Page 46 - Read Online
P. 46
Kim et al. Rare Dis Orphan Drugs J. 2026;5:6 Page 7 of 8
5. Robertson KA, Nalepa G, Yang FC, et al. Pilot phase II trial of imatinib mesylate in neurofibromatosis type 1 patients with plexiform
neurofibromas. Lancet Oncol. 2012;13:1218-24. DOI
6. Widemann BC, Babovic-Vuksanovic D, Dombi E, et al. Phase II trial of pirfenidone in children and young adults with neurofibromatosis
type 1 and progressive plexiform neurofibromas. Pediatr Blood Cancer. 2014;61:1598-602. DOI PubMed PMC
7. Widemann BC, Dombi E, Gillespie A, et al. Phase 2 randomized, flexible crossover, double-blinded, placebo-controlled trial of the
farnesyltransferase inhibitor tipifarnib in children and young adults with neurofibromatosis type 1 and progressive plexiform
neurofibromas. Neuro Oncol. 2014;16:707-18. DOI PubMed PMC
8. Shih CS. Study of sutent®/sunitinib (SU11248) in subjects with NF-1 plexiform neurofibromas; 2018. Available from: https://clinicaltria
ls.gov/study/NCT01402817 [Last accessed on 11 Feb 2026].
9. Gorai S, Rathore G, Das K. Selumetinib-A comprehensive review of the new FDA-approved drug for neurofibromatosis. Indian
Dermatol Online J. 2024;15:701-5. DOI PubMed PMC
10. Akbarzadeh M, Vaez-Gharamaleki Y, Hosseini M. Mirdametinib approval for neurofibromatosis type 1 with inoperable plexiform
neurofibromas: the journey of MEK inhibitors. ESMO Rare Cancers. 2025;3:100015. DOI
11. Solares I, Viñal D, Morales-Conejo M, Rodriguez-Salas N, Feliu J. Novel molecular targeted therapies for patients with
neurofibromatosis type 1 with inoperable plexiform neurofibromas: a comprehensive review. ESMO Open. 2021;6:100223. DOI
PubMed PMC
12. Gross AM, Wolters PL, Dombi E, et al. Selumetinib in children with inoperable plexiform neurofibromas. N Engl J Med.
2020;382:1430-42. DOI
13. FDA. FDA approves selumetinib for adults with neurofibromatosis type 1 with symptomatic, inoperable plexiform neurofibromas.
Available from: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-selumetinib-adults-neurofibromatosis-ty
pe-1-symptomatic-inoperable-plexiform [Last accessed on 11 Feb 2026].
14. Chen AP, Coyne GO, Wolters PL, et al. Efficacy and safety of selumetinib in adults with neurofibromatosis type 1 and symptomatic,
inoperable plexiform neurofibromas (KOMET): a multicentre, international, randomised, placebo-controlled, parallel, double-blind,
phase 3 study. Lancet. 2025;405:2217-30. DOI
15. Moertel CL, Hirbe AC, Shuhaiber HH, et al. ReNeu: a pivotal, phase IIb trial of mirdametinib in adults and children with symptomatic
neurofibromatosis type 1-associated plexiform neurofibroma. J Clin Oncol. 2025;43:716-29. DOI PubMed PMC
16. FDA. FDA approves mirdametinib for adult and pediatric patients with neurofibromatosis type 1 who have symptomatic plexiform
neurofibromas not amenable to complete resection. Available from: https://www.fda.gov/drugs/resources-information-approved-drugs/fd
a-approves-mirdametinib-adult-and-pediatric-patients-neurofibromatosis-type-1-who-have-symptomatic [Last accessed on 11 Feb 2026].
17. Mol I, Bell T, Hu Y, Langseth A, Weber M, Moradian H. CO122 indirect treatment comparison (ITC) of mirdametinib and selumetinib
for the treatment of children with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN). Value Health. 2025;28:S45.
DOI
18. Gross AM, Dombi E, Wolters PL, et al. Long-term safety and efficacy of selumetinib in children with neurofibromatosis type 1 on a
phase 1/2 trial for inoperable plexiform neurofibromas. Neuro Oncol. 2023;25:1883-94. DOI PubMed PMC
19. Healx Limited. A phase 2, open-label, single arm, non-controlled, single-stage study to evaluate the safety and effects of HLX-1502 in
patients with plexiform neurofibroma and neurofibromatosis type 1 (INSPIRE-NF1). Available from: https://clinicaltrials.gov/study/NC
T06541847 [Last accessed on 11 Feb 2026].
20. Korf B. Phase II study of binimetinib in children and adults with NF1 Plexiform neurofibromas (NF108-BINI). Available from: https://cl
inicaltrials.gov/study/NCT03231306 [Last accessed on 11 Feb 2026].
21. Fisher MJ, Shih CS, Rhodes SD, et al. Cabozantinib for neurofibromatosis type 1-related plexiform neurofibromas: a phase 2 trial. Nat
Med. 2021;27:165-73. DOI PubMed PMC
22. Dhall G. Study of cabozantinib with selumetinib for plexiform neurofibromas (NF113). Available from: https://clinicaltrials.gov/study/N
CT06502171 [Last accessed on 11 Feb 2026].
23. Sundby RT, Szymanski JJ, Pan AC, et al. Early detection of malignant and premalignant peripheral nerve tumors using cell-free DNA
fragmentomics. Clin Cancer Res. 2024;30:4363-76. DOI
24. Bai RY, Shi J, Liu J, et al. Development of an adeno-associated virus vector for gene replacement therapy of NF1-related tumors. Nat
Commun. 2025;16:8594. DOI PubMed PMC
25. Mueller S, Reddy AT, Dombi E, et al. NFB-17. MEK inhibitor binimetinib shows clinical activity in children with neurofibromatosis
type 1- associated plexiform neurofibromas: a report from pnoc and the nf clinical trials consortium. Neuro Oncol. 2020;22:iii420-1.
DOI PMC
26. Klesse LJ, Jordan JT, Radtke HB, et al. The use of MEK inhibitors in neurofibromatosis type 1-associated tumors and management of
toxicities. Oncologist. 2020;25:e1109-16. DOI PubMed PMC

