NDA Submitted for CPP-1X/sul, a Treatment for FAP

According to a recent press release, biotechnology company Cancer Prevention Pharmaceuticals (CPP) submit a New Drug Application (NDA) for accelerated approval of their drug therapy CPP-1X/sul to treat familial adenomatous polyposis (FAP).

Familial Adenomatous Polyposis (FAP)

Familial adenomatous polyposis (FAP) is a rare genetic condition resulting from a defective APC gene. It causes hundreds of polyps to form by the time a patient is 16. Polyps (abnormal clumps of tissue) develop in the large intestine, upper gastrointestinal track, and/or rectum. Without treatment, this condition nearly almost results in cancer by age 40.

Symptoms of FAP include:

  • Skin cysts
  • Dental abnormalities
  • Bony growths
  • Stomach, small bowel, and duodenum polyps
  • Freckling on the whites of the eyes
  • Bloody stool
  • Unexplained weight loss
  • Gassiness and bloating
  • Colorectal cancer

Learn more about FAP.

New Drug Application

Basically, a New Drug Application (NDA) is submitted when a company asks the FDA to allow the sale and marketing of a specific drug. According to the FDA, there are three main goals of a NDA, which determine:


  • Whether the drug is safe and effective in its proposed use(s), and whether the benefits of the drug outweigh the risks;
  • the drug’s proposed labeling (package insert) is appropriate, and what it should contain; [and]
  • whether the methods used in manufacturing the drug and the controls used to maintain the drug’s quality are adequate to preserve the drug’s identity, strength, quality, and purity.


As CPP-1X/sul received Orphan Drug Designation from the FDA, which means it gained special status for its effective treatment of a rare disease, it is likely the NDA will be approved. The FDA describes Orphan Drug Designation as:

special status [granted] to a drug or biological product to treat a rare disease or condition [affecting 200,000 people or less]. Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing.

CPP-1X/sul for FAP

CPP-1X/sul is a combination drug containing CPP-1x (eflornithine) and Sulindac. At first, researchers wanted to understand whether the combination was safer, more effective, and more well-tolerated than either drug on its own. While the combination was not necessarily shown to be more effective, it did show certain benefits. For example, patients with FAP may be able to be treated for longer periods of time (up to 4 years), reducing symptoms and delaying the onset of cancer, polyps, or other symptoms.
Additionally, when tested in a study of 171 patients, researchers found that CPP-1X/sul prevented >90% of pre-cancerous sporadic adenomas in patients with large bowel polyps as compared to patients taking placebos. As many current treatments are more time-consuming and invasive, CPP-1X/sul could provide immense benefits to patients.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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