Most PAH patients who’ve taken Uptravi for a decade are still alive
Therapy mimics prostacyclin, which prompts blood vessels to relax and widen
Most people with pulmonary arterial hypertension (PAH) who were treated with Uptravi (selexipag) and followed up for 10 years in an extension study are still alive, according to new data.
Adding two other types of PAH therapy or starting triple therapy within six months of a diagnosis increased survival at five years.
The findings were presented last month at the 2025 International Society for Heart & Lung Transplantation (ISHLT) Annual Meeting in a presentation titled, “10-year Data on Oral Selexipag: Long-Term Survival, Safety, and Dosing Insights in Pulmonary Arterial Hypertension (PAH) from the GRIPHON Study and Its Open-Label Extension (OLE).”
“These comprehensive data offer clinicians’ robust evidence to guide therapeutic decision making regarding the incorporation of Uptravi into clinical practice,” Leonardo Diaz Galvis, MD, vice president of medical affairs, pulmonary hypertension, at Johnson & Johnson Innovative Medicine, a part of Johnson & Johnson, said in a written Q&A with Pulmonary Hypertension News.
“The data presented at this year’s ISHLT congress, which includes our 10-year GRIPHON open label extension, are a testament to the progress made in the PAH treatment landscape,” said Samuele Butera, president of pulmonary hypertension and retina at Johnson & Johnson.
Uptravi is an approved oral therapy for PAH that mimics prostacyclin, a signaling molecule that lowers blood pressure by prompting blood vessels to relax and widen.
Survival rates with Uptravi
The Phase 3 GRIPHON trial (NCT01106014) compared Uptravi against a placebo in more than 1,000 people with PAH. Among the 574 patients who received Uptravi in the trial, 330 continued into an open-label extension (NCT01112306) where they continued to receive the therapy and were being followed for long-term safety and survival outcomes. The new data come from this extension study, with some patients followed for up to a decade of Uptravi treatment.
“These data represent the most comprehensive long-term safety, tolerability, and survival data for [Uptravi], and provide the longest follow-up period published to date for a PAH therapy,” the researchers wrote in their abstract.
Six out of 10 patients followed for 10 years are still alive after a decade on the therapy, the results showed.
“The 60% survival rate at 10 years further validates Uptravi ‘s role in optimizing patient outcomes and advancing treatment paradigms for PAH,” Galvis said.
Among 176 patients, nearly three-quarters (74%) were still alive at five years. The five-year survival rate was even higher among those treated with Uptravi along with two other types of PAH therapy — specifically an endothelin receptor antagonist and a phosphodiesterase type 5 inhibitor. The five-year survival rate was 80% for patients given this type of triple therapy.
For patients who started triple therapy within six months of being diagnosed, the five-year survival rate was nearly 90%, said Diaz Galvis, who noted that 179 of the 574 patients initially given Uptravi in GRIPHON were on triple therapy, with 23 initiating it within six months of their diagnosis.
According to Butera, these data “support the benefit of early Uptravi initiation in optimizing long-term patient outcomes, contributing to the advancement of PAH treatment.”
Uptravi’s safety profile
Safety data from the open-label extension were consistent with the therapy’s known safety profile. Common side effects include headache, flushing, nausea, vomiting, diarrhea, and pain in the muscles, jaw and extremities. Slightly more than a third of the open-label trial participants discontinued Uptravi over a decade of follow-up.
“Uptravi has demonstrated a consistent, long-term safety profile … These data can provide confidence to clinicians and patients of the long-term safety profile of Uptravi,” Diaz Galvis said.
Uptravi isn’t Johnson & Johnson’s only approved PAH therapy. Last year, the company won U.S. approval for Opsynvi, a once-daily tablet combining macitentan — an endothelin receptor antagonist sold by Johnson & Johnson as Opsumit — with tadalafil, a phosphodiesterase 5 inhibitor sold in the U.S. by United Therapeutics under the brand name Adcirca. The availability of a two-in-one therapy lets patients receive combination treatment while minimizing the number of daily pills they have to take, according to Johnson & Johnson.
“As administration of macitentan and tadalafil are commonly prescribed together for initial therapy for PAH, the introduction of a single tablet combination therapy is promising for clinicians treating patients, as it may help bridge the gap between clinical guidelines and everyday clinical practice,” Butera said. “Additionally, we continue to drive research that digs deeper into the issues impacting the PAH community.”
Johnson & Johnson is also running SPHERE, “the largest real-world registry of a prostacyclin pathway agent [Uptravi] to date in the U.S.,” Butera said. Ongoing Phase 3 trials include UNISUS (NCT04273945), which is designed to test if macitentan at 75 mg is better than a 10 mg dose at prolonging survival or the time to a morbidity event in participants with symptomatic PAH.
“The burden of PAH is still heavy, with many patients facing delays in diagnosis and receiving suboptimal treatment. We remain steadfast in seeking ways to bridge these gaps,” Butera said.
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