrior™ for the treatment of adult patients with stable Wilson’s disease who are de-coppered and tolerant to penicillamine. News release. Orphalan. Accessed May 2, 2022. https://www.globenewswire.com/news- release/2022/05/02/2433290/0/en/Orphalan-announces-FDA-approval-of-Cuvrior-for-the-treatment-of-adult-patients-with-stable-Wilson-s-disease-who- are-de-coppered-and-tolerant-to-penicillamine.html
  2. Cuvrior. Package insert. Orphalan; 2022. Accessed May 2, 2022. https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/215760s000lbl.pdf

This article originally appeared on MPR

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