SpringWorks Therapeutics (SWTX) said late Tuesday the US Food and Drug Administration has approved Gomekli, or mirdametinib, for the treatment of certain patients with neurofibromatosis type 1, a genetic disorder with symptoms including abnormal pigmentation and tumor growth.
The approval is based on findings from a phase 2b trial in which Gomekli showed a 41% objective response rate in adults and 52% in children, the company said.
SpringWorks received a rare pediatric disease priority review voucher and intends to make Gomekli available in the US within two weeks, it said.
The company also said it is seeking mirdametinib approval from the European Medicines Agency, with a decision expected this year.
免責聲明：投資有風險，本文並非投資建議，以上內容不應被視為任何金融產品的購買或出售要約、建議或邀請，作者或其他用戶的任何相關討論、評論或帖子也不應被視為此類內容。本文僅供一般參考，不考慮您的個人投資目標、財務狀況或需求。TTM對信息的準確性和完整性不承擔任何責任或保證，投資者應自行研究並在投資前尋求專業建議。