SpringWorks Therapeutics Receives Approval for GOMEKLI (Mirdametinib) in NF1-PN Treatment
Wednesday, February 12, 2025
SpringWorks Therapeutics has received approval from the US Food and Drug Administration (FDA) for GOMEKLI (mirdametinib), a MEK inhibitor, for use in adults and children aged two years and older with neurofibromatosis type 1 (NF1) and symptomatic plexiform neurofibromas (PN) that cannot be completely removed by surgery.
NF1 is a genetic disorder that affects approximately 100,000 individuals in the United States. Patients with this condition have a 30-50% lifetime risk of developing plexiform neurofibromas, which are tumours that grow along peripheral nerves and can cause disfigurement, pain, and functional impairment.
An estimated 40,000 people in the United States live with NF1-PN, the majority being adults who previously lacked an approved treatment.
These tumours can sometimes develop into malignant peripheral nerve sheath tumours, a serious and potentially life-threatening condition. Surgery is often difficult due to the infiltrative growth pattern of these tumours, with around 85% deemed unsuitable for complete removal.
This approval has also granted SpringWorks a rare paediatric disease priority review voucher.
The FDA approval was based on data from the Phase 2b ReNeu trial, which included 114 patients with NF1-PN aged two years and above.
The study demonstrated a confirmed objective response rate (ORR) of 41% in adults and 52% in children, with significant tumour volume reductions and long-term symptom relief.
Patients also reported improvements in pain and quality of life. The most common side effects in adults were rash, diarrhoea, nausea, musculoskeletal pain, vomiting, and fatigue, while children experienced additional effects such as abdominal pain and headaches.
GOMEKLI has received Orphan Drug and Fast Track designations.
It will be available in capsule and tablet forms and is expected to be distributed in the United States within two weeks.
The European Medicines Agency (EMA) is currently reviewing SpringWorks' application, with a decision anticipated in 2025.
Source: springworkstx.com
