FDA Grants Orphan Drug Status to PharmassêtX's Green Tea-Based IBD Treatment

PharmassêtX Inc., a pharmaceutical development company at the late preclinical stage, has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to epigallocatechin gallate (EGCG). EGCG, a component of green tea, is being developed by the company as a high-purity, high-potency drug aimed at treating inflammatory bowel disease (IBD).

The FDA’s designation specifically targets the treatment of pouchitis, a painful and rare form of IBD that occurs in patients who have had their large intestine surgically removed. Currently, there are no FDA-approved treatments for pouchitis, making this designation a significant development for patients with this condition.

Terry Minton, Co-Founder and CEO of PharmassêtX, expressed gratitude for the FDA’s recognition, highlighting the company’s ongoing mission to improve the quality of life for IBD patients. He emphasized that this achievement represents an essential step in fulfilling their mission to provide innovative treatments to underserved communities.

Clinical studies have shown EGCG to be both safe and effective for IBD. Evidence published in the journal "Inflammatory Bowel Diseases" demonstrated that patients with mild to moderate ulcerative colitis, a common form of IBD, saw clinical improvements after receiving treatment with an EGCG-enriched green tea extract. Another study, published in the "International Journal of Colorectal Diseases," found that refractory pouchitis patients experienced significant relief from symptoms without serious adverse events.

These studies laid the groundwork for the establishment of PharmassêtX, driven by founder Gerald W. Dryden, Jr., MD, PhD. Dr. Dryden’s clinical experience and collaboration with notable colleagues worldwide have reinforced the safety and efficacy of EGCG for various forms of IBD. He noted that previous efforts to develop EGCG for cancer chemoprevention were halted due to corporate changes unrelated to the drug’s effectiveness or safety. This interruption highlighted the urgent need to revive EGCG development for treating IBD, particularly pouchitis.

EGCG’s mechanism targets NF-κB, a regulator of mucosal immunity involved in the expression of multiple pro-inflammatory genes in IBD. Other potential benefits include decreased activation of the NLRP3 inflammasome, reduced secretion of pro-inflammatory interleukins, and enhanced integrity of the intestinal barrier. Studies suggest that targeting NF-κB can be a viable strategy for managing IBD, either alone or combined with other treatments.

To expedite the clinical development of their ultra-pure EGCG drug, PSX-514, PharmassêtX has partnered with Alimentiv, a global contract research organization based in Canada. Alimentiv specializes in conducting clinical trials for gastrointestinal conditions, including Crohn’s disease, ulcerative colitis, and pouchitis. This collaboration aims to bring PSX-514 to clinical trials next year, a crucial step for regulatory approval and insurer coverage.

Inflammatory bowel disease affects nearly 1 in 100 Americans, according to a 2023 study by the Crohn's and Colitis Foundation. IBD encompasses disorders causing chronic gastrointestinal inflammation, with symptoms such as diarrhea, abdominal pain, nausea, fever, loss of appetite, fatigue, and rectal bleeding. Crohn’s disease and ulcerative colitis are the two most common forms of IBD, and there is currently no cure.

Pouchitis, a common complication following surgery for ulcerative colitis, remains a significant challenge due to the lack of FDA-approved treatments. The FDA’s ODD facilitates the development of new treatments for rare diseases by offering incentives like tax credits for clinical trials, exemption from user fees, and potential market exclusivity.

EGCG, derived from the tea plant Camellia sinensis, has been extensively researched for its health benefits, evidenced by thousands of peer-reviewed studies. PharmassêtX, founded in 2024 and based in Louisville, Kentucky, aims to address the unmet needs of IBD patients. The company looks forward to advancing its mission through ongoing research and development efforts.