Lilly's appeal unlocks EU approval of Alzheimer's drug

Eli Lilly's appeal of a decision by the EMA's human medicines committee not to approve its Alzheimer's disease therapy Kisunla has paid off, albeit with a narrower label than it originally sought.

The CHMP recommended granting a marketing authorisation for anti-amyloid therapy Kisunla (donanemab) only in people who do not have a copy of the ApoE4 gene, a risk factor for Alzheimer's, or in those who have only one copy of the gene.

In March, the panel said it was unable to recommend the drug because its modest benefits did not outweigh risks, including amyloid-related imaging abnormalities (ARIA) like brain bleeding and swelling, prompting Lilly to ask for a re-evaluation.

Key to the CHMP's change of heart is a new dosing regimen for Kisunla, recently approved by the US FDA, which has been shown in trials to reduce the risk of ARIA simply by deferring one dose in the titration phase of treatment, without compromising its ability to clear the amyloid plaques that characterise Alzheimer's.

The CHMP's decision on Kisunla mirrors the process that occurred with Eisai and Biogen's rival amyloid-targeting Alzheimer's therapy Leqembi (lecanemab), which was initially rejected by the committee, but later approved by the European Commission – in a similar patient population with only one or no copy of the ApoE4 genetic variant – in April.

Other CHMP decisions

The Kisunla decision came among other positive recommendations from the CHMP at its July meeting, including a green light for Gilead Sciences' twice-yearly HIV pre-exposure prophylaxis (PrEP) therapy Yeytuo (lenacapavir), which was also cleared by the FDA as Yeztugo in June.

KalVista Pharma got the go-ahead for Ekterly (sebetralstat), becoming the first treatment in the EU that can provide oral, on-demand treatment of hereditary angioedema (HAE) attacks, while Deciphera's Romvimza (vimseltinib) was recommended for tenosynovial giant cell tumours (TGCT) that are not treatable with surgery.

Others included Ionis' Tryngolza (olezarsen) for familial chylomicronaemia syndrome (FCS), Servier's Voranigo (vorasidenib) for rare brain tumours low-grade astrocytoma or oligodendroglioma, IntraBio's Aqneursa (levacetylleucine), for the treatment of the rare genetic disorder Niemann-Pick type C, and Biogen's postpartum depression (PPD) treatment Zurzuvae (zuranolone).

Finally, the CHMP gave a positive opinion to update the composition of two COVID-19 vaccines – Pfizer/BioNTech's Comirnaty and Moderna's Spikevax – to target the new LP.8.1 variant of SARS-CoV-2 in the 2025/26 vaccination season.