February 2025
Published: 13 Feb 2025
Author: Precedence Research
SpringWorks Therapeutics has gained approval from the FDA for GOMEKLI (mirdametinib) in the treatment of adult and pediatric patients diagnosed with plexiform neurofibromas associated with type 1 neurofibromatosis (NF1-PN). This is the first and only medicine indicated for both adults and children suffering from NF1-PN.
This specific lesion affects over 100,000 children and adults across the U.S.; patients experience a lifetime risk of developing plexiform neurofibromas (PN) of approximately 30-50%, which can cause severe disfigurement, pain, and functional impairment. Historically, surgery was the main treatment modality; however, 85% of cases cannot be completely resected. The approval of GOMEKLI heralds a new era in treatment and is the first FDA-approved treatment option adults with NF1-PN have ever had, which addresses a huge unmet need in treatment for NF1-PN.
FDA approved GOMEKLI, a treatment for NF1-PN, based on the findings of clinical trials conducted in the Phase 2b ReNeu study. The study was performed on 114 patients wherein the confirmed objective response rate was 41% for adults and 52% for children. There was also a significant degree of tumor shrinkage, durable responses, and a good safety profile. The treatment showed some early and durable signs of symptom improvement for pain and quality of life.
The CEO of SpringWorks Therapeutics, Saqib Islam, expressed his excitement about the Approval and stated that the NF1-PN patient community could use additional treatment options. GOMEKLI might be able to shrink tumors and provide relief from symptoms. He recognized the contributions of all clinical trial participants, investigators, and patient advocacy groups, underscoring the commitment of the company to bring forward therapies for devastating diseases.
Dr. Christopher Moertel, the leading investigator in the ReNeu trial, has described great advances with the therapy GOMEKLI, which treats NF1-PN and provides deep and durable responses coupled with a manageable safety profile. FDA approval of GOMEKLI illustrates how much can be achieved when industry, researchers, and advocacy organizations cooperate to accelerate scientific progress and provide faster access to therapies for patients. Perhaps more importantly, this approval serves as the most recent ray of hope for families affected by NF1-PN, a difficult and devastating illness that does not just involve individual patients but the families as a whole.
The safety and tolerability evaluations for GOMEKLI have verified it to have an acceptable safety profile. Common adverse reactions include rash, diarrhea, nausea, and vomiting in adults while vomiting is found in children. Among the warnings are ocular toxicity, left ventricular dysfunction, dermatologic adverse reactions, and embryo-fetal toxicity. GOMEKLI is available in capsules and tablets and can be accessed through specialty pharmacies and distributor networks. SpringWorks CareConnections™ provides all-inclusive patient support that includes such resources as financial assistance, insurance coverage assistance, educational resources, and emotional support.
The European Medicines Agency (EMA) has received SpringWorks' Marketing Authorization Application for mirdametinib after the FDA approved GOMEKLI. The application is currently under review after being validated. A decision on it is likely to be reached in 2025. The approval represents a major milestone for patients with NF1-PN, being the first-ever treatment option for adults and children alike. With such a strong clinical profile and a very patient-friendly administration, SpringWorks Therapeutics is sure to change the field.
February 2025
February 2025
February 2025
February 2025