FDA Approves SpringWorks’ Gomekli for People with NF1-PN

Rare Daily Staff

The U.S. Food and Drug Administration approved SpringWorks Therapeutics’ Gomekli for neurofibromatosis type 1 with plexiform neurofibromas, a rare genetic disorder that causes tumors to grow on nerves.

Gomekli is the first and only medicine approved for both adults and pediatric patients 2 years of age and older with neurofibromatosis type 1(NF1)-plexiform neurofibromatomas (PN) not amenable to complete resection.

People with NF1 have approximately a 30 to 50 percent lifetime risk of developing PNs, which are tumors that grow in an infiltrative pattern along the peripheral nerve sheath and that can cause severe disfigurement, pain, and functional impairment. PN can transform into malignant peripheral nerve sheath tumors, an aggressive and potentially fatal disease.6 Surgical removal can be challenging due to the infiltrative tumor growth pattern of plexiform neurofibromas along nerves, and up to approximately 85 percent of PNs are considered not amenable to complete resection.

Gomekli is an oral, small molecule MEK inhibitor approved in the United States for the treatment of adult and pediatric patients 2 years of age and older with NF1 who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.

The FDA approved Gomekli under Priority Review and SpringWorks received a rare pediatric disease priority review voucher from the FDA. Gomekli was previously granted Orphan Drug and Fast Track designations for the treatment of NF1-PN.

The approval of Gomekli was based on positive data from phase 2b ReNeu trial that showed treatment with Gomekli resulted in robust objective response rate, deep and durable reductions in tumor volume, and a manageable safety profile.

“Patients with NF1-PN often face significant challenges with their health and have had limited treatment options to manage this devastating condition,” said Christopher Moertel, medical director of pediatric neuro-oncology and neurofibromatosis programs and lead investigator of the ReNeu trial. “This approval represents an important advance, especially for adults who previously did not have an approved treatment.”