Actelion initiates Phase III study to evaluate macitentan (Opsumit) in children with PAH

Actelion Pharmaceuticals Ltd / Actelion initiates Phase III study to evaluate macitentan (Opsumit) in children with PAH . Processed and transmitted by NASDAQ OMX Corporate Solutions. The issuer is solely responsible for the content of this announcement.

• Phase III study TOMORROW aims to show the long-term benefits of macitentan (Opsumit^®) in children with pulmonary arterial hypertension (PAH)
  
  

Global program has received endorsement from the US FDA (through a pediatric Written Request) and in the EU (through a Paediatric Investigation Plan)

ALLSCHWIL/BASEL, SWITZERLAND - 06 July 2016 - Actelion Ltd (SIX: ATLN) announced today that it will be initiating a Phase III study to evaluate the effect of macitentan on delaying disease progression in children with PAH using a pediatric formulation of macitentan (Opsumit).

TOMORROW (pediaTric use Of Macitentan tO delay disease pRogRessiOn in PAH Worldwide) is a multicenter, controlled, randomized, open-label event-driven study to assess the efficacy, safety and pharmacokinetics of macitentan versus standard of care in children with PAH.

The study will enroll children between the age of 1 month and 18 years in more than 20 countries. Initially, only children aged between 2 and < 18 years will be enrolled, and pharmacokinetic profiles collected in the study for up to 40 patients will allow Actelion to determine the doses for younger patients. Once the dose is determined, the study protocol will be amended to include children below 2 years of age. The children will be randomized in a 1:1 ratio into two treatment groups - macitentan administered as a pediatric formulation or standard of care as per local practice. The pediatric formulation of macitentan is a round dispersible tablet that is neutral in taste. It is available in three different dose strengths, containing 0.5 mg, 2.5 mg and 5.0 mg macitentan. To make them easier for children to swallow, the tablets can be dispersed in water on a spoon.

The trial is expected to last up to 6 years, with children being enrolled until 187 primary efficiency endpoint events have occurred. The primary efficacy endpoint is defined as the time to the first Clinical Event Committee (CEC) confirmed outcome event.

Maurice Beghetti, Head of Paediatric Cardiology at the University Hospital of Geneva, commented: "Clinical studies in children come with big challenges, such as the influence of growth stage and body weight on the dosage scheme and on potential side effects. PAH is a rare disease in adults and even more so in children, so pediatric PAH physicians have to mostly rely on research data collected in adults when weighing up treatment options for their younger patients. There is a significant medical need for showing the benefit of adequately adapted formulations and doses of PAH-specific medications in order to provide children with PAH with the most appropriate treatment. TOMORROW as a robust study assessing long-term treatment with the appropriate pediatric formulation of macitentan will deliver the sought-after data on all fronts."