On June 18, 2020 Cancer Prevention Pharmaceuticals, Inc. (CPP), a private biotech company developing novel therapeutics to prevent cancer and other diseases,reported that it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for CPP-1X/sul for treatment of adults with familial adenomatous polyposis (FAP) (Press release, Cancer Prevention Pharmaceuticals, JUN 18, 2020, https://www.canprevent.com/wp-content/uploads/2020/06/MAA-Submission-Press-Release-FINAL-2020-06-18.pdf [SID1234563866]).
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FAP is a rare genetic disease that if left untreated progresses to colorectal cancer in nearly 100% of patients. The clinical development of CPP-1X/sul was designed to establish this fixed dose combination product as a potential pharmaco-preventive drug treatment specifically for FAP patients.
"The MAA submission for our lead drug candidate, CPP-1X/sul, represents a significant milestone for FAP patients and their families," said CPP CEO Jeff Jacob. "For most FAP patients, current medical practice involves a lifetime of periodic monitoring as well as highly invasive surgical procedures. If approved, CPP-1X/sul could provide an alternative to surgery for some patients, significantly improving their quality of life."
CPP-1X/sul has received an orphan medicinal product designation for FAP by the European Commission following a favorable assessment provided by the EMA’s Committee for Orphan Medicinal Products. Among the potential benefits arising from the designation are 10 years of orphan market exclusivity as well as eligibility of the product being assessed and approved centrally in the European Union leading to grant of a marketing authorization, which is valid in all Member States of the EU and the European Economic Area.
CPP also plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration this month seeking accelerated approval for CPP-IX/sul for the same indication.
About CPP-1X/sul
CPP-1X/sul is a combination of CPP-1X (eflornithine) and sulindac (CPP-1X/sul). In a clinical trial in patients with large bowel polyps, the CPP-X/sul combination prevented > 90% subsequent pre-cancerous sporadic adenomas versus placebo. Based on the close biologic similarities with FAP, a phase 3 pivotal trial compared this same combination to each drug alone (FAP-310).
"With up to four years of treatment, the combination appears to greatly delay the need for major surgeries in the colon, rectum or surgical pouch. We hope that this new drug regimen will soon be available as an adjunct in the management of FAP patients facing large bowel surgery," said Dr. Alfred Cohen, Chief Medical Officer of CPP.
The Phase 3, pivotal FAP-310 trial of CPP-1X/sul enrolled 171 FAP patients at 17 research institutes in the U.S., Canada, and Europe. It was the largest ever prospective, controlled study performed in FAP and treated patients for up to 48 months, much longer than any other clinical trial in this population. The study was designed to determine if CPP-1X/sul is superior to sulindac or eflornithine as single agents in delaying time to the first occurrence of any FAPrelated event, such as surgical removal of the colon, rectum, surgical pouch, duodenum and/or high-risk adenomas. The trial design included FAP patients with varying lower and upper GI disease. 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 (p≤0.02) in delaying surgical events in the lower GI for up to 4 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.