Emerald Health Pharmaceuticals (EHP) announced the European Medicines Agency (EMA) has granted Orphan Designation for its product candidate, EHP-102, for the treatment of Huntington’s disease (HD).
“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,” said Jim DeMesa, MD, President and CEO of EHP. “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 HD where EHP strives to transform lives by treating the disease with a unique therapy.”
The European Commission’s European Medicines Agency (EMA) plays a central role in facilitating the development and authorization of medicines for rare diseases, or “orphan medicines." To qualify for orphan designation in Europe, a medicine must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating; the prevalence of the condition in the European Union must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development; and no satisfactory existing method of diagnosis, prevention or treatment of the condition concerned can be authorized or, if such a method exists, the new medicine must be of significant benefit to those affected by the condition.
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Orphan Designation provides potential incentives from the EU to develop a medicine for a rare disease, including protocol assistance, reduced fees, funding from the European Commission for clinical trials, and protection from competition once the medicine is placed on the market, including ten years of market exclusivity.
EHP-102 is an oral formulation of a patented, synthetic new chemical entity (NCE) derived from cannabigerol (CBG), a cannabinoid that has been shown to be anti-inflammatory with evidence of neuroprotection. The NCE in EHP-102 has been modified to provide even more potent benefits compared to CBG by also affecting the peroxisome proliferator-activated receptor gamma (PPARγ), a key molecular target for the treatment of Huntington’s disease, as well as targeting other physiologic pathways involved in neuronal survival. EHP is developing this proprietary new drug product candidate initially for Huntington’s disease and Parkinson’s disease. EHP-102 received Orphan Drug Designation by the U.S. FDA for Huntington’s disease in February 2018.
Huntington’s disease is a genetic disorder that causes progressive degeneration of nerve cells in the brain. It is a devastating and disabling disease that affects middle-aged people, with typical onset between the ages of 30 and 50. Symptomatic HD affects approximately 60,000 people in Europe and 35,000 people in the U.S., with another 200,000 people in the U.S. at-risk of inheriting the disease. There is a 50% chance that HD will be passed to an offspring. One of the characteristic signs and symptoms of HD is involuntary (choreaform) movements. Additional symptoms include difficulty swallowing, slurred speech and choking. Death is usually from secondary causes of the disease such as choking and infection. There is no curative treatment for HD. Treatment is mostly directed at symptomatic relief with suppression of the movement disorders.