SpringWorks Gets Speedy FDA Review of Mirdametinib in Rare Genetic Disorder

Dow Jones08-28

By Colin Kellaher

SpringWorks Therapeutics has won Food and Drug Administration priority review for its proposed mirdametinib treatment for certain patients with the rare genetic disorder neurofibromatosis type 1, or NF1.

SpringWorks said the FDA set a target action date of Feb. 28, 2025, for the application, which covers adults and children with NF1-associated plexiform neurofibromas, adding that the agency indicated that it doesn't currently plan to hold an advisory committee meeting.

The FDA grants priority review to medicines that have the potential to provide significant improvements in the treatment of a serious disease, and the designation shortens the review period.

NF1 is a genetic condition that causes changes in skin pigment and tumors that can grow anywhere in the nervous system, including the brain, spinal cord and nerves.

SpringWorks said NF1 patients have a 30% to 50% risk of developing plexiform neurofibromas, or PN, which are tumors that grow along the peripheral nerve sheath and can cause severe disfigurement, pain and functional impairment.

The Stamford, Conn., biopharmaceutical company said mirdametinib has the potential to be the first approved therapy for adults with NF1-PN and a best-in-class therapy for children with the disorder.

SpringWorks also said the European Medicines Agency has validated its application for mirdametinib, confirming the submission is complete and beginning the EMA's centralized review procedure.

Write to Colin Kellaher at colin.kellaher@wsj.com

(END) Dow Jones Newswires

August 28, 2024 06:58 ET (10:58 GMT)

Copyright (c) 2024 Dow Jones & Company, Inc.

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