Neurofibromatosis Type 1- Pipeline Insight, 2025

DelveInsight's, "Neurofibromatosis Type 1- Pipeline Insight, 2025" report provides comprehensive insights about 10+ companies and 12+ 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.

Geography Covered:

• Global coverage

Neurofibromatosis Type 1: Understanding

Neurofibromatosis Type 1: Overview

Neurofibromatosis Type 1 is a genetic condition that causes tumors to grow along the nerves. The tumors are usually non-cancerous (benign) but may cause a range of symptoms. Neurofibromatosis type 1 (NF1) is a type of neurofibromatosis, that affects skin and nervous system (brain, spinal cord and nerves). NF1 affects how often certain cells grow in the body. This leads to the formation of benign (noncancerous) tumors. These specifically are nerve tumors called neurofibromas. Tumors form on the nerves in your brain, spinal cord and skin.

Symptoms of neurofibromatosis type 1 happen because of pressure on nerves. Common signs and symptoms of NF1 include: birthmarks known as cafe au lait spots, which are light or dark brown patches that can be anywhere on the body, soft, non-cancerous tumors on or under the skin (neurofibromas), clusters of freckles in unusual places - such as the armpits, groin and under the breast and problems with the bones, eyes and nervous system. A genetic change (mutation) to the neurofibromin 1 gene causes NF1. Neurofibromin 1 gives body instructions to create the neurofibromin protein. This protein is a tumor suppressor. It manages how often a cell divides and replicates. When the body doesn't have the proper instructions to create neurofibromin that means some cells in the body won't divide and replicate as they should. Instead, they'll make too many copies of themselves. As a result, tumors form. A tumor is an abnormal cluster of cells that form a solid mass of tissue. One can inherit the genetic change from one of the biological parents. This is an autosomal dominant pattern of inheritance. But the patient can also have this genetic change without any family history. Around half of all cases of NF1 occur spontaneously. This means that the genetic change happens randomly, and don't inherit it from anyone.

It's usually easy to diagnose NF1 in adults and older children by checking for the typical symptoms.

The doctor will diagnose neurofibromatosis type 1 after a physical exam and testing. During the exam, they'll look for signs of the condition and learn more about the symptoms. Tests may include: eye exam, hearing and balance exams, imaging tests. X-rays, CT scans or MRIs can help identify bone abnormalities, tumors in the brain or spinal cord, and very small tumors and Genetic tests to identify NF1 and NF2 are available and can be done in pregnancy before a baby is born. There's currently no cure for NF1. Treatment involves regular monitoring and treating any problems as they occur. Treatment can involve: Monitoring yearly age-appropriate checkups to assess the child's skin for new neurofibromas or changes in existing ones, Selumetinib (Koselugo) is a treatment for plexiform neurofibroma in children. Tumor removal surgery, Chemotherapy for tumors that develop into cancer, Surgery or support (braces) for bone growth abnormalities, Medications to treat tumor growth (selumetinib), Medications to manage symptoms (like ADHD). Researchers are testing gene therapies for NF1. Potential new treatments could include replacing the NF1 gene to restore the function of neurofibromin.

"Neurofibromatosis Type 1- Pipeline Insight, 2025" report by DelveInsight 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, II/III 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

• NFX-179: Nflection Therapeutics, Inc

NFX-179 is an investigational mitogen-activated protein kinase 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. NFlection is developing NFX-179 Gel for the treatment of cutaneous neurofibromas in people with neurofibromatosis type 1 and has received Orphan Designation in the United States and European Union for this indication. Currently, the drug is in the Phase II stage of its development for the treatment of 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. 10+ 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. Preregistration include, SpringWorks Therapeutics, Inc.

• Phases

DelveInsight's 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

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

• Intravenous
• Subcutaneous
• Oral
• Intramuscular
• Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal antibody
• Small molecule
• Peptide
• 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 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

• SpringWorks Therapeutics, Inc
• Nflection Therapeutics, Inc
• Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
• Pasithea Therapeutics Corp

Key Products

• Mirdametinib
• NFX-179
• FCN-159
• PAS-004