Clinical-stage biopharmaceutical company Invex Therapeutics Ltd (ASX:IXC) has been granted orphan drug designation (ODD) from the European Medicines Agency (EMA) for Exenatide in the treatment of moderate to severe Traumatic Brain Injury (TBI).
This is the second ODD for Exenatide in Europe, with Invex receiving an ODD for Idiopathic Intracranial Hypertension (IIH) in 2017, alongside an ODD from the US Food and Drug Administration (FDA), also for IIH.
The EMA provides a range of incentives in the European Union (EU) for medicines that have been granted an orphan designation, including ten years market exclusivity from the date of approval, clinical trial protocol assistance, access to the centralised authorisation procedure in Europe, and certain fee reductions.
TBI affects adult and paediatric population globally and remains one of the major cause of traumatic death and disability across all ages worldwide. Overall, 57,000 TBI-related deaths and 1.5 million hospitalisations occur every year in the European Union.
Management of intracranial pressure (ICP) elevation is considered critical in patients with moderate to severe TBI, however, at present, there are no EMA or FDA approved therapies specifically for the treatment of intracranial hypertension in this patient population.
Invex is currently investigating Presendin, a once per week, sub-cutaneous, sustained-release (SR) Exenatide microsphere formulation in a randomised, double-blind, placebo-controlled Phase III clinical trial (IIH EVOLVE) in 240 IIH patients across the globe.
Invex believes Presendin will be able to reduce ICP in a number of conditions associated with elevated ICP, including moderate and severe traumatic brain injury.
Since we first discovered the potent ICP lowering effects of Exenatide in pre-clinical models and within our completed Phase II Pressure IIH trial, the company has explored other elevated intracranial pressure disorders, including TBI,” Dr Thomas Duthy Executive Director of Invex said.
“Securing a European ODD for Exenatide is an important initial step in protecting the commercial opportunity in moderate to severe TBI, which is forecast to severe TBI, which is forecast to represent an annual market opportunity of US$2.45 billion by 2027, growing 9% per annum.”