This designation is intended to expedite the development and review of new therapies that fill an unmet medical need in serious illnesses. It includes more frequent meetings and communication with the FDA, as well as the possibility of accelerated approval and priority review.
Emerald Health Pharmaceuticals, the company developing EHP-101, is opening a Phase 2 clinical trial (NCT04166552) in Australia, New Zealand, and the U.S. This study, yet to start enrolling, will test the therapy’s safety, tolerability, pharmacokinetics (its absorption, distribution, metabolism, and excretion) and preliminary efficacy, at low and high daily doses, in an estimated 36 adults with diffuse cutaneous scleroderma.
Effectiveness will be measured through questionnaires and standardized clinical tests, such as the American College of Rheumatology composite response index in diffuse cutaneous Systemic Sclerosis.
EHP-101’s active ingredient, called VCE-004.8, is a synthetic derivative of CBD, which is extracted from the cannabis plant and works to limit inflammation in scleroderma by activating two protein receptors — peroxisome proliferator-activated receptor gamma (PPARɣ) and cannabinoid receptor type 2 (CB2). VCE-004.8, a non-psychotropic compound, is not considered a controlled substance by the U.S. Drug Enforcement Administration (DEA).
EHP-101 also targets the hypoxia-inducible factor (HIF) pathway that is involved in fibrotic (scarring) diseases such as scleroderma.
In a mouse model of scleroderma, EHP-101 demonstrated anti-inflammatory and anti-fibrotic effects, promoted the recovery of damaged blood vessels, and reduced skin thickness.
These findings supported a Phase 1 trial (NCT03745001) testing EHP-101’s safety in healthy volunteers. The therapy was well-tolerated, with only mild-to-moderate side effects reported at higher doses, which Emerald Health said were much higher than the anticipated doses used in scleroderma patients.
Both the FDA and European Medicines Agency previously granted orphan drug designation to EHP-101 for the same indication.
“This Fast Track designation is another positive step for us in our development of this much-needed therapy,” Jim DeMesa, MD, president & CEO of Emerald Health, said in a press release. “With a Phase 2 study being initiated, this Fast Track designation, along with the previously granted Orphan designations, gives us a regulatory path to most efficiently achieve our clinical development plans.”
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