PT001 Receives Orphan Drug Designation To Treat Pulmonary Arterial Hypertension

Release Date: 03-Sep-2021

PulmoSIM Therapeutics receives Orphan Drug Designation from US FDA for PT001 for the treatment of Pulmonary Arterial Hypertension (PAH). PAH is a disturbing, progressive and life-threatening disorder characterized by increased pressure in the arteries that carry blood from the heart to the lungs. The increased pressure strains the heart that can limit physical activity and thus, resulting in heart failure and reduced life expectancy.


PAH affects 15-50 persons per million within the U.S and Europe, and the usual survival rate after diagnosis is less than 3 years. The approved therapies are unable to target the underlying condition, making PAH a ‘rare disease epidemic’ with no long-term treatment option. PT001 targets several responsible pathways in PAH, to provide a more efficacious curative treatment.


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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