Neurofibromatosis Type 1 Global Market Report 2025

The neurofibromatosis type 1 (NF1) market size has grown strongly in recent years. It will grow from $8.06 billion in 2024 to $8.86 billion in 2025 at a compound annual growth rate (CAGR) of 9.9%. The growth during the historical period can be credited to factors such as increased awareness, higher healthcare spending, population growth, supportive government policies, and advancements in medical infrastructure.

The neurofibromatosis type 1 (NF1) market size is expected to see strong growth in the next few years. It will grow to $12.77 billion in 2029 at a compound annual growth rate (CAGR) of 9.6%. The growth during the forecast period can be attributed to the rising number of blood cancer cases, an increase in the incidence of plexiform tumors, a higher prevalence of nerve sheath tumors, the growing occurrence of neurofibromatosis, and the expanding use of neurofibromatosis treatments. Key trends during this period include ongoing research and development, the creation of new therapies, advancements in diagnostic techniques, tumor-specific treatments, and the development of new medications.

The rise in spontaneous (de novo) mutations is expected to drive the growth of the neurofibromatosis type 1 (NF1) market. A spontaneous (de novo) mutation refers to a genetic change that occurs for the first time in an individual, due to errors during DNA replication or environmental influences, rather than being inherited from parents. The increasing occurrence of these mutations is linked to factors such as advanced parental age, environmental exposures such as radiation and chemicals, and errors in DNA replication during cell division. Neurofibromatosis type 1 (NF1) is particularly prone to de novo mutations, as the NF1 gene experiences a high mutation rate, including large deletions, insertions, or point mutations during gametogenesis. This leads to new cases of NF1 without a family history. For example, a report published in August 2023 by the journal Molecular Biology and Evolution found 534 potential de novo mutations (DNMs) in certain families. Therefore, spontaneous (de novo) mutations are contributing to the growth of the NF1 market.

Companies in the NF1 market are introducing innovative treatments, such as kinase inhibitors, to improve targeted therapy and patient outcomes. Kinase inhibitors are drugs that block the activity of kinases, enzymes responsible for transferring phosphate groups to proteins and regulating various cellular processes. In May 2023, AstraZeneca plc, a UK-based pharmaceutical company, received approval in China for Koselugo (selumetinib), a kinase inhibitor, to treat symptomatic, inoperable plexiform neurofibromas (PN) in pediatric patients with NF1, aged three years and older. This approval expands targeted treatment options for pediatric NF1 patients, improving disease management and quality of life. The introduction of Koselugo (selumetinib) in China is in line with the growing emphasis on precision medicine, providing a non-surgical option for managing inoperable plexiform neurofibromas.

In October 2022, Pasithea Therapeutics Corp., a US-based biotechnology company, acquired AlloMek Therapeutics LLC for an undisclosed amount. This acquisition aims to advance Pasithea Therapeutics’ efforts in developing treatments for central nervous system disorders and other conditions, as well as establishing anti-depression clinics and offering business support services. AlloMek Therapeutics LLC is involved in developing therapeutics for catastrophic cardiomyopathy, congenital muscular diseases, and neurofibromatosis type 1, contributing to the expansion of NF1 treatment options.

Major players in the neurofibromatosis type 1 (NF1) market are Pfizer Inc., F. Hoffmann-La Roche Ltd, Merck & Co. Inc, Novartis AG, AstraZeneca Plc, Takeda Pharmaceutical Company Limited, Boehringer Ingelheim International GmbH, Vertex Pharmaceuticals Incorporated, Shanghai Fosun Pharmaceutical (Group) Co. Ltd, BeiGene Ltd., BioMarin Pharmaceutical Inc, Exelixis Inc., Blueprint Medicines Corporation, SpringWorks Therapeutics Inc, Onclive LLC, Healx Limited, Infixion Bioscience Limited, NFlection Therapeutics Inc, Pasithea Therapeutics Corporation, Mulberry Biotherapeutics Inc.

North America was the largest region in the neurofibromatosis type 1 (NF1) market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in neurofibromatosis type 1 (NF1) report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.

The countries covered in the neurofibromatosis type 1 (NF1) market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Neurofibromatosis type 1 (NF1) is a genetic disorder that primarily impacts the nervous system, leading to the formation of benign tumors along nerves in areas such as the skin, brain, and other parts of the body. It is caused by mutations in the NF1 gene, which results in a deficiency of neurofibromin, a protein responsible for regulating cell growth. This deficiency causes a range of symptoms, including café-au-lait spots (light brown patches on the skin), freckling in uncommon areas, learning difficulties, skeletal issues, and, in some instances, vision or nerve-related problems.

The primary treatment for neurofibromatosis type 1 (NF1) generally involves medication, surgery, radiation therapy, and other forms of treatment. Medications used in NF1 include pharmaceutical options such as MEK inhibitors, pain relievers, and antihypertensive drugs, which help to slow tumor growth, alleviate pain, and manage associated complications. The disease encompasses conditions such as plexiform neurofibromas, cutaneous neurofibromas, optic gliomas, and others. These treatments are distributed through various channels, including hospital pharmacies, retail pharmacies, and online pharmacies, and are used by different healthcare providers, including hospitals, specialty clinics, ambulatory surgical centers, and other medical facilities.

The neurofibromatosis type 1 (NF1) market research report is one of a series of new reports that provides neurofibromatosis type 1 (NF1) market statistics, including the neurofibromatosis type 1 (NF1) industry global market size, regional shares, competitors with the neurofibromatosis type 1 (NF1) market share, detailed neurofibromatosis type 1 (NF1) market segments, market trends, and opportunities, and any further data you may need to thrive in the neurofibromatosis type 1 (NF1) industry. This neurofibromatosis type 1 (NF1) market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.

The neurofibromatosis type 1 (NF1) market consists of revenues earned by entities by providing services such as medical monitoring, genetic counselling, and specialized care. The market value includes the value of related goods sold by the service provider or included within the service offering. The neurofibromatosis type 1 (NF1) market also includes sales of medical devices, MEK inhibitors, anticonvulsants, and analgesics. Values in this market are ‘factory gate’ values, that is, the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

This product will be delivered within 3-5 business days.