Neurofibromatosis 1

5
Pipeline Programs
3
Companies
6
Clinical Trials
0
Approved Products

Pipeline by Development Stage

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

Drug Modality Breakdown

Small Molecule
2100%
+ 3 programs with unclassified modality

Competitive Landscape

3 companies ranked by most advanced pipeline stage

FP
Fosun PharmaAustralia - Sydney
3 programs
2
1
Test groupPhase 31 trial
FCN-159Phase 1/21 trial
Luvometinib TabletsPhase 1/21 trial
Active Trials
NCT04954001Active Not Recruiting160Est. Mar 2025
NCT07024394Not Yet Recruiting65Est. Sep 2029
NCT05913037Active Not Recruiting167Est. Jun 2026
AstraZeneca
AstraZenecaCAMBRIDGE, United Kingdom
1 program
1
SelumetinibPhase 1Small Molecule
M&
Merck & Co.RAHWAY, NJ
1 program
1
SelumetinibPhase 1Small Molecule3 trials
Active Trials
NCT05309668Active Not Recruiting36Est. Apr 2028
NCT04590235Active Not Recruiting32Est. Aug 2026
NCT03745989Completed30Est. Mar 2021

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
Fosun PharmaTest group
Fosun PharmaLuvometinib Tablets
Merck & Co.Selumetinib
Fosun PharmaFCN-159
Merck & Co.Selumetinib
Merck & Co.Selumetinib

Clinical Trials (6)

Total enrollment: 490 patients across 6 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
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
NCT05309668Merck & Co.Selumetinib

Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN

Start: Jan 2022Est. completion: Apr 202836 patients
Phase 1/2Active Not 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
NCT04590235Merck & Co.Selumetinib

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
NCT03745989Merck & Co.Selumetinib

Study of MK-8353 + Selumetinib in Advanced/Metastatic Solid Tumors (MK-8353-014)

Start: Feb 2019Est. completion: Mar 202130 patients
Phase 1Completed

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

1 late-stage (Phase 3) programs — potential near-term approvals
3 companies competing in this space