FDA Grants MEK Inhibitor PD-0325901 For Orphan Drug Treatment of Neurofibromatosis Type 1
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Last Update: 2020-06-11
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Source: Internet
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Author: User
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type 1 neurofibromatosis (NF1) is a rare genetic disease caused by mutations in the NF1 gene that can affect children and adultsrecently, SpringWorks(
FDA
has granted MEK inhibitor PD-0325901 an orphan drug for treatment of neurofibromatosis type 1 (NF1)PD-0325901 is an oral small molecule drug that selectively inhibits MEK1 and MEK2recently, the FDA also granted SpringWorks the fast-track status of adult patients with oral small molecule selective gamma-secretion inhibitor nirogacestat for therapeutic, non-restoctable, recurrent or refractory hard fibroids or deep fibroidsSpringWorks Works, a first-time innovator that was spun off from Pfizer in September 2017, received $103 million in funding for round A financingSpringWorks Works' four core drugs are from Pfizer, including nirogacestat (PF-03084014), MEK 1/2 inhibitor PF-0325901, senicapoc (PF-05416266) and FAAH inhibitor PF-0445784
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