FDA Greenlights SpringWorks' Gomekli for Neurofibromatosis Type 1 Treatment

The US Food and Drug Administration (FDA) has granted approval to SpringWorks Therapeutics for their drug Gomekli (mirdametinib) to address neurofibromatosis type 1 (NF1). This genetic disorder affects around 100,000 individuals in the United States and presents a range of symptoms across different organ systems. These symptoms include abnormal pigmentation, skeletal deformities, tumor growth, and neurological complications.

Gomekli is specifically targeted at treating both adults and children aged two and above who suffer from symptomatic plexiform neurofibromas (PN) that cannot be surgically removed. Approximately 40,000 NF1 patients in the US are affected by PNs. These are tumors that develop along the peripheral nerve sheath and can lead to severe disfigurement, pain, and functional impairment. Moreover, these tumors may progress into malignant peripheral nerve sheath tumors, which pose a significant health risk due to their aggressive and potentially fatal nature.

The newly approved Gomekli represents a significant advancement as it is the first and only FDA-approved oral small molecule MEK inhibitor for both adults and children diagnosed with NF1-PN. The decision by the FDA was heavily influenced by the outcomes of the phase 2b ReNeu trial. This trial revealed an objective response rate of 41% in adults and 52% in pediatric patients suffering from NF1-PN. The trial results further indicated that reductions in tumor volume were substantial and long-lasting. Specifically, 50% of adult participants and 48% of children saw sustained responses for at least 24 months. Additionally, patients in both groups reported early and continuous improvements in pain management and quality of life.

Saqib Islam, the Chief Executive Officer of SpringWorks, emphasized the importance of this approval, highlighting the significant need for additional treatment options within the NF1-PN patient community. He expressed that with this approval, the company is honored to support both adult and pediatric patients by providing a therapy that has the potential to reduce tumor size and offer significant symptomatic relief.

In conjunction with the approval of Gomekli, the FDA has also awarded SpringWorks a priority review voucher (PRV). This voucher allows the company to have another drug reviewed under the FDA’s priority review pathway, which is a faster review process compared to standard timelines. The PRV program was established to stimulate the development of new drugs and biological products aimed at preventing and treating specific rare pediatric diseases. This voucher holds additional strategic value as SpringWorks has the option to sell or transfer it to another company, which can be a lucrative opportunity. This aspect of the approval not only highlights the therapeutic promise of Gomekli but also opens up potential avenues for SpringWorks to further expand its impact within the pharmaceutical industry.