FDA grants orphan drug designation to pouchitis treatment derived from green tea
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The FDA has granted orphan drug designation to PharmassêtX’s PSX-514, a drug derived from an active component of green tea, for the treatment of pouchitis, according to a company press release.
Currently, there is no approved treatment for pouchitis, a rare complication for patients with ulcerative colitis who undergo ileal pouch-anal anastomosis – or J-pouch procedure. PharmassêtX has been developing epigallocatechin gallate (EGCG), the main ingredient in green tea, to create a “high-purity, high-potency drug for the treatment of inflammatory bowel disease.”
The company noted that EGCG, obtained from the Camellia sinensis tea plant has demonstrated efficacy in targeting NF-B, a key regulator of mucosal immunity, “which promotes the expression of multiple pro-inflammatory genes dysregulated in IBD.” Targeting NF-B could represent an effective strategy for IBD management, either alone or “as an adjunct in combination with other therapeutics,” according to the release.
The FDA based its decision on data from a randomized, double-blinded, placebo-controlled, investigator-initiated clinical study of an EGCG-enriched green tea extract, previously published in Inflammatory Bowel Diseases. According to study results, patients with mild to moderate UC demonstrated clinical improvement, as well as endoscopic improvement, following treatment with no reported serious adverse events.
“We are grateful to be positioned to provide a much-needed innovative drug, for which there is already substantial evidence of safety and effectiveness, to the underserved community of pouchitis patients,” Terry Minton, co-founder and CEO of PharmassêtX, said in the release. “Our mission as an organization is to improve outcomes and enhance quality of life for IBD patients. The granting of this ODD is an important milestone along the critical path to accomplishing that mission.”
The company has recently partnered with Alimentiv, a clinical research organization specializing in gastroenterology, to “expedite patient access to PSX-514 and advance the drug into the clinic next year.”
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
Perspective
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Jessica Philpott, MD, PhD
More than half of IBD patients who undergo colectomy with restorative ileocolonic anastomosis may experience pouchitis. Therapy is limited, and for some, use of IBD advanced therapies, including biologics, are required for ongoing management. As such, advancement in pouch therapy is welcome.
The compound EGCG, found in green tea and touted for its antioxidant effect, has been studied as a treatment for various diseases from Parkinson’s to cancer. It is currently available as an over-the-counter, unregulated supplement. EGCG appears to be low-risk, although animal studies and human cases suggest a risk for liver and renal toxicity, usually due to excess dosing. The proposed mechanism of effect in IBD is intriguing.
When recommending therapies, we weigh efficacy, safety and cost. Using OTC supplements to treat disease can be problematic due to uncertain sources and cost to patients. If EGCG is to be produced and marketed by PharmassêtX, we would look forward to reassurance of a pure supplement with a regulated dose and institutional support that might allow insurance coverage.
I personally will be looking for more data on safety and efficacy before I routinely recommend EGCG. I also will look for the pharmaceutical industry and researchers to continue efforts on rigorous studies to result in more FDA-approved therapies for this “orphan” disorder.
Reference:
Phytomedicine. 2024;130. https://www.sciencedirect.com/journal/phytomedicine/vol/130/suppl/C.
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