Katherine Kroner


SILVER SPRING, MD -- Friday, the U.S. Food and Drug Administration (FDA) approved United Therapeutics' oral treprostinil, an extended-release tablet commercially known as Orenitram, for the treatment of pulmonary arterial hypertension (PAH). Orenitram is the first orally administered prostacyclin analogue approved by the FDA for any disease.

"PHA is always pleased as new treatments become available. They increase options for patient treatment, both individually and in combination with others," says PHA President Rino Aldrighetti. "This is the twelfth treatment for PAH, the third new therapy to be FDA approved in the last two months. During the past 10 years, the average number of drugs approved per year for all rare diseases is fewer than thirty. To have this kind of advancement for PAH is extraordinary and speaks to the medical and research structure that has been built in this field."

Most Effective Use of Treatments Requires Early Diagnosis

While huge strides have been made in the advancement of therapeutic options, the time it takes to reach a correct PH diagnosis has not changed over the last 20 years. Many patients will see three or more different physicians over a three year period before they are properly diagnosed with PH, and almost three-fourths have advanced PH by the time they are diagnosed.

“Misdiagnosis and delayed diagnosis have an impact on patient survival and quality of life,” explains PHA Senior Director of Volunteer Services Debbie Castro. "Because PHA sees early diagnosis as a huge priority, we are working with medical professionals and patients to improve diagnosis through our early diagnosis campaign, Sometimes it’s PH."

More Needs to be Done for Children

While there are 12 treatments for pulmonary arterial hypertension (PAH), none are specifically approved for pediatric patients. PHA has launched the only pediatric PH research program in the United States, the Robyn Barst Pediatric Research and Mentoring Fund.

And, says Rino, “Ultimately, our goal is a cure. With increasing numbers of researchers engaged in more studies every year, we are hopeful and encouraged as we see that hope turning into action.”

Learn more: Read article | PH treatments in the U.S. | PH treatments worldwide | Sometimes it’s PH website | Robyn Barst Pediatric Research and Mentoring Fund

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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.