Monopar Initiated Rolling NDA for ALXN1840

Monopar Therapeutics began its rolling FDA submission for ALXN1840 to treat Wilson disease on July 22, 2026.

Updated on Sept. 23, 2026 in Diseases — General

Bold flat-color editorial illustration depicting a single medical vial, representing the pharmaceutical regulatory submission process for Wilson disease. — Monopar Therapeutics has initiated the rolling FDA submission for ALXN1840, a potential new treatment for Wilson disease. AI Illustration. Upload story photo >

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Monopar Therapeutics started a rolling New Drug Application (NDA) submission to the FDA on July 22, 2026, for its Wilson disease treatment, ALXN1840. The agency authorized the rolling process, which allows the company to submit completed sections while finalizing others.

Why it matters

Wilson disease is a genetic condition caused by ATP7B gene mutations that prevents the body from excreting copper, causing it to accumulate to toxic levels. ALXN1840 functions by mobilizing and sequestering excess copper, potentially becoming the first therapy with this mechanism of action approved in decades.

In a Phase 3 trial, ALXN1840 met its primary endpoint over a 48-week period. The treatment program included 645 patient-years of follow-up among 266 total participants.

The players

Monopar Therapeutics

A biopharmaceutical company based in Wilmette, Illinois, focused on developing treatments for patients with cancer and other serious diseases.

FDA

The United States Food and Drug Administration is the federal agency responsible for protecting public health by regulating the safety and efficacy of medical products.

The details

Monopar Therapeutics is advancing ALXN1840 through the regulatory process following its June 2026 Rare Pediatric Disease designation from the FDA. The drug also maintains Fast Track and Orphan Drug status, which may allow for a pediatric Priority Review Voucher upon successful approval.

June 2026: ALXN1840 received Rare Pediatric Disease designation.
July 22, 2026: Monopar initiated the rolling NDA submission.

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The Big Picture

The FDA Rare Pediatric Disease designation program provides a critical pathway for developers to earn Priority Review Vouchers when bringing niche treatments to market. Monopar is utilizing this framework to incentivize the development of ALXN1840 for a population affected by Wilson disease.

For patients living with Wilson disease, this application represents the movement toward a potential new treatment option that could help manage toxic copper accumulation. If approved, it may offer a novel mechanism of action compared to existing long-term management therapies.

The takeaway

The initiation of this rolling application marks a significant regulatory milestone in the effort to provide new therapeutic options for those with rare genetic copper metabolism disorders. Patients should consult with their healthcare providers regarding the current standard of care while monitoring the drug's progress toward final approval.