Ambrisentan

To address the lack of a pediatric formulation for the pulmonary arterial hypertension drug ambrisentan, a 1 mg/mL suspension was prepared using commercial suspending agents. A validated HPLC method showed that samples stored at room temperature with light exposure or under refrigeration in the dark retained >90% potency over 90 days, with no detectable breakdown products and consistent color and odor. This extemporaneously compounded ambrisentan suspension is stable for up to 90 days under both storage conditions, supporting its use in pediatric patients.

Fig. 1 Reversed-phase high-performance liquid chromatography chromatograms of forced degradation experiments of standard ambrisentan showing degradation products (DP). (Cramer J, et al., 2021)

The ALEPH trial is a multicenter, randomized, double‑blind, placebo‑controlled study designed to evaluate ambrisentan monotherapy in patients with early‑stage pulmonary arterial hypertension (mean pulmonary artery pressure >20 and <25 mmHg, pulmonary vascular resistance >2 and ≤3 WU, WHO low‑risk, treatment‑naïve). Participants receive ambrisentan or placebo for 12 months. The primary endpoint is a composite of PAH progression; secondary endpoints include hemodynamic and echocardiographic changes, NT‑proBNP, functional class, 6‑minute walk distance, and time to hospitalization or death. This study aims to provide high‑quality evidence on early therapeutic intervention in mild hemodynamic disease.

Fig. 2 Study Design and Follow-Up Schedule. (Kan JY, et al., 2026)