Approval decision on Yutrepia for PH-ILD expected in January

Note: This story was updated Jan. 30, 2024, to correct the approved indications of available treprostinil formulations. Amid ongoing litigation, the U.S. Food and Drug Administration (FDA) has agreed to review an application from Liquidia that seeks to extend the approval of Yutrepia — an inhaled dry powder…

CorVista Device to Diagnose PH Gets FDA Breakthrough Designation

The U.S. Food and Drug Administration (FDA) granted breakthrough device designation to the CorVista System, a point-of-care diagnostic tool that aims to use non-invasive tests and machine learning to help diagnose pulmonary hypertension and other cardiovascular disorders. The designation gives CorVista Health, the system’s developer, certain benefits aimed…

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.