DECEMBER 2022 CHMP POSITIVE OPINIONS
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.