Familial adenomatous polyposis Pipeline

DelveInsight’s, “Familial adenomatous polyposis - Pipeline Insight, 2025” report provides comprehensive insights about 4+ companies and 4+ pipeline drugs in Familial adenomatous polyposis pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

Geography Covered

• Global coverage

 

Familial adenomatous polyposis: Understanding

Familial adenomatous polyposis: Overview

Familial adenomatous polyposis (FAP) is an autosomal dominant syndrome caused by germline mutations in the APC gene, with variable penetrance and diverse phenotypic manifestations, including Gardner and Turcot syndromes. Individuals with FAP develop hundreds to thousands of colorectal polyps, often beginning in early adolescence, which, if untreated, confer an almost certain lifetime risk of colorectal cancer, typically by age 40.

It is caused by autosomal dominant germline mutations in the APC tumor suppressor gene on chromosome 5. Classic FAP, often resulting from nonsense mutations, is characterized by early-onset, extensive colorectal polyposis, while mutation location influences phenotypic variability, including extracolonic manifestations. Attenuated FAP, arising from mutations at specific APC codons, presents with fewer polyps, later onset, and slower progression to cancer. Variants such as Gardner syndrome and Turcot syndrome involve colonic polyposis alongside extracolonic features, including osteomas, soft tissue tumors, and central nervous system malignancies. Genetic testing is recommended for individuals with more than 20 adenomatous polyps or FAP-associated cancers, and family members should undergo surveillance even when no APC mutation is detected.

The APC gene functions as a tumor suppressor, playing a key role in chromosome alignment during metaphase and promoting apoptosis in colonic epithelial cells. Mutations in APC disrupt these processes, enabling uncontrolled cell proliferation and the formation of adenomas. Progression from adenoma to carcinoma follows a well-established sequence, with APC inactivation as the initiating event, followed by additional mutations in oncogenes and tumor suppressors such as KRAS and p53, driving dysplasia and eventual carcinoma. APC mutations are common in both familial cases and the majority of sporadic colorectal cancers.

The diagnosis of familial adenomatous polyposis (FAP) is primarily based on clinical evaluation of colorectal adenomatous polyps, with a diagnosis established in individuals presenting with 100 or more polyps, or fewer than 100 polyps accompanied by a family history of FAP. Attenuated FAP (AFAP), a milder variant, typically manifests later and with 10–99 polyps, often located in the right colon, while exhibiting variable phenotypic expression within families. Genetic testing confirms the diagnosis, with germline mutations in the APC gene on chromosome 5q21 accounting for nearly all cases. APC, a tumor suppressor, regulates Wnt signaling, cell adhesion, migration, cytoskeletal organization, and chromosome segregation, with mutations disrupting these processes and driving adenoma formation. Specific mutation sites correlate with age of onset, polyp burden, and extracolonic manifestations such as desmoid tumors, osteomas, epidermoid cysts, and congenital hypertrophy of the retinal pigment epithelium, though phenotypic expression is highly variable. Recent insights suggest that APC also regulates intestinal cell fate via modulation of DNA methylation, linking gene mutations to disrupted differentiation and tumorigenesis.

Management of familial adenomatous polyposis (FAP) is individualized based on the severity of colonic and extracolonic manifestations, patient age, health, and preferences. Definitive treatment of colonic disease involves surgical removal of at-risk tissue, with options including total proctocolectomy with ileal pouch-anal anastomosis or end ileostomy, and total abdominal colectomy with ileorectal anastomosis, each balancing cancer risk reduction against quality-of-life considerations such as continence, sexual, and reproductive function. Attenuated FAP may require less extensive surgery, with ongoing endoscopic surveillance postoperatively. Non-surgical strategies, such as sulindac or celecoxib, can reduce adenoma burden but do not prevent recurrence after discontinuation. Extracolonic manifestations, including upper gastrointestinal polyps and desmoid tumors, require vigilant screening and intervention. Gastric polyps are generally monitored, while duodenal polyps may need endoscopic or surgical resection depending on size or dysplasia, guided by Spigelman staging. Desmoid tumors, particularly mesenteric, are managed cautiously to minimize surgical risk, with medical therapies such as tyrosine kinase inhibitors used to reduce tumor size pre- and postoperatively.

 

"Familial adenomatous polyposis- Pipeline Insight, 2025" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Familial adenomatous polyposis pipeline landscape is provided which includes the disease overview and Familial adenomatous polyposis treatment guidelines. The assessment part of the report embraces, in depth Familial adenomatous polyposis commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Familial adenomatous polyposis collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

 

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence Familial adenomatous polyposis R&D. The therapies under development are focused on novel approaches to treat/improve Familial adenomatous polyposis.

 

Familial adenomatous polyposis Emerging Drugs Chapters

This segment of the Familial adenomatous polyposis report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, Preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

 

Familial adenomatous polyposis Emerging Drugs

• eRapa: Biodexa Pharmaceuticals

eRapa is a proprietary oral tablet formulation of rapamycin (sirolimus), a potent mTOR inhibitor. mTOR plays a key role in regulating cellular metabolism, growth, and proliferation and is often activated during tumorigenesis. Overexpression of mTOR in FAP polyps provides a strong rationale for using eRapa as a targeted therapy. The drug has received Orphan Designation in the US, with plans to pursue similar designation in Europe. Currently, the drug is in Phase III stage of its clinical study for the treatment of Familial adenomatous polyposis.

 Further product details are provided in the report……..

 

Familial adenomatous polyposis: Therapeutic Assessment

This segment of the report provides insights about the different Familial adenomatous polyposis drugs segregated based on following parameters that define the scope of the report, such as:

 

Major  Players in Familial adenomatous polyposis

• There are approx. 4+ key companies which are developing the therapies for Familial adenomatous polyposis. The companies which have their Familial adenomatous polyposis drug candidates in the most advanced stage, i.e. Phase III include, Biodexa Pharmaceuticals.

 

Phases

DelveInsight’s report covers around 4+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

 

Route of Administration

Familial adenomatous polyposis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

 

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

 

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

 

Familial adenomatous polyposis: Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Familial adenomatous polyposis therapeutic drugs key players involved in developing key drugs.

 

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Familial adenomatous polyposis drugs.

 

Familial adenomatous polyposis Report Insights

• Familial adenomatous polyposis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

 

Familial adenomatous polyposis Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

 

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Familial adenomatous polyposis drugs?
• How many Familial adenomatous polyposis drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Familial adenomatous polyposis?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Familial adenomatous polyposis therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Familial adenomatous polyposis and their status?
• What are the key designations that have been granted to the emerging drugs?