Therapyx receives FDA Orphan Drug Designation for FAPXIL, a first-in-class immune therapy for the treatment of Familial Adenomatous Polyposis

Buffalo New York, September 15, 2023 /PRNewswire/ — Therapyx today announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Product Development (OOPD) has granted the company’s request for orphan-drug designation of FAPXIL, the company’s investigational therapy in development for the treatment of familial adenomatous polyposis (FAP). The autosomal dominant disease is characterized by the development of hundreds to thousands of adenomas in the rectum and colon during the second decade of life. FAP has an incidence at birth of about 1/8,300, it manifests equally in both sexes, and affects up to 50,000 Americans. There is no cure.

FAPXIL consists of the key immune regulatory cytokine interleukin 10, formulated for oral delivery and sustained release in the gut. Its putative immune mechanism of action in FAP, the neutralization of disease-causing regulatory T cells, would make it a first-in-class immune therapy for FAP with potential applications in other diseases, such as inflammatory bowel disease.

“We are delighted that the FDA has granted orphan-drug designation to FAPXIL. Combined with our recently awarded phase IIb grant to continue the product’s development, this highlights the importance of advancing new treatments for patients living with FAP,” said Dr. Nejat K. Egilmez, CEO at Therapyx. “FAP is a devastating disease of childhood that has no cure and few treatment options. We look forward to advancing FAPXIL to the clinic and are currently exploring partnership opportunities to help bring this novel, oral treatment to FAP patients and their families as quickly and safely as possible.”

Therapyx continues the development of its other products, PCX12TM, GneX12TM and NGoXIMTM towards the clinic. The company also has a deep pipeline of discovery stage products. Moreover, certain products are likely to be eligible for FDA orphan drug designation, as well as 505(b)(2) accelerated regulatory development pathway.

Therapyx is developing a proprietary drug delivery system called ExStaM, based on mild encapsulation of highly potent protein therapeutics into nano/microparticles that preserves bioactivity, achieves controlled tissue specific release, reduced toxicity and shelf-stability.  Its lead products are aimed at cancer, mucosal infections and immune mediated inflammatory diseases. For more information on Therapyx, Inc. visit the Company’s website at

CAUTIONARY STATEMENT: This press release may contain forward-looking statements concerning the potential and prospects of the Company’s drug discovery program and its drug candidates. Any statement describing a goal, expectation, intention or belief of the Company is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, including the ability to complete preclinical and clinical trials successfully and within specified timelines, if at all; the ability to obtain regulatory approval; the Company’s future capital needs and ability to obtain additional funding; the ability of the Company to protect its intellectual property rights and to not infringe the intellectual property rights of others; the development of competitive products by other companies; and other risks detailed from time to time in the Company’s filings and press releases. Actual results may differ materially from those suggested in this press release.

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