Orphan status is given to medications designed to treat, prevent or diagnose rare life-threatening or chronically debilitating disorders that affect no more than five in 10,000 people in the European Union. The designation comes benefits including financial incentives for drug development and commercialization, EU market exclusivity for 10 years following approval, funding from the European Commission for clinical trials, and fee exemptions and reductions.
“There is an urgent need for new, innovative treatments for patients with Huntington’s disease, a genetic disease which causes progressive degeneration of nerve cells in the brain and is associated with significant morbidity and mortality,” Jim DeMesa, MD, president and chief executive officer of EHP, said in a press release.
“This EMA orphan designation adds to the previous granting of orphan drug designation by the FDA, providing us additional global advantages by recognizing the unmet need with [Huntington’s disease] where EHP strives to transform lives by treating the disease with a unique therapy,” DeMesa said.
EHP-102 is a patented therapy based on cannabigerol (CBG) — a compound with strong anti-inflammatory and neuroprotective properties originally found in the cannabis plant — that is currently being developed for the treatment of Huntington’s and Parkinson’s disease.
To boost the therapy’s clinical benefits, researchers modified EHP-102 so that it would also target peroxisome proliferator-activated receptor gamma — a key molecular target for the treatment of Huntington’s — as well as other signaling pathways involved in neural survival.
A study published in the journal Scientific Reports showed that EHP-102 prevented the loss of neurons in Huntington’s-like disease mouse models, improved motor function and boosted antioxidant defenses in the brain.
The findings led EHP scientists to conclude the compound could be a promising candidate for the treatment of Huntington’s and other neurodegenerative diseases associated with brain inflammation, such as Parkinson’s.
In a follow-up study, EHP-102 was compared to two other CBG-like compounds — CBGA-Q and CBGA-Q-Na Salt — that had previously shown anti-inflammatory and neuroprotective effects in a mouse model of Parkinson’s. Of the three compounds, EHP-102 was the one that led to better responses in a mouse model of Huntington’s.
The company is completing all required preclinical studies before EHP-102 enters human clinical trials.