NMC Guidelines For Competency-Based Training Programme For DM Medical Genetics
Neurofibromatosis Type 1 Market Set To Witness Significant Growth During The Study Period (2020–2034) Driven By Advances In TreatmentDelveInsight
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.
Neurofibromatosis Type 1 Report Metrics
Details
Study Period
2020–2034
Neurofibromatosis Type 1 Report Coverage
7MM [The United States, the EU-4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]
Neurofibromatosis Type 1 Market Size in 2024
USD 430 Million
Key Neurofibromatosis Type 1 Companies
Fosun Pharmaceutical, Healx, NFlection Therapeutics, Pasithea Therapeutics, AstraZeneca, Merck, SpringWorks Therapeutics, and others
Key Neurofibromatosis Type 1 Therapies
FCN-159, HLX-1502, NFX-179, PAS-004, KOSELUGO, GOMEKLI, and others
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
1
KEY INSIGHTS
2
REPORT INTRODUCTION
3
EXECUTIVE SUMMARY OF NF1
4
EPIDEMIOLOGY AND MARKET METHODOLOGY
5
NF1-PN MARKET OVERVIEW AT A GLANCE
5.1
MARKET SHARE (%) DISTRIBUTION OF NF1 BY THERAPIES IN 2020 IN THE 7MM
5.2
MARKET SHARE (%) DISTRIBUTION OF NF1 BY THERAPIES IN 2034 IN THE 7MM
6
KEY EVENTS
7
DISEASE BACKGROUND AND OVERVIEW
7.1
INTRODUCTION
7.2
GENETIC MUTATIONS
7.3
CLINICAL MANIFESTATIONS
7.4
CAUSES
7.5
DIAGNOSIS
7.5.1
Differential Diagnosis
7.5.2
Diagnostic Algorithm
7.5.3
Diagnostic Guidelines
7.6
TREATMENT AND MANAGEMENT
7.6.1
Treatment Algorithm
8
EPIDEMIOLOGY AND PATIENT POPULATION
8.1
KEY FINDINGS
8.2
ASSUMPTION AND RATIONALE
8.3
TOTAL DIAGNOSED PREVALENT CASES OF NF1 IN THE 7MM
8.4
THE UNITED STATES
8.4.1
Total Diagnosed Prevalent Cases of NF1 in the United States
8.4.2
Diagnosed Prevalent Cases of NF1 Manifestations in the United States
8.4.3
Age-specific Diagnosed Prevalent Cases of NF1 in the United States
8.4.4
Severity-specific Diagnosed Prevalent Cases of cNFs in the United States
8.4.5
Age-specific Diagnosed Prevalent Cases of NF1-PN in the United States
8.4.6
NF1-PN Cases by Clinical Symptoms in the United States
8.4.7
NF1-PN Cases Eligible for Surgery in the United States
8.5
EU4 AND THE UK
8.5.1
Total Diagnosed Prevalent Cases of NF1 in EU4 and the UK
8.5.2
Diagnosed Prevalent Cases of NF1 Manifestations in EU4 and the UK
8.5.3
Age-specific Diagnosed Prevalent Cases of NF1-PN in EU4 and the UK
8.5.4
Severity-specific Diagnosed Prevalent Cases of cNFs in EU4 and the UK
8.5.5
Age-specific Diagnosed Prevalent Cases of NF1-PN in EU4 and the UK
8.5.6
NF1-PN Cases by Clinical Symptoms in EU4 and the UK
8.5.7
NF1-PN Cases Eligible for Surgery in EU4 and the UK
8.6
JAPAN
8.6.1
Total Diagnosed Prevalent Cases of NF1 in Japan
8.6.2
Diagnosed Prevalent Cases of NF1 Manifestations in Japan
8.6.3
Age-specific Diagnosed Prevalent Cases of NF1-PN in Japan
8.6.4
Severity-specific Diagnosed Prevalent Cases of cNFs in Japan
8.6.5
Age-specific Diagnosed Prevalent Cases of NF1-PN in Japan
8.6.6
NF1-PN Cases by Clinical Symptoms in Japan
8.6.7
NF1-PN Cases Eligible for Surgery in Japan
9
PATIENT JOURNEY
10
MARKETED DRUGS
10.1
KEY CROSS COMPETITION
10.2
KOSELUGO (SELUMETINIB): ASTRAZENECA AND MERCK
10.2.1
Product Description
10.2.2
Regulatory Milestones
10.2.3
Other Development Activities
10.2.4
Clinical Development
10.2.5
Safety and Efficacy
11
EMERGING DRUGS
11.1
KEY CROSS COMPETITION
11.2
MIRDAMETINIB (PD-0325901): SPRINGWORKS THERAPEUTICS
11.2.1
Product Description
11.2.2
Other Developmental Activities
11.2.3
Clinical Development
11.2.4
Safety and Efficacy
11.3
FCN-159: FOSUN PHARMACEUTICAL
11.3.1
Product Description
11.3.2
Clinical Development
11.3.3
Safety and Efficacy
11.4
HLX-1502: HEALX
11.4.1
Product Description
11.4.2
Other Developmental Activities
11.4.3
