According to an observational study published in the Journal of Heart and Lung Transplantation in April, the use of Actelion’s oral Uptravi (selexipag) was shown to be safe and well-tolerated in children with pulmonary arterial hypertension when given as an add-on treatment. Selexipag is the only prostacyclin receptor agonist approved to target the prostacyclin pathway in adults with pulmonary arterial hypertension and is indicated for delaying disease progression and reducing the risk of hospitalisation. According to GlobalData, children under age 18 years had a higher number of adjusted incident cases of pulmonary arterial hypertension in the six major markets (6MM) (US, UK, France, Germany, Italy, Spain) in 2019 compared to the 18–19 years old age group. Moreover, pediatric patients often may not tolerate invasive procedures or continuous intravenous administration of a prostacyclin analogue therapy such as epoprostenol. GlobalData believes oral selexipag could improve treatment for the pediatric population based on its convenient oral administration, better safety, and better tolerability profile compared to currently available options.
Moreover, treatments for children with pulmonary arterial hypertension differ from adults. Although not approved in the pediatric population, the study showed that oral selexipag had minimal adverse effects with a transient dose reduction approach, as well as improved right arterial pressure, pulmonary arterial pressure, and systemic arterial pressure. Additionally, children had lower pulmonary vascular resistance at follow-up. According to the World Symposia on Pulmonary Hypertension Association, calcium channel blockers remain the treatment of choice in children with pulmonary arterial hypertension. Other treatments choices include bosentan and sildenafil. One benefit of oral selexipag over current treatment choices is that it has demonstrated that it significantly lowered the incidence of morbidity-mortality events and disease progression, both as a monotherapy and as a combination therapy agent. Key opinion leaders interviewed by GlobalData have emphasised that selexipag in adults may also have an advantage over another marketed prostacyclin mimetic, United Therapeutics’ marketed Orenitram (oral treprostinil), given its favourable safety and tolerability.
This study was the largest exploratory pediatric cohort to take place and consisted of researchers from the European Pediatric Pulmonary Vascular Disease Network at three pulmonary hypertension centres. A total of 15 children from each centre were evaluated. The children’s ages ranged from seven months to 17 years. Despite the study’s promise, these results must be confirmed in larger prospective studies before current pulmonary arterial hypertension treatments in children are altered, including the use of selexipag in the younger population.