Mirdametinib 1 mg tablets for oral suspension & 1 mg/2 mg capsules
Trade Name: Gomekli™
Company: SpringWorks Therapeutics
Approval Dates/Comments: In February 2025, the US FDA approved the MEK1/2 inhibitor mirdametinib to treat adult and pediatric patients ≥2 years of age with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. This marks the second FDA-approved treatment for NF and the first approved therapy for adults. The regulatory decision was based on results from the Phase 2b ReNeu trial (NCT03962543), a multicenter, open-label study that enrolled 114 patients aged 2 to 17 years (58 adults and 56 children) with inoperable NF1-associated PN causing significant comorbidity. Mirdametinib met the primary endpoint of confirmed objective response rate (ORR), as assessed by blinded independent central review, demonstrating a 41% ORR (N= 24/ 58) in adults and 52% in children (N=29/56). Deep and long-lasting tumor volume reductions were observed. The median best percentage change in target PN volume was -41% (range: -90 to 13%) in adults and -42% (range: -91 to 48%) in children. Eighty-eight percent of adults and 90% of children with a confirmed response experienced a response of at least 12 months duration, and 50% and 48%, respectively, had a response lasting at least 24 months. Patients in both groups also experienced early and sustained significant improvements from baseline in pain and quality of life, as assessed across multiple patient-reported outcome measures. The most common adverse events (>25%) reported in adults treated with mirdametinib were rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue; whereas in children the most common adverse events (>25%) were rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea. More information is available at: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-mirdametinib-adult-and-pediatric-patients-neurofibromatosis-type-1-who-have-symptomatic
Ustekinumab-stba SC/IV injection
Trade Name: Steqeyma®
Company: Celltrion
Approval Dates/Comments: In December 2024, the FDA approved this biosimilar to the originator product Stelara® (ustekinumab), for the treatment of multiple chronic inflammatory diseases in adults and pediatric patients. This human monoclonal antibody targeting interleukin (IL)-12 and IL-23 is indicated for the treatment of moderate to severe plaque psoriasis in adult and pediatric patients ≥6 years of age who are candidates for phototherapy or systemic therapy; active psoriatic arthritis in adult and pediatric patients ≥6 years of age; and moderately to severely active Crohn disease or ulcerative colitis in adults.
Nivolumab + hyaluronidase-nvhy for SC injection
Trade Name: Opdivo Qvantig™
Company: Bristol Myers Squibb
Approval Dates/Comments: In December 2024, the FDA approved a new dosage form of nivolumab and hyaluronidase-nvhy for subcutaneous (SC) injection across approved adult, solid tumor nivolumab indications as monotherapy, monotherapy maintenance following completion of Opdivo® (nivolumab) + Yervoy® (ipilimumab) combination therapy, or in combination with chemotherapy or cabozantinib. This approval makes Opdivo Qvantig™ the first and only SC administered PD-1 inhibitor, allowing faster drug delivery for patients to receive this immunotherapy treatment in 3-5 minutes vs. 30 minutes intravenously with Opdivo®.
