WILMINGTON, DE — Inhibikase Therapeutics Inc. (Nasdaq: IKT) has received U.S. Food and Drug Administration Orphan Drug Designation for its lead pulmonary arterial hypertension drug candidate, a regulatory milestone that could provide tax incentives and market exclusivity if the treatment is ultimately approved.
The designation covers IKT-001, an investigational prodrug of imatinib mesylate being developed to treat pulmonary arterial hypertension, a rare, progressive disease affecting an estimated 50,000 people in the United States, according to the company.
The FDA’s Office of Orphan Products Development granted the designation based on the active ingredient, imatinib, rather than the specific formulation of IKT-001.
Orphan Drug Designation is available for therapies targeting diseases affecting fewer than 200,000 people in the United States. The designation provides potential development incentives, including tax credits for qualified clinical trial costs, exemptions from certain FDA user fees and the possibility of seven years of market exclusivity if the drug receives regulatory approval.
“The grant of Orphan Drug Designation for IKT-001 by FDA is another important milestone for Inhibikase and reflects the high unmet medical need” among patients with pulmonary arterial hypertension, Chief Executive Officer Mark Iwicki stated.
The company also cited preclinical data presented earlier this year at the American Thoracic Society International Conference, where IKT-001 demonstrated improvements in pulmonary vascular and hemodynamic markers associated with pulmonary arterial hypertension, along with a lower potential for gastrointestinal toxicity than imatinib mesylate.
IKT-001 remains an investigational therapy and has not been approved by the FDA for the treatment of pulmonary arterial hypertension.
Support the local news that supports Chester County. MyChesCo delivers reliable, fact-based reporting and essential community resources—free for everyone. If you value that, click here to become a patron today.