Katherine Kroner

FDA Approves Tenth Drug for One Rare Disease Affecting Just 20-30,000 Patients: Pulmonary Hypertension Association Highlights Factors Contributing to Rapid Progress

Only a handful of the 7,000 rare diseases in the US have more than one treatment, and most have none. Why have science and medicine grown so much in pulmonary arterial hypertension, and how is the field addressing the next big challenge -- early diagnosis?

Silver Spring, MD - Oct. 10, 2013 -- The US Food and Drug Administration this week approved the tenth treatment, riociguat, for pulmonary hypertension (PH), a rare and life-threatening lung disease affecting roughly 20,000-30,000 patients, continuing the remarkable pace of recent progress that distinguishes PH from all but a handful of the 7,000 other rare diseases in this country. An eleventh PH drug will be considered by the FDA later this month. 

Only a few other conditions have as many treatments as PH has or more.  Most, in fact, have one or none at all. Whereas physicians treating PH once delivered terminal diagnoses, they now expect their patients to live with their condition. Many patients survive for years -- even 10-20 years or more -- with proper treatment.

The development of treatments has also solved one old problem while creating a new one -- physicians are now better able to treat this disease than they are to diagnose it. Addressing this new issue, PHA is running a new campaign, Sometimes it's PH,, to promote early diagnosis mainly through informing medical professionals.

Scientific Backstory

The roots of this progress stretch back to the 1970s when a handful of physician-researchers fascinated by the pulmonary vascular system each worked independently in different cities. In the '80s, this group first worked together on building the original PH registry in the U.S. through the National Heart, Lung and Blood Institute. From there, much collaboration and discovery emerged, gaining momentum, and creating a fast-growing segment of medicine treating this disease.

The physician-researchers in the vanguard of making PH treatable -- members of PHA's Scientific Leadership Council -- remain in the field, and the number of specialists and specialty care centers treating PAH has increased dramatically.  Long-term surviving patients now offer hope and support to newer patients, including young people now able to live long enough to attend college, hold jobs, get married and start families.

About the Pulmonary Hypertension Association

PHA, headquartered in Silver Spring, Md., is the country's leading organization connecting pulmonary hypertension patients, families, and medical professionals. Its mission is to find ways to prevent and cure pulmonary hypertension and provide hope for the community through support, education, research, advocacy and awareness. PHA provides free access to information on its website about pulmonary hypertension and facilitates over 240 support groups around the nation, helping many of the estimated 20,000 - 30,000 diagnosed patients in the U.S. To learn more about PHA, visit:


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The National Organization for Rare Disorders (NORD) awarded PHA the Abbey S. Meyers Leadership Award in 2012 for outstanding service to PHA members in advocacy, education and other key areas.