A study published in The Lancet evaluated the safety and efficacy of ralinepag in patients with pulmonary arterial hypertension (PAH).
PAH is a rare condition affecting the heart and lungs, characterized by abnormally high blood pressure (hypertension) in the pulmonary artery. Symptoms include shortness of breath during exercise and fainting spells. The symptoms tend to get worse over time and may include dizziness, edema of the ankles or legs, chest pain, and a racing pulse. In most cases of PAH, the cause is unknown but some cases are due to genetic changes in the BMPR2 gene. It can also occur secondary to underlying conditions such as connective tissue diseases, HIV infection, chronic hemolytic anemia, and congenital heart disease, or be induced by certain drugs and toxins.
Ralinepag is an oral, once-daily, selective prostacyclin IP receptor agonist developed to treat PAH. ADVANCE OUTCOMES was a randomized, double-blind, placebo-controlled, event-driven, phase 3 clinical trial (NCT03626688) testing ralinepag in adult patients with PAH.
A total of 687 patients were included in the full analysis and safety sets, of whom 350 received ralinepag and 337 received placebo. Median follow-up from randomization to clinical worsening event, censoring, or study closure was 85 weeks in the ralinepag group and 78.4 weeks in the placebo group. At baseline, patients had a mean 6-minute walk distance (6MWD) of 438.9 m and 80% of patients were receiving dual background PAH therapy.
Overall, 18% of patients in the ralinepag group and 36% of patients in the placebo group had a first clinical worsening event. The largest numerical between-group differences in components of the composite outcome were observed for disease progression, initiation of parenteral or inhaled prostacyclin-pathway therapy, and unsatisfactory long-term clinical response.
Adverse events were the primary reason for treatment discontinuation in 19% of the ralinepag group and 3% of the placebo group. Serious adverse events occurred in 28% of the ralinepag-treated group and 31% in the placebo group. Adverse events leading to death occurred in 15 (4%) and 14 (4%) patients, respectively.
To learn more about PAH and other rare lung conditions, visit https://checkrare.com/diseases/lung-diseases/