Thursday, October 10, 2013

New Drug Approved for PAH and CTEPH Patients in U.S.

On Tuesday, the U.S. Food and Drug Administration (FDA) approved Bayer’s riociguat pill for the treatment of pulmonary arterial hypertension (PAH) and for chronic thromboembolic pulmonary hypertension (CTEPH).  Riociguat, commercially known as Adempas, will be used for CTEPH patients after pulmonary thromboendarterectomy (PTE) surgery or if surgery is not possible. This will be the first drug available for CTEPH patients and tenth for PAH.

PHA President Rino Aldrighetti says, “Treatment opportunities for patients are growing rapidly. Eighteen years ago there were no treatments for pulmonary arterial hypertension. Twelve years ago there was one. Today there are ten, with a possible eleventh later this year (Actelion’s macitentan). Of the 7,000 rare diseases, only three have as many or more treatments available. We are fortunate to have a strong and growing research community with great communication and cooperation. That has helped foster industry interest in exploring new options for patients.”

Better Prognosis for PH Patients

The strong progress in the field of pulmonary hypertension over the past two decades has led to a better prognosis for PH patients. 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 PH treatment.

Patients Not Receiving Treatments in a Timely Manner

“The problem we now face is getting these treatments to patients sooner. While we have made huge strides over the past 20 years, the time it takes to reach a correct PH diagnosis has not changed,” explains PHA Vice President for Community Engagement Mollie Katz. “With missed or late diagnosis, the disease continues to get worse, affecting patients’ survival and quality of life. 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. Because PHA sees early diagnosis as a huge priority, second only to a cure, we are working with medical professionals and patients to improve diagnosis through our early diagnosis campaign, Sometimes it’s PH

Not There Yet

While there are 10 treatments for pulmonary arterial hypertension (PAH) and now one for CTEPH, there are none 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: FDA decision | riociguat study findings | PH treatments in the U.S. | PH treatments worldwide | Sometimes it’s PH website


Anonymous said...

Is this a added treatment or is it a parallel treatment to a drug already used like Letaris ??

Anonymous said...

Great new treatment but this article leaves much to be desired regarding information about the drug how it used how administered, value to PHA patients, etc. The PHA site says nothing about this at all.

Anonymous said...

I agree with the comments above. I would most certainly like to know how it is taken ? is it on it's won or with other many and side effects etc.

Anonymous said...

I have been taking Adcirca, Letaris and now Tyvaso breathing treatments every 4 hours yet i still having trouble with my oxygen levels dropping, Will this new medicine work all alone or with others.

Pulmonary Hypertension Association said...

Our Scientific Leadership Council is working on a forthcoming treatment fact sheet. When completed the fact sheet will be available here:

Until then you can view the FDA press release for more details here:

Anonymous said...

Man, this site stinks. Sorry I surfed onto it just now. I will NOT waste my time coming back to it! Here's what upsets me about this posting on your blog. I am only into this 11 days since I got diagnosed with PH.

Here's what upset me, especially the life expectation being only 10-20 yrs, that's rubbish!!

The upsetting lines:

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 PH treatment.

Anonymous said...

"how is it taken?"
"what's the value?"
"Is this in combination or solo?"

Guess what Anonymous friends... these are all questions for your doctor.

This web post is more to break the news on the new drug. The best place for that information is the drug company's website.

ACTUALLY, it's better to get medical information from your doctor because she or he will know if it's right for you.

The person who was diagnosed 11 days ago -- don't fret. The statistic is for the AVERAGE expectancy. This doesn't mean that YOU are necessarily going to have 10-20 years left to live. There are many who are 30+ years and don't expect to die any time soon.

It's upsetting to read, yes, but everyone is different and no one has an expiration date

Pulmonary Hypertension Association said...

To the person diagnosed 11 days ago. We are sorry that you feel so frustrated and disappointed in the information you found on the blog. It can be frightening and confusing to be newly diagnosed with PH. While new medicines are becoming available all the time, 2 in the past month, we are seeing life expectancies grow and like the previous commenter shared, no one has an expiration date. We are available if you would like to connect with other patients... additionally we have resources to help you navigate the first days, weeks and months after diagnosis. We don't want anyone to feel alone in their diagnosis. Please know that we are a community committed to finding a cure.