Uptravi is Approved!

Good NewsFDA has Approved Uptravi to Treat Pulmonary Arterial  Hypertension

We can add another drug to our arsenal of treatments for PAH.  On December 21st, the FDA approved Actelion’s newest medication called Uptravi (Selexipag).  This oral medication is a prostacyclin receptor agonist.  The efficacy of the medicine was established in the largest pulmonary arterial hypertension study ever conducted.  1156 patients were enrolled.

Uptravi reduced the rate of clinical worsening by 40% compared to placebo.  The medication is dosed starting at 200mcg twice daily and gradually increased to the highest tolerated dose or 1600mcg twice daily.  As with other PAH medications, side effects are common.  These include headache, diarrhea, nausea, jaw pain, leg and muscle pains, flushing and joint pains, all consistent with prostacyclin type activity.

Where Does Uptravi Fit into our Treatment Algorithm?

The most direct competitor for Uptravi is Orenitram (oral Treprostinil).  In comparison to Orenitram, Uptravi is dosed twice daily not three times daily (best results with Orenitram are with three times daily dosing, though it may be given twice daily).  Furthermore, the period of gradually increasing the medication dosage (titration schedule) is substantially shorter with Uptravi.  We do not yet know how much Uptravi will cost but we suspect it will be priced less than Orenitram.

In my practice, I anticipate using Uptravi as a third oral agent when patients are progressing despite treatment with an ERA and a PDE5 inhibitor.  Uptravi has not been studied in very sick patients and should not replace continuously infused Flolan or Remodulin in our sickest patients.  It may well be a more convenient alternative to Tyvaso (inhaled Treprostinil).

Important Questions About Uptravi

As Uptravi comes to market in January 2016, a few important issues remain to be addressed.  First, we do not yet have a clear understanding how to transition patients from Uptravi to pump-based therapy if they are unable to take pills.  Actelion is working on some guidance for the PAH community.  Second, perhaps rather than starting two medications initially in newly diagnosed PAH patients, three treatments might be better.  This question will be studied in an upcoming clinical trial called TRITON.  Lastly, as the complexity of PAH treatment continues to increase, so does the cost.  As a PAH community, we need to continue to advocate for cost-effective treatments.