Results from study show oral selexipag for PAH effective and safe in children, says GlobalData

Oral selexipag could improve treatment for the pediatric population based on its convenient administration, better safety and better tolerability profile compared to currently available options according to GlobalData, a leading data and analytics company.

Selexipag is the only prostacyclin receptor agonist approved to target the prostacyclin pathway in adults with PAH, and is indicated for delaying disease progression and reducing the risk of hospitalization. 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.

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 centers. A total of 15 children from each center were evaluated. The children’s ages ranged from seven months to 17 years.

Kajal Jaddoo, Associate Pharma Analyst at GlobalData, comments: “According to thea recently published observational study in the Journal of Heart and Lung Transplantation, selexipag was shown to be safe and well tolerated in children with pulmonary arterial hypertension (PAH) when given as an add-on treatment. 

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 emphasized that selexipag in adults may also have an advantage over another marketed prostacyclin mimetic, United Therapeutics’ marketed Orenitram, given its favorable safety and tolerability.

Jaddoo continues: “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.”

Children under age 18 years had a higher number of adjusted incident cases of PAH 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 analog therapy such as epoprostenol. Therefore, 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.

Jaddoo adds: “Despite the study’s promise, these results must be confirmed in larger prospective studies before current PAH treatments in children are altered, including the use of selexipag in the younger population.”

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