European Regulators Endorse New Drugs and Label Expansions, Reaffirm Leqembi's Positive Opinion

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has concluded a productive meeting, recommending approval for several new drugs and label expansions while reaffirming its support for Eisai and Biogen's Alzheimer's treatment Leqembi.

CHMP Backs Four New Drugs, Endorses Multiple Label Expansions

In its latest session, the CHMP recommended four new drugs for approval in the European Union. These include Takeda's immunoglobulin drug Deqsiga, Regeneron's multiple myeloma antibody Lynozyfic, Krystal Biotech's dystrophic epidermolysis bullosa gene therapy Vyjuvek, and Accord Healthcare's generic drug trabectedin for advanced soft tissue sarcoma and relapsed platinum-sensitive ovarian cancer.

The committee also endorsed 16 potential label expansions for existing medications. Notable among these are Eli Lilly's Jaypirca for relapsed or refractory chronic lymphocytic leukemia, AbbVie's Rinvoq for giant cell arteritis, and Novartis' Fabhalta for C3 glomerulopathy, an ultrarare kidney disease currently lacking approved treatments.

Other significant label expansions include Vertex's Kaftrio and Kalydeco combination for cystic fibrosis in patients aged 2 and older with specific genetic mutations, and Valneva's chikungunya vaccine Ixchiq for active immunization in children from age 12.

Leqembi Emerges Unscathed from Safety Reappraisal

In a major development for Eisai and Biogen, the CHMP reaffirmed its positive opinion on Leqembi (lecanemab) for Alzheimer's disease, originally issued in November. This decision comes after the European Commission requested a review of new safety data that emerged following the initial recommendation.

The CHMP's endorsement specifically supports Leqembi for treating mild cognitive impairment or mild dementia from Alzheimer's in patients carrying one or no copy of the ApoE4 gene. This restriction is based on clinical trial data indicating a higher risk of brain swelling or bleeding side effects in patients with two copies of the gene.

Biogen stated that the safety profile of lecanemab reported in clinical practice in various countries post-launch aligns with the approved labels, with no new safety signals identified. The final approval decision for Leqembi now rests with the European Commission, the ultimate authority on marketing authorization in the EU.

Notable Withdrawals and Future Considerations

While many drugs advanced through the regulatory process, some applications were withdrawn. Sanofi pulled its application for a label expansion of Dupixent to treat moderate to severe chronic spontaneous urticaria in adults and adolescents. Additionally, Spanish biotech HIPRA withdrew its application for an adapted version of its COVID-19 vaccine Bimervax targeting an updated SARS-CoV-2 strain.

These developments highlight the dynamic nature of the pharmaceutical regulatory landscape, with companies continually reassessing their strategies based on evolving data and market conditions.