Basal Cell Nevus Syndrome (BCNS) - Pipeline Insight, 2024

This “Basal Cell Nevus Syndrome - Pipeline Insight, 2024,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Basal Cell Nevus Syndrome 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

Basal Cell Nevus Syndrome Understanding

Basal Cell Nevus Syndrome: Overview

The nevoid basal cell carcinoma syndrome (NBCCS) is a rare, complex genetic disorder characterized by a wide variety of developmental abnormalities and a predisposition to developing certain forms of cancer, particularly a type of skin cancer known as basal cell carcinoma. The other names for BCNS include basal cell carcinoma nevus syndrome (BCCNS), Gorlin-Goltz syndrome, Gorlin syndrome, and nevoid basal cell carcinoma syndrome. These basal cell naevi are actually tiny basal cell carcinomas. It is mostly caused by mutations in the PTCH1 gene, sometimes mutations in the SUFU and PTCH2 genes can cause a similar set of characteristics (phenotype). Common symptoms include multiple basal cell carcinomas, recurrent keratocystic odontogenic tumors of the jaws, pits of the palms and soles, and skeletal malformations. Some affected individuals may have distinctive facial features. A diagnosis of Basal Cell Nevus Syndrome (BCNS) is based upon physical examination, detection of a pathogenic genetic variant, identification of characteristic clinical findings and imaging studies. Specific therapeutic procedures and interventions may vary, depending upon numerous factors, such as disease severity; tumor size; the presence or absence of certain symptoms; an individual’s age and general health; and/or other elements. Future treatment options for BCNS patients include PTCH1- or SMO-specific targeted drugs which prevent the expression of tumor mediating genes within the hedgehog pathway.

Basal Cell Nevus Syndrome - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Basal Cell Nevus Syndrome pipeline landscape is provided which includes the disease overview and Basal Cell Nevus Syndrome treatment guidelines. The assessment part of the report embraces, in depth Basal Cell Nevus Syndrome 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, Basal Cell Nevus Syndrome 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 Basal Cell Nevus Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Basal Cell Nevus Syndrome.

Basal Cell Nevus Syndrome Emerging Drugs Chapters

This segment of the Basal Cell Nevus Syndrome report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.

Basal Cell Nevus Syndrome Emerging Drugs

Patidegib: PellePharmPellePharm is focused on developing patidegib, an investigational topical treatment designed to mitigate the tumor burden in patients with Gorlin Syndrome and Basal Cell Carcinomas (BCCs), and other potential indications. As of November 2018, PellePharm and LEO Pharma entered into a strategic collaboration to address the unmet medical needs for rare skin conditions, such as Gorlin Syndrome and High Frequency Basal Cell Carcinoma (HF-BCC). PellePharm has received both Orphan Drug Designation and Breakthrough Therapy Designation for Patidegib Topical Gel in Gorlin Syndrome from the FDA, as well as Orphan Drug Designation in Gorlin Syndrome from EMA’s Committee for Orphan Medicinal Products in the EU.Currently, the drug is in Phase III stage of Clinical trial evaluation for the treatment of Basal cell nevus syndrome.

Vismodegib: RocheVismodegib is an experimental, oral smoothened antagonist and thus inhibits hedgehog pathway. It is under Phase II development for Basal cell nevus syndrome.

Basal Cell Nevus Syndrome: Therapeutic Assessment

This segment of the report provides insights about the different Basal Cell Nevus Syndrome drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Basal Cell Nevus Syndrome

There are approx. 5+ key companies which are developing the therapies for Basal Cell Nevus Syndrome. The companies which have their Basal Cell Nevus Syndrome drug candidates in the most advanced stage, i.e. Phase III include, PellePharm.

Phases

This report covers around 5+ 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

Basal Cell Nevus Syndrome 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
• Parenteral
• Intravenous
• Subcutaneous
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type

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

Basal Cell Nevus Syndrome: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Basal Cell Nevus Syndrome 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 Basal Cell Nevus Syndrome drugs.

Basal Cell Nevus Syndrome Report Insights

• Basal Cell Nevus Syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Basal Cell Nevus Syndrome 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 Basal Cell Nevus Syndrome drugs?
• How many Basal Cell Nevus Syndrome drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Basal Cell Nevus Syndrome?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Basal Cell Nevus Syndrome 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 Basal Cell Nevus Syndrome and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Roche
• PellePharm
• Palvella Therapeutics

Key Products

• Vismodegib
• Patidegib
• QTORIN

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