Author Archives: Marisa Wexler MS

LIQ861, Inhaled Treprostinil for PAH, Back Up for FDA Approval

An updated application has been filed with the U.S. Food and Drug Administration (FDA), again asking that LIQ861 be approved as an inhalation treatment of pulmonary arterial hypertension (PAH), its developer Liquidia announced. The company first made this request in an application to the FDA submitted in April 2020. But the…

Potential PAH Therapy Identified by Cellular Screen of Thousands

By screening thousands of potential candidates, researchers have identified a compound, called AG1296, that may be useful for the treatment of pulmonary arterial hypertension (PAH). Their findings were published in the journal Science Translational Medicine, in a study, “iPSC–endothelial cell phenotypic drug screening and in silico…

Fungus Used in Chinese Medicine Eased PAH Damage in Rat Model

Treatment with a fungus called Ophiocordyceps sinensis eased disease signs and improved survival in a rat model of pulmonary arterial hypertension (PAH), suggesting a potential to treat patients. The findings were in the study “Substantial involvement of TRPM7 inhibition in the therapeutic effect of Ophiocordyceps sinensis on…

Pemziviptadil Shows Promise in Small Clinical Trial

PhaseBio Pharmaceuticals‘s investigational therapy pemziviptadil (PB1046) showed a good safety profile and clinically meaningful effects on physical function in a small clinical trial of three people with pulmonary arterial hypertension (PAH). The findings support further evaluation of pemziviptadil as…

Sotatercept Receives Orphan Drug Designation in Europe

The European Commission has granted orphan drug designation to sotatercept for the treatment of people with pulmonary arterial hypertension (PAH). This designation is given to medications with the potential to substantially improve care for rare diseases — defined as those that affect fewer than five individuals per 10,000…