Neurofibromatosis Type 1 Market Set to Witness Significant Growth During the Study Period (2020–2034) Driven by Advances in Treatment | DelveInsight

The neurofibromatosis type 1 market remains niche, but awareness campaigns, increased diagnoses, and treatment adoption, along with ongoing clinical trials, will expand the NF1 therapeutics market during the forecast period (2025-2034). Unmet needs, particularly in adult populations and systemic disease management, create significant opportunities for new entrants and differentiated strategies

New York, USA, May 15, 2025 (GLOBE NEWSWIRE) — Neurofibromatosis Type 1 Market Set to Witness Significant Growth During the Study Period (2020–2034) Driven by Advances in Treatment | DelveInsight 

The neurofibromatosis type 1 market remains niche, but awareness campaigns, increased diagnoses, and treatment adoption, along with ongoing clinical trials, will expand the NF1 therapeutics market during the forecast period (2025-2034). Unmet needs, particularly in adult populations and systemic disease management, create significant opportunities for new entrants and differentiated strategies.

DelveInsight’s Neurofibromatosis Type 1 Market Insights report includes a comprehensive understanding of current treatment practices, emerging neurofibromatosis type 1 drugs, market share of individual therapies, and current and forecasted neurofibromatosis type 1 market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan].

Key Takeaways from the Neurofibromatosis Type 1 Market Report

• According to DelveInsight’s analysis, the total market size of NF1 in the 7MM in 2024 was approximately USD 430 million. This is anticipated to grow by 2034, driven by the entry of new emerging therapies. 
• Based on DelveInsight’s assessment in 2024, the 7MM had ~239K diagnosed prevalent cases of NF1. These cases are expected to increase by 2034. 
• Prominent companies working in the domain of neurofibromatosis type 1, including Fosun Pharmaceutical, Healx, NFlection Therapeutics, Pasithea Therapeutics, and others, are actively working on innovative neurofibromatosis type 1 drugs. These novel neurofibromatosis type 1 therapies are anticipated to enter the neurofibromatosis type 1 market in the forecast period and are expected to change the market.
• Some of the key neurofibromatosis type 1 therapies in the pipeline include FCN-159, HLX-1502, NFX-179, PAS-004, and others.
• In November 2024,  AstraZeneca and Merck announced positive topline results from the Phase III KOMET trial in adults with neurofibromatosis type 1 who have symptomatic, inoperable plexiform neurofibromas. 
• In October 2024, Healx announced that the US FDA granted FTD to HLX-1502 for the treatment of NF1. 

Discover which therapies are expected to grab the neurofibromatosis type 1 market share @ Neurofibromatosis Type 1 Market Report

Neurofibromatosis Type 1 Overview

Neurofibromatosis Type 1 (NF1) is the most prevalent genetic condition associated with tumor development, caused by mutations in the NF1 gene. These mutations lead to a loss of neurofibromin, a protein that normally regulates RAS signaling, resulting in the formation of plexiform neurofibromas—tumors of the peripheral nerve sheath that can significantly affect quality of life. Neurofibromatosis Type 1 is typically diagnosed during early childhood (as NF1-PN) or later in adulthood (as cNF), often based on visible indicators like café-au-lait spots or mild tissue overgrowth. 

However, plexiform neurofibromas that develop deep within the body may go unnoticed until symptoms such as pain appear, prompting the need for imaging to confirm their presence. These tumors are particularly challenging due to their tendency to infiltrate nearby tissues, their irregular structure, and high blood vessel density. They frequently occur in sensitive areas such as the head, neck, chest, or spine, making surgical intervention risky and complex. 

Early recognition, appropriate imaging, and expert evaluation are key to diagnosis. Effective management requires a multidisciplinary team, including specialists in genetics, neurology, radiology, and surgery, to address both the physical and emotional burden on children and their families.

Neurofibromatosis Type 1 Epidemiology Segmentation

The neurofibromatosis type 1 epidemiology section provides insights into the historical and current neurofibromatosis type 1 patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders.

The neurofibromatosis type 1 market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:

• Total Diagnosed Prevalent Cases of Neurofibromatosis Type 1
• Diagnosed Prevalent Cases of Neurofibromatosis Type 1 Manifestations
• Age-specific Diagnosed Prevalent Cases of Neurofibromatosis Type 1
• Severity-specific Diagnosed Prevalent Cases of cNFs
• Age-specific Diagnosed Prevalent Cases of Neurofibromatosis Type 1-PN
• Neurofibromatosis Type 1-PN Cases by Clinical Symptoms
• Neurofibromatosis Type 1-PN Cases Eligible for Surgery 

Download the report to understand which factors are driving neurofibromatosis type 1 epidemiology trends @ Neurofibromatosis Type 1 Epidemiological Insights

Neurofibromatosis Type 1 Treatment Market 

Currently, there is no definitive cure for neurofibromatosis, so care focuses on regular monitoring and addressing complications as they arise. For NF1, treatment is mainly aimed at managing symptoms and related health issues.

KOSELUGO (selumetinib), a kinase inhibitor, received FDA approval in April 2020 for use in children aged 2 and older with neurofibromatosis type 1 who have symptomatic, inoperable plexiform neurofibromas. It represents the first FDA-approved therapy specifically targeting PN, providing a new treatment pathway for this rare and challenging condition.

