AstraZeneca’s Alexion unit received U.S. Food and Drug Administration approval for Koselugo (cobimetinib) as a treatment for adults with neurofibromatosis type 1 (NF1). The approval expands the company’s rare‑disease portfolio and offers a new therapeutic option for a condition that has historically had limited treatment choices.
The decision was based on the Phase III KOMET trial, the largest and only placebo‑controlled study in this patient population. In the trial, 20 % of patients treated with Koselugo achieved a measurable reduction in plexiform neurofibroma size, compared with 5 % in the placebo arm. Eighty‑six percent of responders maintained a response for at least six months, and the safety profile matched that seen in pediatric patients.
From a business perspective, the approval provides continuity of care for patients who have progressed from the pediatric indication to adulthood. It also strengthens Alexion’s rare‑disease strategy, which has been a key growth engine for AstraZeneca. The company’s Q3 2025 revenue of $15.19 billion and core earnings per share of $2.38—up 12 % and 14 % year‑over‑year, respectively—illustrate the robust financial foundation that will support the launch of Koselugo in the adult market.
Management highlighted the significance of the approval. Alexion CEO Marc Dunoyer said the expanded indication “enables much‑needed continuity of care and supports patients across the disease journey in the U.S.” Professor Pierre Wolkenstein, a lead investigator in the KOMET trial, noted that the approval “reaffirms the role of Koselugo as a strong option for the treatment of adult and pediatric patients with NF1 PN.”
AstraZeneca’s stock reflected the positive market reaction. The share price was up 0.66 % at $89.58, and the company had recently reached a 52‑week high of $86.57. Over the past month, the stock climbed 8 %, contributing to a 27.6 % year‑to‑date return. Analyst sentiment was supportive, with Leerink Partners raising its price target to $87 from $85 and Jefferies upgrading the rating from Hold to Buy.
The competitive landscape for adult NF1 remains limited. While other MEK inhibitors such as mirdametinib are in development, Koselugo is the first approved therapy for symptomatic, inoperable plexiform neurofibromas in adults. This first‑in‑class status positions the drug as a primary treatment option and could capture a significant share of the rare‑disease market.
In summary, the FDA approval of Koselugo for adults with NF1 represents a strategic milestone for AstraZeneca’s rare‑disease portfolio, offers a new treatment pathway for patients, and is expected to contribute to the company’s continued revenue growth and shareholder value.
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