FDA Fast Tracks First Possible Treatment for Familial Adenomatous Polyposis

In late September, Food and Drug Administration designated “fast track” status to the research and development of CPP-1X/sul for adults diagnosed with Familial Adenomatous Polyposis (FAP).
Overseeing the project are Cancer Prevention Pharmaceuticals and Sucampo Pharmaceuticals, who hope to provide the first approved therapy to victims of FAP.

The experimental drug, currently undergoing a Phase 3 Clinical trial, is a combination of the ornithine decarboxylase inhibitor CPP-1x and a anti-inflammatory, non-steroidal supplement, suldinac. Researchers are currently investigating whether or not the combination proves more successful than treatment with each drug as separate agents. CPP, who just last year bought the exclusive licensing rights to commercialize CPP-1X from Sucampo, hopes to roll out the drug as soon as full FDA approval is met.

CPP CEO Jeff Jacob said in a statement“The FDA’s decision to grant Fast Track status for CPP-1X/sul is good news for FAP patients who currently have no approved therapies.” The “fast track” status also opens up CPP’s drug to FDA Accelerated Approval and Priority Review, further quickening its release.

Familial Adenomatous Polyposis (FAP) is a genetic disorder caused by germline mutations in the APC gene, resulting in the formation of excessive polyps in the large intestine and rectum. Symptoms may include abdominal pain, weight loss, uncontrollable bowels, or blood or mucus in the stools. Because of previous lack of treatment, FAP would be left to progress into colorectal cancer in 100% of cases.

To read more about this FDA move, check out the Rare Disease Report here.

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