Imatinib Received US FDA Orphan Drug Designation

Release Date: 24-Sep-2021

US FDA has granted Aerami Therapeutics orphan drug designation for Imatinib for the treatment of pulmonary arterial hypertension (PAH). Pulmonary Arterial Hypertension (PAH) is affecting almost 68,000 patients worldwide with limited treatment options.


Oral imatinib is a tyrosine kinase inhibitor used to cure numerous types of cancers which has been formerly investigated in a Phase 3 clinical trial, IMPRES, as an oral formulation for the treatment of PAH, given its potential to decrease the tightening and stiffness of pulmonary arteries. Oral imatinib proved clinically meaningful and statistically significant improvement in pulmonary hemodynamics and physical exercise capacity but the indication was not followed because of substantial contrary events believed to be a result of systemic toxicity associated with the high oral doses used in the study. Currently, dosing of patients in Phase 1 trial is going on and also, plans to enter Phase 2/3 Trials in early 2022.


The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S and it also gives several benefits, like tax credits for qualified clinical trials, potential seven years of market exclusivity after approval and exemption from user fees.

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