G-BA certifies added benefit of pegcetacoplan for C3 glomerulopathy

The Federal Joint Committee (G-BA) decided in its meeting on August 6, 2026, to amend the Pharmaceuticals Directive (AM-RL) and to include a new area of application for the active substance Pegcetacoplan (trade name Aspaveli) in Annex XII. The announcement of the decision was published on September 2, 2026, in the Federal Gazette (Bundesanzeiger) under the official reference BAnz AT 02.09.2026 B3.

The subject is the benefit assessment of Aspaveli for adult and adolescent patients aged 12 to 17 years with C3 glomerulopathy (C3G) in combination with a renin-angiotensin system (RAS) inhibitor, provided that this is not intolerable or contraindicated. The G-BA attests an indication of a non-quantifiable added benefit, as the scientific data basis does not allow for quantification.

Legal Framework and Authorization

Pegcetacoplan is authorized as a medicinal product for the treatment of a rare disease according to Regulation (EC) No 141/2000 on orphan medicinal products. Pursuant to Section 35a (1) sentence 11 first half-sentence of the SGB V (Social Code Book V), the medical added benefit is considered proven by the authorization. The authorization for the new area of application is dated January 15, 2026.

The assessment is based on the G-BA dossier assessment of May 15, 2026. The decision comes into force as of the date of its publication on the G-BA website on August 6, 2026. The supporting reasons are published at www.g-ba.de.

Study Data from VALIANT

The basis for the assessment is the randomized, double-blind, placebo-controlled Phase III VALIANT study with a blinded, placebo-controlled study phase over 26 weeks. For the relevant subpopulation with C3G, the following results were obtained:

• Mortality: no differences relevant to the benefit assessment (1 out of 51 vs. 0 out of 45 events)
• Morbidity: data not assessable
• Health-related quality of life: no relevant differences
• Side effects: no relevant differences

Regarding the primary endpoint, the change in proteinuria (log-transformed FMU-uPCR) up to week 26, a statistically significant group difference of –1.07 (95% CI [–1.37; –0.78], p < 0.0001) was shown in favor of Pegcetacoplan. The change in eGFR did not differ significantly (5.2; 95% CI [–1.0; 11.4], p = 0.10).

Number of Patients and Therapy Costs

The number of patients with C3G aged 12 to 17 years who are candidates for treatment is estimated to be approximately 180 to 370. The annual therapy costs for Pegcetacoplan amount to 362,401.56 euros per patient (as of Lauer-Taxe: June 1, 2026); costs for additional necessary SHI (GKV) services are not applicable.

Requirements for Application

The initiation and monitoring of treatment must be carried out by specialist physicians experienced in the therapy of renal diseases. In accordance with the requirements of the European Medicines Agency (EMA), the pharmaceutical company provides training materials for medical professionals and patients, including a patient identification card, which specifically points out the increased risk of infection with encapsulated bacteria under Pegcetacoplan.

The decision was signed by the Chairperson of the G-BA, Dr. Optendrenk. For patients, physicians, and health insurance funds, the benefit assessment provides clarity regarding the added benefit of a high-priced medicinal product for a rare kidney disease, even if the current data situation does not allow for a quantitative classification of the benefit.

Source: Bundesanzeiger, No. 02.09.2026, Announcement, pp. 1–5 (official reference: BAnz AT 02.09.2026 B3).