Clinical Development
11.5
NFX‑179: NFlection Therapeutics
11.5.1
Product Description
11.5.2
Clinical Development
11.5.3
Safety and Efficacy
12
NF1-PN: SEVEN MAJOR MARKET ANALYSIS
12.1
KEY FINDINGS
12.2
MARKET OUTLOOK
12.3
CONJOINT ANALYSIS
12.4
KEY MARKET FORECAST ASSUMPTIONS
12.4.1
Cost Assumptions and Rebates
12.4.2
Pricing Trends
12.4.3
Analogue Assessment
12.4.4
Launch Year and Therapy Uptakes
12.5
TOTAL MARKET SIZE OF NF1 IN THE 7MM
12.6
THE UNITED STATES
12.6.1
Total Market Size of NF1 in the US
12.6.2
Market Size of NF1 by Therapies in the US
12.7
EU4 AND THE UK
12.7.1
Total Market Size of NF1 in the EU4 and the UK
12.7.2
Market Size of NF1 by Therapies in EU4 and the UK
12.8
JAPAN
12.8.1
Total Market Size of NF1 in Japan
12.8.2
Market Size of NF1 by Therapies in Japan
13
UNMET NEEDS
14
SWOT ANALYSIS
15
KOL VIEWS
16
MARKET ACCESS AND REIMBURSEMENT
16.1
UNITED STATES
16.1.1
Centre for Medicare and Medicaid Services (CMS)
16.2
EU4 AND THE UK
16.2.1
Germany
16.2.2
France
16.2.3
Italy
16.2.4
Spain
16.2.5
United Kingdom
16.3
JAPAN
16.3.1
MHLW
16.4
MARKET ACCESS AND REIMBURSEMENT OF NF1
17
APPENDIX
17.1
BIBLIOGRAPHY
17.2
REPORT METHODOLOGY
18
DELVEINSIGHT CAPABILITIES
19
DISCLAIMER
20
ABOUT DELVEINSIGHT
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Treating Neurofibromatosis Type 1 With Gomekli
Gomekli (mirdametinib) is the first treatment that is FDA approved for both adults and children with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas (PN). Selumetinib, a drug that works similarly to mirdametinib, is only approved for children with this condition.
Mirdametinib is a medicine that helps people with NF1 who have PN, which are tumors that can't be fully removed by surgery. Gomekli works by blocking specific enzymes in the body called mitogen-activated kinases 1 and 2 (MEK1 and MEK2). These enzymes send signals that help tumors grow. By stopping these signals, Gomekli slows tumor growth and can shrink tumors. Since surgery isn't always an option (because tumors grow around important body parts like nerves and organs), Gomekli gives people another way to manage their condition.
Your health care provider will decide the correct dose of Gomekli based on your height and weight.
Gomekli is taken by mouth twice a day, about 12 hours apart. The treatment follows a 28-day cycle, where you take Gomekli twice daily for 21 days, followed by seven days without Gomekli.
If you have trouble swallowing Gomekli capsules, the Gomekli tablets are grape-flavored and can be mixed with water to make an oral liquid. To prepare the liquid, add the prescribed number of tablets to a dosing cup with 5 to 10 milliliters of water. Gently swirl for two to four minutes until the tablets dissolve completely. The liquid will look white and cloudy. Drink it immediately from the cup or use an oral syringe. After drinking, add 5 to 10 milliliters of water to the cup, swirl to mix any leftover medicine, and drink it to ensure the full dose is taken. Throw away any leftover liquid that is not taken within 30 minutes of mixing.
The ReNeu study evaluated the effectiveness and safety of Gomekli in 111 people 2 years old and older with NF1 with PN who had tumors that made them uncomfortable or affected their movement and that could not be removed by surgery. People in the study took Gomekli twice a day for 21 days, followed by seven days off, in repeating 28-day cycles. The dose of Gomekli was based on height and body weight. People continued treatment for up to 24 cycles or until their disease worsened or they had side effects that required stopping treatment. To measure effectiveness, people in the study received MRI scans to track tumor size.
