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.