SpringWorks Therapeutics receives FDA approval for GOMEKLI™ (mirdametinib) to treat adults and children with NF1-PN.
Wednesday, February 12, 2025
SpringWorks Therapeutics has received FDA approval for GOMEKLI™ (mirdametinib), a MEK inhibitor, to treat adults and children aged two years and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) that cannot be fully removed through surgery. With this approval, the company has been granted a rare paediatric disease priority review voucher by the FDA.
NF1 is a genetic condition affecting approximately 100,000 people in the United States. Individuals with NF1 have a 30-50% risk of developing plexiform neurofibromas, tumours that grow along the peripheral nerve sheath and can cause pain, disfigurement, and functional impairments. Around 40,000 people in the United States live with NF1-PN, most of whom are adults who previously had no approved treatment options. These tumours can sometimes become malignant, leading to a severe and potentially fatal condition. Due to their infiltrative growth pattern, surgical removal is often not possible, with up to 85% of cases considered inoperable.
GOMEKLI was approved based on data from the Phase 2b ReNeu trial, which included 114 patients aged two years and older. The study showed a confirmed objective response rate (ORR) of 41% in adults and 52% in children, as determined by blinded independent central review. Tumour reductions were substantial and lasting, with median best percentage changes in tumour volume of -41% in adults and -42% in children. Many patients also experienced significant improvements in pain and quality of life.
The treatment demonstrated a manageable safety profile. The most common side effects in adults included rash, diarrhoea, nausea, musculoskeletal pain, vomiting, and fatigue. In children, common side effects also included abdominal pain, headache, paronychia, left ventricular dysfunction, and nausea.
GOMEKLI will be available in capsule and tablet forms through a speciality pharmacy and distributor network in the United States within two weeks. SpringWorks has also submitted a Marketing Authorisation Application to the European Medicines Agency (EMA), with a decision expected in 2025.
To support patients, SpringWorks offers the CareConnections™ programme, which provides insurance assistance, financial support, and educational resources. More information is available through the company’s dedicated support services.
Source: globenewswire.com
