FDA approves Gomekli for neurofibromatosis type 1 with plexiform neurofibromas
Key takeaways:
- Gomekli is the first treatment of its kind to be approved for both children and adults with the condition.
- The drug will be available in 1 mg and 2 mg capsules or a 1 mg tablet for oral suspension.
The FDA has approved an oral, small molecule MEK inhibitor for adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection.
Individuals with NF1-PN, a rare genetic disorder that causes pain, disfigurement and functional impairment, have an 8- to 15-year mean reduction in life expectancy compared with the general population and may experience a 30% to 50% lifetime risk for developing plexiform neurofibromas, according to a press release. The infiltrative tumors, an estimated 85% of which cannot be completely resected, grow along nerves and can lead to permanent nerve damage.
In the press release, manufacturer SpringWorks Therapeutics Inc. said Gomekli, (mirdametinib), the first treatment of its kind for both children and adults, was approved in both 1 mg and 2 mg capsules as well as a 1 mg tablet for oral suspension. The treatment will be available through specialty pharmacy and distributor networks in the United States.
The approval was based on results from the phase 2b ReNeu trial, which enrolled 114 patients with the condition.
“The approval of Gomekli marks another important advance for the care of people living with NF1-PN,” Christopher L. Moertel, MD, lead investigator of the ReNeu study and medical director of the pediatric neuro-oncology and neurofibromatosis program at the University of Minnesota School of Medicine, told Healio. “The documented improvement in tumor size and accompanying symptom improvement with Gomekli is gratifying for those of us who care for this patient group.”
According to the release, Gomekli met the study’s primary endpoint of confirmed objective response rate (ORR), demonstrating a 41% ORR in adults (24 of 58) and 52% in children (29 of 56). Additionally, median change in tumor volume was 41% in adults and 42% in children.
Further, 88% of adults and 90% of children showed a confirmed response of at least 12 months with Gomekli, while the response over 24 months for adults and children were 50% and 48%, respectively.
Gomekli additionally demonstrated a favorable safety and tolerability profile, according to the company.
SpringWorks received a rare pediatric disease priority review voucher from the FDA for Gomekli, which had previously been granted orphan drug and fast-track designations.