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Neurofibromatosis 1

8
Pipeline Programs
10
Companies
10
Clinical Trials
1 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
3
0
3
0
2
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
7100%
+ 7 programs with unclassified modality

Competitive Landscape

10 companies ranked by most advanced pipeline stage

Sharp Therapeutics
Sharp TherapeuticsPA - Pittsburgh
2 programs
1
1
SelumetinibPhase 3Small Molecule
SelumetinibPhase 1Small Molecule
MSD
MSDIreland - Ballydine
2 programs
1
1
SelumetinibPhase 3Small Molecule1 trial
SelumetinibPhase 1Small Molecule1 trial
Active Trials
NCT04590235Active Not Recruiting32Est. Aug 2026
NCT04924608Active Not Recruiting145Est. Feb 2029
KeChow Pharma
KeChow PharmaChina - ZZ
1 program
1
HL-085Phase 21 trial
Active Trials
NCT05331105Recruiting70Est. Oct 2028
NFlection Therapeutics
1 program
1
NFX-179 GelPhase 21 trial
Active Trials
NCT04435665Completed48Est. Apr 2021
Sandoz
SandozAustria - Kundl
1 program
1
TrametinibPhase 2Small Molecule1 trial
Active Trials
NCT03741101Unknown15Est. Dec 2024
Angeles Therapeutics
Angeles TherapeuticsCA - Los Angeles
1 program
1
LovastatinPhase 1Small Molecule1 trial
Active Trials
NCT00352599Completed44Est. Mar 2014
Fosun Pharma
Fosun PharmaAustralia - Sydney
3 programs
FCN-159PHASE_1_21 trial
Luvometinib TabletsPHASE_1_21 trial
Test groupPHASE_31 trial
Active Trials
NCT04954001Active Not Recruiting160Est. Mar 2025
NCT07024394Not Yet Recruiting65Est. Sep 2029
NCT05913037Active Not Recruiting167Est. Jun 2026
Utah Medical
Utah MedicalIreland - Dublin
1 program
Cheek swabN/A
Spine BioPharma
Spine BioPharmaNY - New York
1 program
Cheek swabN/A1 trial
Active Trials
NCT01776125Completed59Est. Aug 2015
Merck & Co.
Merck & Co.RAHWAY, NJ
1 program
SelumetinibPHASE_1Small Molecule

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
Fosun PharmaTest group
MSDSelumetinib
KeChow PharmaHL-085
NFlection TherapeuticsNFX-179 Gel
SandozTrametinib
Fosun PharmaLuvometinib Tablets
Fosun PharmaFCN-159
MSDSelumetinib
Angeles TherapeuticsLovastatin
Spine BioPharmaCheek swab

Clinical Trials (10)

Total enrollment: 805 patients across 10 trials

NCT05913037Fosun PharmaTest group

FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas

Start: Jun 2023Est. completion: Jun 2026167 patients
Phase 3Active Not Recruiting
NCT04924608MSDSelumetinib

Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas

Start: Nov 2021Est. completion: Feb 2029145 patients
Phase 3Active Not Recruiting
NCT05331105KeChow PharmaHL-085

HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas

Start: Oct 2021Est. completion: Oct 202870 patients
Phase 2Recruiting
NCT04435665NFlection TherapeuticsNFX-179 Gel

NFX-179 Topical Gel Treatment in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)

Start: Aug 2020Est. completion: Apr 202148 patients
Phase 2Completed
NCT03741101SandozTrametinib

Treatment of NF1-related Plexiform Neurofibroma With Trametinib

Start: Jun 2019Est. completion: Dec 202415 patients
Phase 2Unknown
NCT07024394Fosun PharmaLuvometinib Tablets

Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosis Type 1

Start: Jun 2025Est. completion: Sep 202965 patients
Phase 1/2Not Yet Recruiting
NCT04954001Fosun PharmaFCN-159

Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1

Start: Mar 2021Est. completion: Mar 2025160 patients
Phase 1/2Active Not Recruiting
NCT04590235MSDSelumetinib

A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)

Start: Dec 2020Est. completion: Aug 202632 patients
Phase 1Active Not Recruiting
NCT00352599Angeles TherapeuticsLovastatin

Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I (NF1)

Start: Sep 2009Est. completion: Mar 201444 patients
Phase 1Completed
NCT01776125Spine BioPharmaCheek swab

Genetic Evaluation of NF1 and Scoliosis Patients

Start: Aug 2010Est. completion: Aug 201559 patients
N/ACompleted

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

2 late-stage (Phase 3) programs, potential near-term approvals
1 actively recruiting trials targeting 805 patients
10 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.