Cancer Prevention Pharmaceuticals has submitted a New Drug Application (NDA) to the FDA seeking accelerated approval for CPP 1X/sul for treatment of adults with familial adenomatous polyposis (FAP). The FDA’s accelerated approval process allows the agency to approve drugs which address a serious or life-threatening condition based on an intermediate or surrogate endpoint that is likely to predict a clinical long-term benefit. The FDA takes into account such factors as the severity, rarity, or the lack of effective current treatments. The designation has been frequently applied to cancer drugs.
In addition, CPP 1X/sul received an orphan drug designation from the FDA. Among the benefits are eligibility for seven years of market exclusivity upon approval of a drug and tax credits for various costs of clinical testing.
CPP also recently submitted a Marketing Authorization Application (MAA) with the European Union (EU) for CPP 1X/sul for the same indication. The drug received an orphan medicinal product designation for FAP following a favorable assessment provided by the European Medicines Agency’s Committee for Orphan Medicinal Products.
The filing is partly based on data from the CPP FAP-310 trial. CPP 1X/sul did not demonstrate statistical significance in outperforming the single agents in the overall population; however, further analysis of the data showed key differential effects of the agents in the lower vs upper GI anatomy. Focusing on lower GI anatomy (patients with an intact colon, retained rectum or surgical pouch), the data showed statistically significant benefit for CPP 1X/sul vs both single agents in delaying surgical events in the lower GI for up to four years.
Also, the safety profile of the combination did not significantly differ from that already known for the single agents to support the overall safety assessment of the fixed combination product for long-term therapeutic use.