Data suggests that KOSELUGO’s average launch price in the EU exceeds that of the US (based on weight-adjusted dosing), potentially reflecting differences in payer expectations across regions during pricing negotiations. In the US, KOSELUGO has been introduced under value-based agreements, which may indicate greater uncertainty regarding its cost-effectiveness due to variable dosing requirements.

In February 2025, the FDA approved GOMEKLI (mirdametinib), a MEK inhibitor developed by SpringWorks, for use in adults and children aged 2 and older with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas (PN) that cannot be completely removed surgically. Alongside the approval, SpringWorks received a Priority Review Voucher (PRV) under the FDA’s Rare Pediatric Disease program. GOMEKLI was approved for Priority Review and had previously received Orphan Drug and Fast Track designations for treating NF1-associated PN.

Learn more about the neurofibromatosis type 1 treatment options @ Cure for Neurofibromatosis Type 1 

Neurofibromatosis Type 1 Emerging Drugs and Companies

Some of the drugs for NF1 in the pipeline include FCN-159 (Fosun Pharmaceutical), HLX-1502 (Healx), NFX-179 (NFlection Therapeutics), and PAS-004 (Pasithea Therapeutics), among others. 

HLX-1502 is an orally administered tablet that functions through a novel mechanism, offering a distinct investigational treatment option for patients with NF1. Healx has received clearance from the US FDA for an IND application, enabling the start of a Phase II clinical trial focused on adults with NF1 and inoperable plexiform neurofibroma.

In October 2024, the US FDA granted Fast Track Designation (FTD) to HLX-1502 for treating NF1. Additionally, in September 2023, HLX-1502 was awarded Orphan Drug Designation (ODD) for the same condition.

NFlection is developing a topical gel formulation of NFX-179, a proprietary “soft” (metabolically labile) MEK inhibitor, designed to treat cutaneous neurofibromas (cNFs) that persistently develop in patients with neurofibromatosis type 1 (NF1). In preclinical studies using an NF1 mouse model, MEK inhibitors significantly reduced neurofibroma tumor volume after two months of treatment. 

While an oral MEK inhibitor is currently approved for treating plexiform neurofibromas in NF1 patients, its systemic administration often leads to serious side effects such as severe acneiform rash, gastrointestinal issues, fatigue, edema, hypertension, cardiomyopathy, and retinal detachment. To address this, NFlection created a cosmetically appealing topical version of NFX-179 to minimize systemic exposure while targeting cNFs. When tested on human cNF explants, NFX-179 showed a dose-dependent reduction in phosphorylated ERK (pERK), a key marker of Ras/Raf/MEK/ERK pathway activity. 

In a completed Phase 2a clinical trial, the gel effectively reduced pERK levels in cNFs. More recently, NFlection completed a randomized, double-blind, placebo-controlled Phase 2b study with 199 participants, showing that NFX-179 significantly reduced cNF size compared to placebo.

The anticipated launch of these emerging therapies are poised to transform the neurofibromatosis type 1 market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the neurofibromatosis type 1 market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth.

To know more about neurofibromatosis type 1 clinical trials, visit @ Neurofibromatosis Type 1 Treatment Drugs 

Neurofibromatosis Type 1 Market Dynamics

The neurofibromatosis type 1 market dynamics are anticipated to change in the coming years. The introduction of MEK inhibitors has transformed the treatment landscape for plexiform neurofibromas, offering significant short-term clinical benefits, particularly in children, by modifying the natural growth progression of these tumors. Additionally, the burden of cutaneous neurofibromas in NF1 patients further emphasizes the need for safe and effective therapies, representing a valuable research and development opportunity in the NF1 market

Furthermore, many potential therapies are being investigated for the treatment of neurofibromatosis type 1, and it is safe to predict that the treatment space will significantly impact the neurofibromatosis type 1 market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate is expected to drive the growth of the neurofibromatosis type 1 market in the 7MM.

However, several factors may impede the growth of the neurofibromatosis type 1 market. Despite recent efforts to provide recommendations, the absence of definitive consensus guidelines for plexiform neurofibroma surveillance and predictive factors for cutaneous neurofibroma development in NF1 leads to practice variability, while the rise of MEK inhibitors such as FCN-159 may intensify market competition, and accessibility challenges—such as the large capsule size of KOSELUGO for pediatric patients—remain a key limitation.

Moreover, neurofibromatosis type 1 treatment poses a significant economic burden and disrupts patients’ overall well-being and QOL. Furthermore, the neurofibromatosis type 1 market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the neurofibromatosis type 1 market growth.

Scope of the Neurofibromatosis Type 1 Market Report

• Neurofibromatosis Type 1 Therapeutic Assessment: Neurofibromatosis Type 1 current marketed and emerging therapies
• Neurofibromatosis Type 1 Market Dynamics: Conjoint Analysis of Emerging Neurofibromatosis Type 1 Drugs
• Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
• Unmet Needs, KOL’s views, Analyst’s views, Neurofibromatosis Type 1 Market Access and Reimbursement

Discover more about neurofibromatosis type 1 drugs in development @ Neurofibromatosis Type 1 Clinical Trials

Table of Contents

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