FDA Expands Approval of Alexion’s Koselugo to Include Adults with NF1

Rare Daily Staff

The U.S. Food and Drug Administration has approved expanded use of Alexion, AstraZeneca Rare Disease’s Koselugo for the treatment of adult patients with neurofibromatosis type 1 who have symptomatic, inoperable plexiform neurofibromas.

The FDA originally approved Koselugo in April 2020 for pediatric patients age 2 and older with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PN).

The expanded approval was based on positive results from KOMET, the largest and only placebo-controlled global Phase 3 trial in an adult patient population with the condition.

NF1 is a rare, progressive, genetic condition that is usually diagnosed in early childhood but often progresses into adulthood. It can affect every organ system. Up to 50 percent of people living with NF1 may develop a type of nonmalignant tumor called PN that can affect the brain, spinal cord, and nerves. PN may appear later in life and can grow and become large, leading to pain, disfigurement, and muscle weakness, among other debilitating symptoms.

Koselugo is a kinase inhibitor that blocks specific enzymes (MEK1 and MEK2) involved in stimulating cells to grow. In NF1, these enzymes are overactive, causing tumor cells to grow in an unregulated way and create plexiform neurofibromas. By blocking these enzymes, Koselugo slows the growth of tumor cells and the progression of PN.

Koselugo is approved in the United States, European Union, Japan, China, and other countries for the treatment of certain pediatric patients with NF1 who have symptomatic, inoperable PN. It has been granted orphan drug designation in those regions for the treatment of NF1.

“This expanded approval of Koselugo in adults with NF1 PN, together with the recently approved granule formulation for young children age 1 and older, enables much-needed continuity of care and supports patients across the disease journey in the U.S.,” said Marc Dunoyer, CEO of Alexion. “As the first approved therapy in NF1 PN, backed by more than a decade of clinical evidence, Koselugo has transformed the treatment standard for this rare disease.”

Photo: Marc Dunoyer, CEO of Alexion