Invex Therapeutics gets EU approval to develop Presendin for adolescents with idiopathic intracranial hypertension
EMA approval of the paediatric investigation plan enables Invex to develop Presendin for treating adolescent patients who have reached puberty and suffer from IIH.
A paediatric investigation plan (PIP) submitted by Invex Therapeutics (ASX: IXC) for the development of lead drug Presendin in the treatment of idiopathic intracranial hypertension (IIH) in adolescents has been accepted by the European Medicines Agency.
The approval will allow Invex to develop Presendin for IIH patients who are under 18 years of age and have reached puberty.
The PIP includes a decision adopting a waiver for Invex from conducting a trial in pre-pubertal males and females under 12 years of age.
It also includes a deferral, allowing for a paediatric trial to commence after the receipt of positive data from Invex’s Evolve phase III clinical trial in adult IIH patients.
Proposed trial
The proposed paediatric trial will be a randomised, placebo-controlled, double-blind, multi-centre format recruiting at least 40 adolescent males and females with IIH and an additional number of adult patients for treatment with either Presendin or a placebo over a 24 week period.
The primary endpoint will be determined by a change in papilloedema (swelling of the optic disc) assessed by optical coherence tomography over the course of the trial.
Key secondary endpoints will include perimetric mean deviation, headache evaluation and visual acuity at 24 weeks, along with safety assessments and concomitant medication use throughout the study.
The final design will be guided by clinical outcomes from the Evolve phase III trial.
A positive outcome to the paediatric trial would permit Invex to gain an additional authorisation in Europe for IIH, expanding market access to an adolescent population where the incidence of IIH continues to climb and associated healthcare costs are high.
Raised intracranial pressure
IIH features severely raised intracranial pressure which causes disabling daily headaches and can compress the optic nerve.
The usual age of onset is between 20 and 30 years of age, and it is most common in women who are obese.
IIH is a rapidly-growing orphan indication and its incidence has increased by more than 350% in the last 10 years.
In the UK, the overall paediatric incidence of IIH is approximately 0.7 per 100,000 individuals.