G-BA certifies added benefit of Selumetinib for young children with NF1

The Federal Joint Committee (G-BA) decided in its meeting on August 6, 2026, to supplement the Pharmaceuticals Directive with a benefit assessment of the active substance selumetinib (trade name Koselugo) for a new area of application. This affects children aged 1 to under 3 years with symptomatic, inoperable plexiform neurofibromas (PN) in neurofibromatosis type 1 (NF1). The announcement of the decision was published on September 14, 2026, in the Federal Gazette (Bundesanzeiger) under the official reference BAnz AT 14.09.2026 B2.

The decision supplements Annex XII of the Pharmaceuticals Directive, in which the benefit assessments of medicinal products with new active substances according to Section 35a of the Fifth Book of the Social Code (SGB V) are documented. It is based on the authorization of January 9, 2026, according to which Koselugo monotherapy is indicated for patients from 1 year to under 7 years, as well as for older patients with swallowing difficulties. The area of application covered by the decision is limited to children from 1 to under 3 years.

Added Benefit and Comparator Therapy

The G-BA defines best supportive care as the appropriate comparator therapy for selumetinib as a monotherapy. The extent and probability of the added benefit compared to this comparator therapy are assessed as evidence of a non-quantifiable added benefit.

The assessment is based on the dossier assessment by IQWiG (A26-09) and the addendum (A26-76). No evaluable data were available for the endpoint categories of mortality, health-related quality of life, and side effects; an advantage was shown in the endpoint “change in tumor volume.”

Study Data from SPRINKLE

The basis is the results of the ongoing, open-label, single-arm phase I/II study SPRINKLE with a data cutoff date of December 16, 2025. For the relevant subpopulation of children from 1 to under 3 years, the following results were obtained:

• Mortality: no deaths in the study
• Volume change of target lesion: mean best change achieved of –9.61 ml (SD 20.86) in 13 patients
• Objective Response Rate (ORR): 2 out of 13 (15.4%)
• Progression-Free Survival (PFS): 5 out of 13 (38.5%)
• Pain (GIS-pNF, parent-reported): at cycle 25, 13 out of 13 (100%) reported no pain
• Total adverse events: 13 out of 13 (100%), serious events in 3 out of 13 (23.1%)

Number of Patients and Therapy Costs

It is estimated that approximately 55 to 95 patients in Germany are eligible for treatment. After deducting legally required discounts (as of Lauer-Taxe: June 1, 2026), the annual therapy costs for selumetinib amount to €81,237.57 to €97,443.08 per patient. The range results from the lower limit for those aged 1 to under 2 years and the upper limit for those aged 2 to under 3 years.

The initiation and monitoring of treatment should be carried out by specialist physicians for internal medicine and hematology and oncology, or for pediatric and adolescent medicine with a focus on neuropediatrics or pediatric hematology and oncology, who are experienced in the therapy of NF1-related tumors. The medicinal product was approved under “special conditions”; the European Medicines Agency (EMA) will evaluate new information at least annually.

The decision comes into effect as of the date of its publication on the G-BA website on August 6, 2026. For affected families, the benefit assessment provides a clear basis for the reimbursement of treatment via statutory health insurance, even if the added benefit is classified as non-quantifiable given the limited data available.

Source: Bundesanzeiger, BAnz AT 14.09.2026 B2, published on September 14, 2026, section Official Announcements (official reference: BAnz AT 14.09.2026 B2).