The US Food and Drug Administration has granted orphan drug designation to Edge Therapeutics's lead product candidate EG-1962 which is in clinical trials treating patients that have suffered an aneurysmal subarachnoid hemorrhage (aSAH), also known as a ruptured brain aneurysm.

Orphan drug designation is granted for novel drugs or biologics to treat rare medical diseases or conditions that affect less than 200,000 people in the United States. The designation qualifies the sponsor for numerous incentives including seven years of market exclusivity after the drug’s approval, tax credits for clinical research costs and application fee reductions.

“We are very pleased to have received orphan drug designation for EG-1962. We believe our product can fundamentally improve patient outcomes following subarachnoid hemorrhage, which is such a catastrophic life-threatening condition,” said Brian Leuthner, president and chief executive officer of Edge Therapeutics.

“The benefits and incentives for an approved drug that has orphan drug designation, including marketing exclusivity periods, are strategically important from a regulatory and commercial perspective.”

In the US, approximately 35,000 people with an average age of 52 are hospitalised each year for aSAH, and approximately 75 per cent of these patients die or suffer permanent brain damage within 30 days.

Edge Therapeutics is a clinical-stage biotechnology company that discovers, develops and seeks to commercialise novel, hospital-based therapies capable of transforming treatment paradigms in the management of acute, life-threatening neurological conditions.

EG-1962 is a novel polymeric nimodipine microparticle that utilises Edge’s proprietary Precisa development platform. EG-1962 is designed to avoid the dose-limiting side effects associated with oral nimodipine, including hypotension, by administering treatment directly to the site of the injury. Edge is also formulating a second compound, EG-1964, for prevention of recurrence of chronic subdural hematoma.

The NEWTON (Nimodipine microparticles to Enhance recovery While reducing TOxicity after subarachNoid hemorrhage) study is a multicentre, randomised, controlled, open-label clinical trial evaluating the safety, tolerability and pharmacokinetics of escalating doses of EG-1962 compared to the current standard of care, oral nimodipine, in subjects with aSAH.

As previously announced, Edge completed patient enrollment in all six cohorts of its ongoing phase 1/2 NEWTON trial in North America. Safety data from the first three cohorts were also reported and no patients experienced EG-1962 related hypotension in the treated group, while 33 per cent of patients treated with oral nimodipine experienced hypotension.

The company is also assessing patient functional outcomes at 90 days in each study cohort, which it believes could be indicative of the potential efficacy of EG-1962. At the mid-point of the study, favourable outcomes were reported in 67 per cent of patients treated with EG-1962 compared with 22 per cent of those patients treated with standard of care oral nimodipine.

EG-1962 and EG-1964 both utilise Edge’s proprietary, programmable, biodegradable polymer-based development platform, known as Precisa. The Precisa platform allows Edge to create therapeutics capable of delivering medicines directly to the site of injury, providing a novel delivery mechanism that enables targeted and sustained drug exposure while potentially avoiding the systemic, dose-limiting side effects often associated with current standards of care.