Benefit assessment for Glybera gene therapy put on hold

Germany's Gemeinsamer Bundesausschuss (G-BA), the federal joint committee responsible for drug benefit assessments, has suspended its assessment deadline for the first time, exceeding the statutory period. The decision concerns Glybera, a drug designated as an orphan drug for the metabolic disorder lipoprotein lipase deficiency (LPLD), a rare inherited condition.

The G-BA was scheduled last Thursday to finalize its assessment of Glybera. Patients with LPLD suffer from an enzyme deficiency affecting fat metabolism. Approximately 500 individuals in Europe are affected by this condition.

The suspension of the benefit assessment is due to new data regarding the drug's efficacy, which has been submitted to the European Medicines Agency (EMA). The EMA is set to review these findings next week, and the subsequent steps for Glybera's assessment will depend on the EMA's decision.

G-BA Chairman Josef Hecken described the situation as an "atypical special case" and has frozen the AMNOG (Arzneimittelmarktneuordnungsgesetz) process indefinitely. He noted that the G-BA typically adheres strictly to deadlines, as demonstrated in approximately 120 previous assessments.