Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.Actelion Pharmaceuticals has announced the results of the first randomized controlled trial in portopulmonary hypertension (PoPH), which showed Opsumit (macitentan) significantly improved pulmonary vascular resistance (PVR) compared with placebo, meeting the primary endpoint of the study.
Image: Actelion’s Opsumit has improved pulmonary vascular resistance in trial of portopulmonary hypertension. Photo: courtesy of Jan Friml / FreeImages.
Subscribe to our email newsletter
Presented as a late-breaking oral presentation at the European Respiratory Society (ERS) meeting in France, the PORtopulmonary hypertension Treatment wIth maCitentan ─ a randOmized clinical trial (PORTICO) showed the safety of macitentan in PoPH was consistent with that observed in previous clinical trials.
PoPH is a subset of pulmonary arterial hypertension (PAH), associated with portal hypertension (increased blood pressure in the portal vein)1 often due to cirrhosis2.
PoPH is increasingly recognized and evidence suggests that it is the fourth most common form of PAH3. In many cases, patients with PoPH are only diagnosed as part of an assessment for liver transplantation; however, severe PAH is a contraindication for liver transplant due to poor post-operative prognosis.
South Paris University respiratory medicine professor and lead investigator Olivier Sitbon said: “The findings of PORTICO are relevant because if patients with PoPH can be treated to successfully lower pulmonary vascular pressure and resistance, more patients may be eligible for liver transplant as they will potentially have a better prognosis for this surgery.
“The fact that the hepatic safety profile of macitentan in patients with PoPH was consistent with that observed in previous trials is particularly reassuring, as PoPH patients are typically excluded from PAH clinical trials on safety grounds.”
Data supporting the use of PAH therapies in PoPH are extremely limited and are mostly from single-center, open-label studies5-7. To date, none of the approved PAH treatments have been shown to improve cardiopulmonary hemodynamics in PoPH in a randomized controlled clinical trial.
In the PORTICO trial8, patients were randomized to receive macitentan 10mg (n=43) or placebo (n=42) once daily.
After 12 weeks of treatment, macitentan significantly improved PVR (primary endpoint met, 35% reduction vs placebo, p<0.0001), mean pulmonary arterial pressure (mPAP; macitentan reduced mPAP by 5.99 mmHg vs placebo, p<0.0001) and cardiac index (macitentan increased cardiac index by 0.52 L/min/m2 vs placebo, p=0.0009)9.
There was no significant difference between macitentan and placebo groups in six-minute walk distance (6MWD) or WHO Functional Class (FC). The most common adverse events (macitentan vs placebo) were peripheral edema (25.6 vs 11.9%) and headache (16.3 vs 16.7%)9.
OPSUMIT is an orally active endothelin receptor antagonist (ERA) that is currently approved in the US and Europe for the treatment of PAH.
Source: Company Press Release
