Alexion, part of AstraZeneca Rare Disease, is all set is expand its life-changing treatment for adults with neurofibromatosis, a rare, genetic condition, thanks to US FDA's recent approval of the same.

 

The approval is for its Koselugo (selumetinib) for adults with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).

 

Koselugo, an oral, selective MEK inhibitor, becomes the first FDA-approved therapy for NF1 PN in adults, following strong results from the global Phase III KOMET trial, the only placebo-controlled study in this population. Data from KOMET were presented at the 2025 ASCO Annual Meeting and published in The Lancet.

 

NF1 is often diagnosed in childhood, with tumors that can grow along nerves, leading to pain, disfigurement, and muscle weakness. Up to 50% of patients develop PN, which can progress into adulthood.

 

“The KOMET trial highlights Koselugo’s potential to tackle the substantial clinical burden of PN in adults, building on its established success in pediatric patients,” said Prof. Pierre Wolkenstein, KOMET trial investigator.

 

Marc Dunoyer, CEO of Alexion, added: “This approval, alongside the recent pediatric granule formulation for children as young as one, ensures continuity of care and reinforces Koselugo’s role as a transformative therapy for NF1 PN.”

 

Patient advocacy groups welcomed the news. Annette Bakker, CEO of the Children’s Tumor Foundation, said: “Koselugo has already changed what is possible for children with NF1, and now adults will benefit. This milestone shows what can be achieved when the NF community, scientists, clinicians, and industry work together to bring effective treatments to patients faster.”

 

In KOMET’s primary analysis, Koselugo demonstrated a 20% overall response rate (ORR) compared to 5% for placebo, with 86% of responders maintaining benefits for at least six months. Safety results were consistent with the drug’s established pediatric profile.

 

Koselugo has also been approved in the EU, Japan, and other countries, with additional regulatory reviews ongoing worldwide.