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February 12, 2025

FDA approves CT drugmaker’s therapy for rare cancer tumors

Contributed GOMEKLI, developed by SpringWorks Therapeutics in Stamford, treats a rare genetic disease that causes cancerous tumors.
By David Krechevsky

Stamford-based SpringWorks Therapeutics, a commercial-stage biotech company developing treatments for rare diseases and cancer, announced Tuesday that federal regulators have approved its drug to treat neurofibromatosis type 1 (NF1), a rare genetic condition that causes tumors to grow aggressively along peripheral nerves.

In a news release, SpringWorks said the U.S. Food and Drug Administration (FDA) has approved GOMEKLI (mirdametinib) to treat adult and pediatric patients aged 2 and up with NF1 who have tumors that cannot be treated surgically.

With the approval, the FDA granted SpringWorks a rare disease priority review voucher (PRV), the company said.

NF1 is a genetic disorder that affects about 100,000 children and adults in the United States. The disorder causes tumors to grow along the peripheral nerve sheath, which can cause severe disfigurement, pain and functional impairment.

GOMEKLI was previously granted Orphan Drug and Fast Track designations for the treatment of NF1-PN. The FDA approval is the company’s second in less than 18 months. In November 2023, SpringWorks received the first FDA approval for a drug to treat desmoid tumors, which are dense, soft-tissue tumors.

News of the latest FDA approval arrived the same day it was revealed that SpringWorks is in “advanced talks” to be acquired by Germany’s Merck, according to Reuters.

SpringWorks, which went public in 2019, is valued at about $4 billion, Reuters said.

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