We continue to advance..

This blog was originally posted on August 8, two days after an FDA advisory panel voted their opinion on Riociguat.  It is now updated following the publication of an August 29 New England Journal of Medicine article  and September 2 presentation of new data on Macitentan at the European Society of Cardiology Conference in the Netherlands.  If approved, both drugs may offer additional treatment options for PAH (and Riociguat also for CTEPH). FDA decisions are expected by mid-October.


Tuesday, August 6 was a rare and important day...

A new drug for PAH and CTEPH was being reviewed by the Cardiovascular and Renal Drugs Advisory Committee of the FDA.  This is generally the final step before the FDA releases a decision on whether a drug is approved for release to market.

Thirteen years ago, I attended an FDA Advisory Committee meeting for the first time.  Actually, I attended two on two consecutive days.  The hearings were for what became the second and third drugs to be approved for PAH -  Tracleer and Remodulin.  It was an exciting two days.  The first treatment for PH - Flolan - had been approved in 1996 and five years later we were looking at a tripling of options for patients.

That was only the beginning.  Over the next decade, we would see another tripling...to nine treatments.  And, this week, we were looking at the strong possibility of a tenth.

Other changes, have taken place as well.  Unlike in those early hearings, three of the 11 panelists on the Committee - Drs. Stuart Rich, John Newman and Steven Kawut - are recognized experts i n the field.  Their contributions to the Committee conversation helped the entire panel understand the nuances and needs of this complex area of medicine.

During the public comments section of the meeting, I had the opportunity to read a letter that had been reviewed and approved by leadership of PHA's Scientific Leadership Council and our Board of Trustees.

While PHA NEVER directly recommends the approval of a drug - we believe it is the FDA's role to determine the safety and effectiveness of a treatment - we ALWAYS speak to the need of additional treatments for their individual and combination value to patients.

By 3:00pm following presentations by the new drug's sponsor (Bayer) and the FDA staff and many questions for both and much discussion, the Advisory Committee took their votes.  On the first, they recommended Riociguat's approval for treatment of pulmonary arterial hypertension. On the second they voted to recommend approval for Riociguat as the first treatment for Chronic Thromboembolic Pulmonary Hypertension (CTEPH) for patients who cannot undergo a pulmonary endarterectomy surgery to remove clots in their lungs, or for those who still have complications after having the procedure.

With nine treatments, PAH has as many or more treatments than all but two of the 7,000 rare diseases identified in the U.S.  Now, we will wait for the FDA to make the final decision on a tenth treatment by early October.


Here's a copy of the letter we sent to the FDA and delivered to the Advisory Committee:

August 2, 2013

Kristina A. Toliver, PharmD
Center for Drug Evalulation and Research
Food and Drug Administration
10903 New Hampshire Avenue
WO31-2417
Silver Spring, Maryland 20993-0002

Dear Dr. Toliver:

While there have been great advances in research and expanded treatment options for pulmonary arterial hypertension in recent years, I want to assure you that it is not enough.  Each week, we continue to send an average of 20 condolence cards to families of our members.  We continue to lose too many patients with this disease.  Different treatments work for different patients.

It is up to the FDA to judge the safety and efficacy of riociguat.  However, we at the Pulmonary Hypertension Association want you to know that, should this drug be deemed effective, the need is there for PAH where it will be a valuable addition in the arsenal of therapies. Additionally, for our patients with CTEPH who are not candidates for surgery or have post-operative pulmonary hypertension, we are excited that, if approved, riociguat will be a valuable therapeutic option. 

Sincerely,

Rino Aldrighetti
President and CEO