GOMEKLI^™ (mirdametinib)

FOR IMMEDIATE RELEASE:

Onco360 Has Been Selected as a National Specialty Pharmacy Partner for GOMEKLI^TM (mirdametinib), Manufactured By SpringWorks Therapeutics

Louisville, KY — February 12, 2025 — Onco360^®, the nation’s leading independent specialty pharmacy, has been selected as a national pharmacy partner by SpringWorks Therapeutics, Inc. for GOMEKLI™ (mirdametinib). The Food & Drug Administration approved GOMEKLI™ for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.¹

“Onco360 is grateful for the opportunity to partner on another therapy with the team at SpringWorks Therapeutics and become a specialty pharmacy provider for GOMEKLI™^”, said Benito Fernandez. “We are proud to add GOMEKLI™ for the treatment of neurofibromatosis type 1 patients to our growing portfolio of rare disease therapies.”

Mirdametinib is a MEK1/2 inhibitor now approved in the US for the treatment of NF1-associated PN (NF1-PN). NF1 is a genetic condition that in which 30-50% of children and adults will develop PN. NF1-PN grow along nerves and often cause debilitating morbidities, including pain, disfigurement, compression of internal organs, impaired physical function, and reduced quality of life. There are limited treatment options currently available for managing these tumors.

The FDA approved GOMEKLI™ based on the results of the ReNeu study, where 114 patients [58 adults (≥18 years of age) and 56 children (2 to 17 years of age)] with NF1 and symptomatic PN were evaluated. GOMEKLI™ met the primary endpoint of confirmed overall response rate (cORR) of 41% in adults and 52% in children. cORR is defined as the proportion of patients with complete response or partial response on MRI of the target PN volume from baseline to Cycle 24 as assessed by blinded independent central review on ≥2 consecutive scans within 2 to 6 months. Both adults and children experienced early and sustained significant improvements from baseline in pain and quality of life (QOL).²

The majority of adverse reactions with GOMEKLI™ were mild to moderate. The most common adverse reactions occurring in ≥ 25% of adult patients were rash, diarrhea, nausea, musculoskeletal pain, vomiting. The most common adverse reactions occurring in ≥ 25% of pediatric patients were rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting and headache.¹

Please see the full Prescribing Information for GOMEKLI™.

Media Contact: Benito Fernandez, Chief Commercial Officer
[email protected]
516-640-1332

References:

¹Gomekli (Mirdametinib) [Package Insert]. Stamford, CT. SpringWorks Therapeutics. 2025.
²Moertel, C, et al. ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma. JCO 0, JCO.24.01034. DOI:10.1200/JCO.24.01034.