Neurofibromatoses - Pipeline Insight, 2025

This “Neurofibromatoses - Pipeline Insight, 2025” report provides comprehensive insights about 10+ companies and 12+ pipeline drugs in Neurofibromatoses 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

Neurofibromatoses: Understanding

Neurofibromatoses: Overview

Neurofibromatoses (NF) are a group of genetic disorders primarily characterized by the development of benign tumors along nerves in the skin, brain, and other parts of the body. There are three main types of neurofibromatosis: NF1, NF2, and schwannomatosis. NF1, also known as von Recklinghausen disease, is the most common type, affecting approximately 1 in 3,000 individuals. It is typically diagnosed in childhood and marked by multiple café-au-lait spots, Lisch nodules in the eyes, and neurofibromas, which are non-cancerous growths on nerves. NF2 is rarer and often manifests in early adulthood, characterized by the development of benign tumors on the nerves that carry sound and balance information from the inner ear to the brain, leading to hearing loss, balance issues, and sometimes facial paralysis. Schwannomatosis is the rarest form, involving multiple schwannomas (tumors of the nerve sheath) without the vestibular nerve tumors seen in NF2, often causing significant pain and neurological problems.

The genetic basis of neurofibromatoses involves mutations in specific genes responsible for regulating cell growth. In NF1, the mutation occurs in the NF1 gene on chromosome 17, which encodes neurofibromin, a protein that helps control cell proliferation. Loss of this function leads to unchecked cell growth and tumor formation. NF2 is caused by mutations in the NF2 gene on chromosome 22, which encodes merlin, another tumor suppressor protein. In schwannomatosis, the genetic mutations are more complex and less well understood but often involve the SMARCB1 and LZTR1 genes. These mutations disrupt normal cellular functions, leading to the development of tumors in the nervous system.

Management of neurofibromatoses is typically multidisciplinary, involving regular monitoring and a variety of treatments aimed at managing symptoms and complications. This can include surgical removal of problematic tumors, radiation therapy, and medication to control pain and other symptoms. Genetic counseling is crucial for affected individuals and their families to understand the hereditary nature of the disorders and the associated risks. Ongoing research aims to improve understanding of the molecular mechanisms underlying these conditions and to develop more effective treatments. 

"Neurofibromatoses- Pipeline Insight, 2025" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Neurofibromatoses pipeline landscape is provided which includes the disease overview and Neurofibromatoses treatment guidelines. The assessment part of the report embraces, in depth Neurofibromatoses 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, Neurofibromatoses 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 Neurofibromatoses R&D. The therapies under development are focused on novel approaches to treat/improve Neurofibromatoses.

Neurofibromatoses Emerging Drugs Chapters

This segment of the Neurofibromatoses 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.

Neurofibromatoses Emerging Drugs

FCN-159: Shanghai Fosun Pharmaceutical

FCN-159 tablet is an innovative small molecule chemical drug self-developed by Fosun Pharma, which is intended to be used primarily for the treatment of advanced solid tumors, eurofibromatosis type 1, histiocytic neoplasms and arteriovenous malformations. It is a MEK1/2 selective inhibitor that can inhibit tumor proliferation caused by abnormal RAS pathway. Currently, the drug is in Phase III stage of its clinical trial for the treatment of Neurofibromatoses.

• PAS-004: Pasithea Therapeutics Corp.

PAS-004 is a small molecule allosteric inhibitor of MEK 1/2, which are dual-specificity protein kinases, in the MAPK signaling pathway. The MAPK pathway has been implicated in a variety of diseases, as it functions to drive cell proliferation, differentiation, survival and a variety of other cellular functions that, when abnormally activated, are critical for the formation and progression of tumors, fibrosis and other diseases. Currently, the drug is in Phase I stage of its clinical trial for the treatment of Neurofibromatoses.

Neurofibromatoses: Therapeutic Assessment

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

Major Players in Neurofibromatoses

• There are approx. 10+ key companies which are developing the therapies for Neurofibromatoses. The companies which have their Neurofibromatoses drug candidates in the most advanced stage, i.e. Phase III include, Shanghai Fosun Pharmaceutical.

Phases

The report covers around 12+ 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

Neurofibromatoses 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.

Neurofibromatoses: Pipeline Development Activities

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

Neurofibromatoses Report Insights

• Neurofibromatoses Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Shanghai Fosun Pharmaceutical
• Pasithea Therapeutics Corp.
• Recursion Pharmaceuticals Inc.
• NFlection Therapeutics, Inc.
• Healx

Key Products

• FCN-159
• PAS-004
• REC-2282
• NFX-179
• HLX-1502

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