A total of 58 adults and 56 children received Gomekli. The median (middle) age of adults was 35 (and ranged from 18 to 69); 64% were female, 85% were White, 9% were Black or African American, 3.4% were Asian, 3.4% were other races; and 1.7% were Hispanic or Latino. The median age of children was 10 (and ranged from 2 to 17); 54% were female; 66% were White, 20% were Black or African American, 9% were other races, 3.6% were Asian, 1.8% were Native American or Alaska Native; and 14% were Hispanic or Latino. More than half of the people in the study had progressive PN (tumors that have demonstrated continuous growth) at the start of the study (adults, 53%; children, 62%) and some had previously received treatment with a MEK inhibitor (adults, 7%; children, 11%). Prior surgery was reported in 69% of adults and 36% of children. People in the study commonly had other complications, including pain (adults, 90%; children, 70%), disfigurement or major deformity (adults, 52%; children, 50%), and problems with movement and coordination (adults, 40%; children, 27%).
Results of the ReNeu study showed that 41% of adults and 52% of children experienced at least a 20% reduction in their tumor size. Additionally, some people saw an even greater shrinkage of their tumors, with 62% of adults and 52% of children having more than a 50% reduction in their tumor size. Among those who responded, tumor shrinkage started as early as four months. The median time to first tumor shrinkage was 7.8 months for adults and 7.9 months for children. Treatment response lasting at least 12 months was reported in 88% of adults and 90% of children. Treatment response lasting at least 24 months was reported in 50% of adults and 48% of children.
Your results may differ from what was seen in clinical studies.
Based on the ReNeu study, Gomekli appears safe and effective for adults and children with NF1 who have PNs that cannot be removed by surgery. Experts in the management of NF1 suggest that MEK inhibitors play an important role in treating people with NF1.
People who respond to Gomekli can have tumor shrinkage in as soon as four months. In the ReNeu study, the median time to first tumor shrinkage was between seven and eight months for adults and children. However, some people did not start to see tumor shrinkage until about 19 months after starting Gomekli.
Your results may differ from what was seen in clinical studies.
Your health care provider will check your progress using MRI scans to see if your tumors are shrinking. You may also feel less pain or have improved movement if the tumors were causing discomfort.
In the ReNeu study, people taking mirdametinib had less pain and a better quality of life from the beginning of the study to cycle 13. These improvements started as early as cycle 3 and lasted throughout the study.
By cycle 5 in adults:
By cycle 13 in adults:
By cycle 5 in children:
By cycle 13 in children:
Also, 71% of adults and 79% of children said they felt at least a little better overall by the end of the study.
Your results may differ from what was seen in clinical studies.
The most common side effects of Gomekli in adults who take the medicine include rash; nausea; vomiting; diarrhea; muscle, bone, or joint pain; and tiredness.
The most common side effects of Gomekli in children who take the medicine include rash; abdominal pain; nausea; vomiting; diarrhea; muscle, bone, or joint pain; headache; skin redness, swelling, or pain around the fingernails or toenails; and heart problems.
If you have nausea or vomiting, try eating smaller meals slowly. You can also try avoiding foods that are fried, greasy, or sweet; eating bland, low-fat foods like crackers, toast, and rice; and avoiding lying down after eating. If you experience diarrhea or vomiting, make sure to drink plenty of fluids to stay hydrated.
Getting plenty of rest and drinking enough water can help with headaches. Headaches and muscle or joint aches and pains can also be managed with over-the-counter pain-relieving medications such as acetaminophen or ibuprofen.
Depending on the severity of your side effects, your health care provider may pause, reduce the dose, or permanently discontinue Gomekli.
These are not all of the possible side effects of Gomekli. Talk with your health care provider if you are having symptoms that bother you. You can also report side effects to the FDA at 800-FDA-1088 (800-332-1088).
Among 133 people who received Gomekli in clinical trials, 62% were exposed for one year or longer, 38% were exposed for two years or longer, and 12% were exposed for three years or longer. It is not known what long-term side effects may occur with extended therapy using Gomekli.
Gomekli can cause serious eye, heart, and skin problems.
Before you start taking Gomekli, your health care provider should check your vision with a comprehensive eye exam. Your health care provider will also monitor your vision regularly during treatment and check for any new or worsening vision problems, such as blurred vision.
Before you start taking Gomekli, your health care provider should also check your heart's pumping ability (ejection fraction) using an echocardiogram. Your ejection fraction should be monitored every three months during the first year of treatment with Gomekli, and then as needed.
