Pune, India | November 21, 2025
The U.S. Food and Drug Administration has approved Koselugo (selumetinib) for adults diagnosed with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). This remarkable decision expands Koselugo’s previous pediatric indication. That marks an important milestone in treating adults with limited therapeutic choices and providing new hope for long-term disease management.
The FDA’s authorization relied on comprehensive data from the global KOMET trial. A randomized, double-blind, placebo-controlled Phase III study involving 145 adult NF1 patients with inoperable PN. After 16 treatment cycles, results showed a confirmed overall response rate of 20 percent in the selumetinib group. That compared with 5 percent among placebo recipients. Consequently, these findings demonstrated a meaningful clinical benefit, reinforcing the drug’s effectiveness in managing tumor growth.
Notably, 86 percent of patients who responded maintained tumor reductions for at least six months, confirming the sustained impact of the therapy. Participants received orally administered doses twice daily, with dosages adjusted according to individual body surface area to provide a tailored therapeutic approach. This careful dosing strategy ensured both safety and effectiveness for diverse patient profiles throughout treatment.
Neurofibromatosis type 1 represents a rare genetic disorder usually diagnosed during childhood but often continuing into adulthood, causing significant medical challenges. Frequently, individuals with NF1 develop plexiform neurofibromas, which are benign tumors that form along nerve sheaths. When these tumors become large or cannot be surgically removed, they cause pain, disfigurement, and disability. It leads to physical and emotional burdens that affect daily living. Research indicates that nearly half of all NF1 patients develop PN. Also highlighting the urgent need for effective interventions that can address progressive symptoms.
Medical experts have praised this regulatory approval as a major advance for adult NF1 care. Clinical investigators emphasized that Koselugo finally delivers an effective therapy for adults, enabling symptom relief and improved functional outcomes. Moreover, specialists recognize that the treatment’s expansion bridges a critical care gap between pediatric and adult management. It ensures that both age groups now have consistent therapeutic access.
The pharmaceutical industry also celebrated this significant expansion. By extending Koselugo’s indication, manufacturers now offer a continuous treatment pathway from childhood to adulthood. This creates a smoother transition across different patient stages. This continuity of care allows individuals who began treatment early to maintain consistent therapy. It ultimately promotes predictable long-term results and a better quality of life.
Additionally, patient advocacy organizations have expressed strong support for this decision. For years, many adults living with NF1-associated PN had few or no therapeutic options, forcing them to manage chronic pain and reduced mobility with limited resources. Therefore, Koselugo’s adult indication brings renewed optimism, introducing a scientifically validated option that may promote symptom improvement and help maintain physical independence. Advocates believe this development demonstrates meaningful progress in addressing the unmet needs of those living with rare neurological disorders.
Although Koselugo’s safety profile remains consistent with earlier findings, physicians must remain alert to key warnings before and during treatment. Reported risks may include cardiovascular dysfunction, ocular toxicity, gastrointestinal disturbances, elevated creatine phosphokinase levels, bleeding tendencies, and potential embryo-fetal harm. Because these risks vary among patients, healthcare providers must individualize care by monitoring and managing side effects proactively.
Experts recommend regular cardiovascular and ophthalmic evaluations, particularly in patients with preexisting conditions or risk factors. Through such timely monitoring, clinicians can identify adverse effects early and adjust dosages if necessary. Consequently, comprehensive surveillance helps ensure that patients can continue therapy without interruptions while maintaining safety and drug efficacy.
With this adult authorization, Koselugo now becomes an officially available treatment for NF1-related plexiform neurofibromas across both pediatric and adult populations within the United States. This milestone represents a pivotal advancement, introducing a critical therapeutic alternative to individuals who previously faced extremely limited treatment availability. By expanding access, this decision reflects the FDA’s ongoing commitment to addressing rare disorders and improving outcomes for underrepresented patient populations.
Furthermore, the FDA’s action aligns with similar global developments. In Europe, the European Medicines Agency had already recommended extending Koselugo’s indication for adult use, which the European Commission formally approved shortly thereafter. Together, these international decisions highlight a growing recognition of Koselugo’s clinical value and its potential to transform adult NF1 care worldwide. They also exemplify increasing global collaboration to support individuals affected by rare and complex diseases.
In summary, the FDA’s approval of Koselugo for adult patients with NF1 and symptomatic, inoperable plexiform neurofibromas stands as a significant regulatory achievement. This decision extends a scientifically validated, effective, and durable treatment option to adults who once lacked meaningful alternatives. Additionally, it harmonizes adult and pediatric treatment approaches, enabling consistent care throughout a patient’s life. Ultimately, this progress provides new hope for improved quality of life, better disease control, and greater health equity for patients living with neurofibromatosis type 1.
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