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Koselugo FDA Approval History

Last updated by Judith Stewart, BPharm on June 24, 2020.

FDA Approved: Yes (First approved April 10, 2020)
Brand name: Koselugo
Generic name: selumetinib
Dosage form: Capsules
Company: AstraZeneca and Merck
Treatment for: Neurofibromatosis Type 1

Koselugo (selumetinib) is an inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2) indicated for the treatment of pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).

  • NF1 is a debilitating genetic condition caused by a spontaneous or inherited mutation in the NF1 gene. It is associated with many symptoms, including cutaneous neurofibromas and skin pigmentation. In 30-50% of patients with NF1, tumors develop on the nerve sheaths (plexiform neurofibromas) that can cause clinical issues such as pain, motor dysfunction, airway dysfunction, bowel/bladder dysfunction and disfigurement.
  • FDA approval of Koselugo was based on positive results from the Phase II SPRINT Stratum 1 trial of 50 patients, with 66% of patients having a confirmed complete or partial response of at least 20% reduction in tumor volume.
  • Koselugo is administered orally, twice daily on an empty stomach.
  • Koselugo may cause serious side effects including cardiomyopathy, ocular toxicity, severe diarrhea, skin rash, and muscle problems (rhabdomyolysis). Common side effects include vomiting, rash, abdominal pain, diarrhea, nausea, dry skin, fatigue, musculoskeletal pain, pyrexia, acneiform rash, stomatitis, headache, paronychia, and pruritus.

Development timeline for Koselugo

Apr 13, 2020Approval FDA Approves Koselugo (selumetinib) for Pediatric Patients with Neurofibromatosis Type 1 Plexiform Neurofibromas
Nov 14, 2019US FDA Accepts Regulatory Submission of New Drug Application for Selumetinib in Neurofibromatosis Type 1 (NF1) and Grants Priority Review
Apr  1, 2019Selumetinib Granted U.S. Breakthrough Therapy Designation in Neurofibromatosis Type 1
Apr  1, 2019Selumetinib Granted US Breakthrough Therapy Designation in Neurofibromatosis Type 1
Feb 15, 2018Selumetinib Granted Orphan Drug Designation by the U.S. FDA for Neurofibromatosis Type 1

Further information

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