Gomekli may cause harm to a fetus. Your health care provider will do a pregnancy test before starting Gomekli treatment if you are able to become pregnant. If you are able to become pregnant, you should use effective birth control during treatment and for six weeks after the last dose. If you are able to get someone pregnant, you should use effective birth control during treatment and for three months after the last dose.
Yes. There is a cost savings and support program offered by the drugmaker that may allow you to pay $0 for your prescription. Whether you are eligible depends on whether you have prescription insurance and what type of insurance you have. You can find out more at www.Gomekli.Com/patient-support-program.
FDA Approves Mirdametinib For Neurofibromatosis Type 1 With Plexiform Neurofibromas
Adult and pediatric patients with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas that are not amenable to complete surgical resection are indicated in the approval.
Mirdametinib (Gomekli; SpringWorks Therapeutics), a kinase inhibitor, for the treatment of adult and pediatric patients aged 2 years and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) and are not seeking complete surgical resection, received FDA approval.1
The trial showed improvements in PN volume, pain severity, and quality of life.
Image Credit: Pawel - stock.Adobe.Com
The approval was based on the findings from the ReNeu (NCT03962543) trial, which is the largest multicenter study of NF1 PN conducted to date.2 Mirdametinib demonstrated a statistically significant overall response rate (ORR) according to blinded independent central review. The trial showed durable reductions in PN volume, along with significant improvements in pain severity, pain interference, and health-related quality of life (HRQOL). Additionally, the safety profile of mirdametinib was manageable in both adults and children.
"We know that plexiform neurofibroma can impact QOL in a number of different ways," study author Christopher L. Moertel, MD, medical director of the Pediatric Neuro-Oncology and Neurofibromatosis Programs at the University of Minnesota, said in an interview with CancerNetwork®.3 "Seeing that in a statistically significant [way] was quite gratifying. Likewise, [for] pain relief—one of the main reasons people present with plexiform neurofibroma and desire treatment is because of the pain it causes—we saw statistically significant relief of pain. Those things, all together, are great."
The multicenter, single-arm study evaluated the efficacy and safety of mirdametinib in 114 patients (58 adults and 56 pediatric patients) with symptomatic, inoperable NF1-associated PN.2 These tumors, often deeply embedded within critical structures, pose significant challenges for complete surgical removal without substantial risk.
The primary efficacy measure was the confirmed ORR, defined as the percentage of patients experiencing a complete or partial response (≥ 20% reduction in PN volume). Results showed a confirmed ORR of 41% in adult patients (95% CI, 29-55) and 52% in pediatric patients (95% CI, 38-65), using volumetric MRI analysis (P < .001).
Common adverse effects reported in adult patients included rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue, with increased creatine phosphokinase levels being the most frequently observed grade 3 or 4 laboratory abnormality.1 Pediatric patients experienced similar adverse effects, along with additional concerns such as abdominal pain, headache, and left ventricular dysfunction. Notably, serious risks associated with mirdametinib include left ventricular dysfunction and ocular toxicity, which may require dosage adjustments or discontinuation.
The FDA granted mirdametinib several designations, including priority review, fast track designation, and orphan drug designation, in response to the urgent need for treatments targeting rare pediatric diseases. The approval process was further expedited by the use of the Assessment Aid, a voluntary submission tool designed to streamline FDA evaluations. Additionally, a priority review voucher was issued as part of the regulatory incentives aimed at encouraging the development of treatments for rare pediatric conditions.
The FDA approval of mirdametinib for the management of NF1-associated plexiform neurofibromas offers another treatment option to patients who previously had limited alternatives. While safety considerations remain important, the drug's profile and regulatory support show its potential to provide meaningful pain relief, improvements in quality of life, and durable tumor reductions.
References
1. FDA approves mirdametinib for adult and pediatric patients with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection. News release. FDA. February 11, 2025. Https://www.Fda.Gov/drugs/resources-information-approved-drugs/fda-approves-mirdametinib-adult-and-pediatric-patients-neurofibromatosis-type-1-who-have-symptomatic
2. Moertel CL, Hirbe AC, Shuhaiber HH, et al. ReNeu: A pivotal phase 2b trial of mirdametinib in children and adults with neurofibromatosis type 1 (NF1)-associated symptomatic inoperable plexiform neurofibroma (PN). JCO 42, 3016-3016(2024). Doi:10.1200/JCO.2024.42.16_suppl.3016
3. Moertel CL. Objective data highlight substantial benefits with mirdametinib in NF1-PN. Cancer Network. February 12, 2025. Accessed February 12, 2025. Https://www.Cancernetwork.Com/view/objective-data-highlight-substantial-benefits-with-mirdametinib-in-nf1-pn
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