Emerald Wins FDA Orphan Status for Cannabis-derived Systemic Scleroderma Therapy

Emerald Health Pharmaceuticals, which develops synthetic medications based on cannabinoid science, has received orphan drug status from the U.S. Food and Drug Administration (FDA) for its lead molecule, EHP-101, to treat systemic scleroderma (SSc).

EHP-101, which also has the potential to treat multiple sclerosis (MS), is one of Emerald’s two proprietary cannabinoid drugs in preclinical development. Cannabinoids are chemical compounds found in the cannabis plant.

San Diego-based Emerald will launch a Phase 1 human clinical study in 2018 and possibly Phase 2 studies for both MS and SSc in 2019.

“Orphan designation represents an important regulatory milestone for our company as we advance EHP-101 for the treatment of systemic scleroderma,” Dr. Jim DeMesa, Emerald’s CEO, said in a press release. “We are working diligently to address the significant unmet medical need in people suffering from this deadly disease, and this designation furthers our mission to develop impactful cannabinoid-derived medicines to improve clinical outcomes for patients with life-threatening diseases.”

An orphan drug designation expedites review of drugs and biologics aimed at treating rare diseases, defined as those affecting fewer than 200,000 Americans. Benefits include seven-year marketing exclusivity, tax credits and federal grants.

SSc is a multi-system autoimmune disease whose symptoms include muscle inflammation, swelling of the fingers and hands, and abnormalities of the immune system that affect connective tissue. Its two subtypes are limited systemic scleroderma (limited skin involvement with few systemic problems) and diffuse systemic scleroderma (widespread skin involvement accompanied by internal organ damage).

Scientists are increasingly turning to cannabinoid-derived drugs as therapeutic solutions for SSc. In March 2016, another research team developed VCE-004.8, which stimulated two receptors — CB1 and CB2 — believed to cause fibrosis development in scleroderma.

The study, “The cannabinoid quinol VCE-004.8 alleviates bleomycin-induced scleroderma and exerts potent antifibrotic effects through peroxisome proliferator-activated receptor-γ and CB2 pathways,” showed how the drug blocked the effects of a leading cause of fibrosis development by slowing collagen production and the transformation of fibroblasts into disease-causing myofibroblasts.