Neurofibromatosis Type 1 - Pipeline Insight, 2025

This “Neurofibromatosis Type 1 - Pipeline Insight, 2025” report provides comprehensive insights about 8+ companies and 10+ pipeline drugs in Neurofibromatosis Type 1 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.

Neurofibromatosis Type 1: Understanding

Neurofibromatosis Type 1: Overview

Neurofibromatosis Type 1 (NF1), also known as von Recklinghausen disease, is a genetic disorder that primarily affects the nervous system and is characterized by the development of multiple benign tumors called neurofibromas. These tumors arise from the nerve tissue and can grow along any nerve in the body. Individuals with NF1 typically present with a variety of physical features, including distinctive café-au-lait spots (light brown skin patches) and axillary freckling (freckles in the armpit area), which are often among the first clinical signs of the condition. These skin manifestations, in combination with neurofibromas, form the hallmark diagnostic criteria for NF1. The pathophysiology of neurofibromatosis type 1 (NF1) is primarily linked to mutations in the NF1 gene, which encodes neurofibromin, a protein that functions as a negative regulator of the RAS signaling pathway.

The etiology of neurofibromatosis type 1 (NF1) is primarily linked to mutations in the NF1 gene, located on chromosome 17. This gene encodes neurofibromin, a protein that acts as a tumor suppressor by regulating the RAS signaling pathway, which is crucial for cell growth and division. Approximately 50% of NF1 cases arise from spontaneous mutations, meaning they occur without a family history of the disorder, while the remaining cases are inherited in an autosomal dominant manner. The mutation rate for pathogenic variants in the NF1 gene is notably high, over 2,800 different mutations have been identified, contributing to the phenotypic variability observed among individuals with NF1. These mutations can lead to a nonfunctional version of neurofibromin, resulting in uncontrolled cellular proliferation and the development of neurofibromas and other associated tumors.

The clinical presentation of neurofibromatosis type 1 (NF1) varies widely, affecting multiple systems of the body. Key features include multiple café-au-lait spots, which are light brown skin lesions that appear in early childhood and increase over time, and freckling in areas like the armpits and groin. Individuals with NF1 often develop neurofibromas, benign tumors on peripheral nerves, which may increase in number and sometimes cause discomfort. Lisch nodules, small benign growths on the iris, and optic pathway gliomas, which can affect vision, are also common. Skeletal abnormalities like scoliosis and pseudoarthrosis, as well as cognitive challenges such as learning disabilities and ADHD, are frequently seen. Symptoms often worsen during puberty or hormonal changes, making regular monitoring essential. The progression of NF1 requires ongoing medical evaluation to manage tumor growth, cognitive development, and other potential complications, highlighting the need for a multidisciplinary care approach.

The treatment of neurofibromatosis type 1 (NF1) focuses on managing the symptoms and complications associated with the condition, as there is currently no cure. Because NF1 affects multiple systems in the body, a multidisciplinary approach is essential for optimal care. Regular monitoring by healthcare professionals is crucial to assess the progression of the disease and address emerging issues, as symptoms can vary greatly among individuals. Surgical intervention may be necessary to remove problematic neurofibromas, which can cause pain, disfigurement, or other complications, or to address skeletal abnormalities such as scoliosis or pseudoarthrosis. In recent years, targeted therapies have become a promising treatment option, with selumetinib, a MEK inhibitor, being approved for the treatment of inoperable plexiform neurofibromas in children. Clinical studies have shown that this therapy can lead to significant tumor shrinkage, offering a new avenue for patients who are not candidates for surgery. In addition to targeted treatments, symptomatic care plays an important role in managing the condition.

'Neurofibromatosis Type 1- Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Neurofibromatosis Type 1 pipeline landscape is provided which includes the disease overview and Neurofibromatosis Type 1 treatment guidelines. The assessment part of the report embraces, in depth Neurofibromatosis Type 1 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, Neurofibromatosis Type 1 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 Neurofibromatosis Type 1 R&D. The therapies under development are focused on novel approaches to treat/improve Neurofibromatosis Type 1.

Neurofibromatosis Type 1 Emerging Drugs Chapters

This segment of the Neurofibromatosis Type 1 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.

Neurofibromatosis Type 1 Emerging Drugs

FCN-159: Shanghai Fosun Pharmaceutical

FCN-159 is a novel, proprietary and highly potent inhibitor of mitogen-activated protein kinase enzymes (MEK). MEK is a key member in the RAS/RAF/MEK/ERK pathway, which regulates several key cellular activities including proliferation, differentiation, migration, survival and angiogenesis. Dysregulation of this pathway frequently occurs in many types of cancer, in particular through mutations in BRAF, KRAS and NRAS. Currently, the drug is in Phase III stage of its clinical trial for the treatment of Neurofibromatosis Type 1.

NFX‑179: NFlection Therapeutics

NFX‑179 is an investigational mitogen-activated protein kinase (MEK) inhibitor. NFX‑179 is a soft (metabolically labile) drug, which, when formulated as NFX‑179 Gel for topical application, is designed to concentrate at the dermal site of action but degrade in systemic circulation, thereby significantly reducing side effects routinely seen with systemically available MEK inhibitors. Currently, the drug is in Phase II stage of its clinical trial for the treatment of Neurofibromatosis Type 1.

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 the Phase I stage of development to treat Neurofibromatosis Type 1.

Neurofibromatosis Type 1: Therapeutic Assessment

This segment of the report provides insights about the different Neurofibromatosis Type 1 drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Neurofibromatosis Type 1

• There are approx. 8+ key companies which are developing the therapies for Neurofibromatosis Type 1. The companies which have their Neurofibromatosis Type 1 drug candidates in the most advanced stage, i.e. Phase III include, Shanghai Fosun Pharmaceutical.

Phases

The report covers around 10+ 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

Neurofibromatosis Type 1 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.

Neurofibromatosis Type 1: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Neurofibromatosis Type 1 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 Neurofibromatosis Type 1 drugs.

Neurofibromatosis Type 1 Report Insights

• Neurofibromatosis Type 1 Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Neurofibromatosis Type 1 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 Neurofibromatosis Type 1 drugs?
• How many Neurofibromatosis Type 1 drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Neurofibromatosis Type 1?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Neurofibromatosis Type 1 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 Neurofibromatosis Type 1 and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Shanghai Fosun Pharmaceutical
• NFlection Therapeutics
• Pasithea Therapeutics Corp.

Novartis

• Pfizer

Key Products

• FCN-159
• NFX‑179
• PAS-004
• Trametinib
• Binimetinib

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