Monopar wins FDA rare pediatric disease tag for Wilson disease drug

Monopar Therapeutics Inc. (NASDAQ: MNPR) announced that the U.S. Food and Drug Administration has granted Rare Pediatric Disease designation to ALXN1840, the company's late-stage candidate for the treatment of Wilson disease.

The FDA grants Rare Pediatric Disease designation to therapies intended to treat serious or life-threatening diseases that primarily affect children from birth to 18 years of age. The designation makes Monopar eligible, upon potential NDA approval, to receive a pediatric Priority Review Voucher, which can be used to obtain priority review of a subsequent marketing application or sold or transferred to another sponsor. Priority review can reduce the FDA's target review time by several months.

ALXN1840, also known as tiomolibdate choline, is an oral, once-daily tablet. In a Phase 3 pivotal trial, the drug met its primary endpoint by demonstrating copper mobilization significantly greater than standard of care over 48 weeks in both previously treated and untreated patients. The trial included 266 patients with 645 patient-years of follow-up.

Wilson disease is a rare genetic disorder affecting approximately 1 in 30,000 people worldwide. It is caused by mutations in the ATP7B gene, which impairs the body's ability to excrete copper, leading to toxic copper accumulation in the liver, brain, and other organs.

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