The Food and Drug Administration has granted Orphan Drug designation to AER-901 (inhaled imatinib) for the treatment of pulmonary arterial hypertension.

AER-901 is an investigational drug-device combination therapy that consists of a nebulized formulation of imatinib, a tyrosine kinase inhibitor, and the Fox® device, a breath-actuated smart nebulizer. The investigational product is expected to improve drug uptake and dosing consistency by delivering imatinib directly into the lungs and small airways while avoiding significant adverse events associated with oral imatinib.

The Company is currently investigating AER-901 in healthy adult volunteers in an ongoing phase 1 trial ( Identifier: NCT04903730), which has a completion target date for the end of 2021. “We are pleased with the progress we are making in our phase 1 trial and believe that AER- 901, which is targeted to enter phase 2/3 trials in the first half of 2022, might, for the first time, provide the opportunity to modify the course of this terrible disease, and offer an important therapeutic option for patients,” said Steve Thornton, chief executive officer of Aerami.

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Imatinib is currently available as an oral formulation to treat certain types of cancers. The oral formulation has been shown to improve pulmonary hemodynamics and physical exercise capacity in patients with pulmonary arterial hypertension; however, due to significant adverse reactions, the indication was not pursued. 

“By delivering Aerami’s proprietary inhaled imatinib, directly to the site of the disease, we believe AER-901 has the potential to significantly reduce the dose necessary to achieve therapeutic benefit thereby avoiding the adverse events seen with oral imatinib,” said Timm Crowder, president of Aerami. 


Aerami Therapeutics announces Orphan Drug designation for imatinib for the treatment of pulmonary arterial hypertension. News release. Aerami Therapeutics, Inc. Accessed August 25, 2021.

This article originally appeared on MPR