August 7th, 2013

FDA Panel Recommends Approval of New Pulmonary Hypertension Drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee recommended approval for Bayer’s new pulmonary hypertension drug, riociguat. The committee voted 11-0 in favor of approving the drug for two forms of pulmonary hypertension: pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTPH).

Bayer proposed an initial dose of 1 mg three times daily and a maximum dose of 2.5 mg three times daily. Due to concerns about hypotension, FDA reviewers initially recommended that the drug not be approved at the 2.5-mg dose and instead recommended a starting dose of 0.5 mg. Ultimately, the panel decided to grant as many options as possible to physicians and recommended a starting dose of 0.5 mg and a maximum dose of 2.5 mg.

The panel meeting was largely devoted to two clinical trials, CHEST-1 and PATENT-1, whose findings were recently published in the New England Journal of Medicine. Both trials showed that patients taking riociguat had a modest improvement in their ability to walk.

CardioExchange’s John Ryan, an expert on pulmonary hypertension, sent the following comment:

The issue will be where PAH specialists feel this agent fits in their therapeutic options. It remains to be seen whether riociguat will become first line therapy and replace oral medicines that PAH doctors are familiar with, namely PDE5 Inhibitors and endothelin receptor antagonists, or if it will be additive on top of these agents. In PATENT-1, 50% of the patients were not taking other agents, so how they interact with each other is largely unknown.





4 Responses to “FDA Panel Recommends Approval of New Pulmonary Hypertension Drug”

  1. Judith Andersen, AB, MD says:

    Could not agree more with John Ryan: this is really tricky territory, and medication adherence, whether research or baseline, very complicated. A new drug, in this case, a very promising agent, riociguat (who came up with this name?. Clinical judgment, rare as it may seem, characterizes many good cardiology practitioners, refusing to take on a new drug or a heap-load of old ones to jeopardize an importunate patien– this is an agent that needs more data. And its population, a better — and more nuanced — outpatient treatment module/.

  2. Judith Andersen, AB, MD says:

    Sorry for the typos — all else is mine. JCA

  3. Judith Andersen, AB, MD says:

    New drug, new data, new side effects — why are we suspicious? There are enormous numbers of people who need new strategies. No major toxicity was documented in the clinical trial, apart from that to be expected from the participants age and comorbidities.

    I am not expert at this — but this drug appears impressive. I would ask more erudite and informed practitioners with experience with these drugs to comment on the Phase I or II clinical trial results.

    Thank you,

    Judith Andersen, MD

  4. I wonder about the group of pulmonary hypertension drugs – they are so expensive – and the data on outcomes seems to focus on modest gains in exercise tolerance (how much did it mean to the functioning of patients) – and I am not clear that they improve survival. And I wonder if we are using them in patients who are pre-pulmonary hypertension. I think it is time for some scrutiny of the practices in this field. Just given what is at stake – the cost and consequences – seems we might want to strengthen the evidence base. And I wonder how Bayer will price this drug.