European Commission grants SpringWorks company mirdametinib orphan drug for neurofibromatosis type 1

The European Commission (EC) has granted mirdametinib (formerly PD-0325901) the right to treat neurofibromatosis type 1 (NF1), springWorkscompany(http:// announcedAbout mirdametinib
mirdametinib is an oral small molecule MEK1 and MEK2 inhibitorIn the United States,FDA(http:// prior to the, mirdametinib has been granted the orphan drug for NF1 treatment and fast-track eligibility for the treatment of NF1-related, unsurgical clumps of neurofibroma to patients aged 2 yearsmirdametinib is an oral MEK1 and MEK2 small molecule inhibitorMEK proteins play a key role in the MAPK pathway, a key signalnetwork for cell growth and survival, playing a central role in a variety of tumor and rare disease indicationsMirdametinib has been evaluated in several Phase I and Phase II clinical studies, with more than 200 subjects receiving treatmentIn a Phase II study involving 19 adolescent and adult patients with no surgery, symptomatic or growth PN, mirdametinib was treated for 12 weeks and 8 patients achieved objective remission (ORR-42%)In this study, mirdametinib was well toleratedSpringWorks expects to launch the ReNeu study of mirdametinib, an open label, one-arm IIb phase study that will enroll children and adults with NF1-PN in the third quarter of 2019 　　Considering that the MAPK pathway plays a key role in the growth and proliferation of multiple types of tumors, SpringWorks also plans to evaluate mirdametinib in combination with other anticancer drugs for the treatment of a wide range of solid tumors