Monopar Therapeutics said the FDA granted rare pediatric disease designation to ALXN1840, its late-stage Wilson disease candidate, a regulatory step that could position the company for a pediatric priority review voucher if the drug wins NDA approval.
The designation matters because Wilson disease affects children and adults, and Monopar is now closer to a potential voucher that could be used for another application or sold. The company did not disclose any financial terms tied to the designation.
ALXN1840 is already in late-stage development. Monopar said the drug met the primary endpoint in its phase 3 pivotal trial, showing rapid and sustained copper mobilization over 48 weeks that was significantly greater than standard of care in both previously treated and untreated patients.
The company also highlighted the size of its safety database: 645 patient-years of follow-up across 266 patients. ALXN1840 is an oral, once-daily tablet.
Wilson disease itself is rare, affecting about 1 in 30,000 people worldwide, and can lead to toxic copper buildup in the liver, brain and other organs if untreated. As a result of these announcements, the company's shares have moved 2.24% on the market, and are now trading at a price of $96.68. For the full picture, make sure to review Monopar Therapeutics's 8-K report.
