FDA Grants Orphan Drug Designation to Aerami Therapeutics’s Imatinib

Aerami Therapeutics—a clinical stage biopharmaceutical company developing inhaled therapies to treat severe respiratory and chronic diseases—announced in an Aug. 25, 2021 press release that FDA granted orphan drug designation for imatinib in AER-901 for the treatment of patients with pulmonary arterial hypertension (PAH). AER-901 is Aerami’s drug-device combination product candidate for inhaled imatinib for the treatment of PAH. It is currently in a Phase I trial that is estimated to be completed toward the end of 2021.

“Receiving orphan drug designation for imatinib in AER-901 is another important milestone to emerge from our PAH development program,” said Steve Thornton, CEO of Aerami, in a press release. “We are pleased with the progress we are making in our Phase I trial and believe that AER-901, which is targeted to enter Phase II/III 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.”

Although there aren’t many patients with PAH and the approved drugs don’t fundamentally modify or halt the progression of the disease, the global market for PAH treatments is an estimated $5.5 billion in worldwide sales in 2020, according to the company press release.