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2022 | CHMP Positive Opinions | EMA Drug Approvals | European Commission | Pharma | News

Updated: Apr 27


CSL's gene therapy, Hemgenix, received a positive CHMP opinion for treating Hemophilia B.

The CHMP gave a positive opinion for Hemgenix (etranacogene dezaparvovec), a gene therapy for treating severe and moderately severe Haemophilia B.

The approval was based on the HOPE-B trial, demonstrating the efficacy of Hemgenix in patients with hemophilia B. Hemgenix showed a stable and durable increase in mean Factor IX activity, and the annual bleeding rate was reduced by 64%.

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Haemophilia B is a rare disease resulting in bleeding in joints, muscles, and internal organs, leading to pain, swelling, and joint damage. The current standard of care is the infusion of factor IX for a lifetime.

Imjudo and Imfinzi combination received CHMP positive opinion for advanced liver and lung cancers.

AstraZeneca's Imfinzi (durvalumab) and Imjudo (tremelimumab) combination received CHMP positive opinion as first-line therapy for patients with advanced or unresectable hepatocellular carcinoma (HCC), and Imfinzi, Imjudo, chemotherapy combination in patients with Stave IV non-small cell lung cancer (NSCLC).

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The approval as first-line treatment in liver cancer patients was based on the Phase 3 HIMALAYA trial. STRIDE regimen (Single Tremelimumab Regular Interval Durvalumab) reduced the risk of death by 22% versus sorafenib. The median OS was 16.4 months in patients on the STRIDE regimen versus 18.4 months in patients on sorafenib.

Positive opinion for Stage IV lung cancer s based on the POSEIDON Phase 3 trial. Anti-CTLA-4 antibody Imjudo, Imfinzi, and four cycles of platinum-based chemotherapy reduced the risk of death by 23% versus chemotherapy alone. After five years, 31% of patients on the Imjudo combination were alive versus 22% of patients on chemotherapy.

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Pombiliti received CHMP positive opinion for Pompe disease.

Amicus Therapeutics' Pombiliti (cipaglucosidase alfa) received CHMP positive opinion for late-onset Pompe Disease.

The positive opinion was based on Phase 3 pivotal study (PROPEL) study, which recruited high unmet need enzyme replacement therapy (ERT)-experienced and ERT-naive patients.