SpringWorks Therapeutics announces FDA fast track designation for PD-0325901 for the treatment of a severe form of neurofibromatosis type 1

SpringWorks Therapeutics

3 June 2019 - SpringWorks Therapeutics today announced that the U.S. FDA has granted fast track designation for PD-0325901, an investigational, oral, small molecule inhibitor of MEK1 and MEK2, for the treatment of patients ≥ 2 years of age with neurofibromatosis type 1-associated inoperable plexiform neurofibromas that are progressing or causing significant morbidity.

In November 2018, the FDA granted orphan drug designation for PD-0325901 for the treatment of neurofibromatosis type 1. SpringWorks expects to initiate a Phase 2b single-arm, open-label study of PD-0325901 in paediatric and adult patients with NF1-PN in the third quarter of 2019.

Read SpringWorks Therapeutics press release

Michael Wonder

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Michael Wonder

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Medicine , US , Fast track