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EMA: New gene therapy treatment for haemophilia B

EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Durveqtix (fidanacogene elaparvovec) to treat severe and moderately severe haemophilia B in adults who do not have factor IX inhibitors (auto-antibodies produced by the immune system against factor IX replacement medicines) and who have no detectable antibodies to variant adeno-associated virus serotype Rh74 …

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EMA: First vaccine to protect adults from Chikungunya

EMA has recommended granting a marketing authorisation in the European Union (EU) for Ixchiq, the first vaccine in the EU to protect adults 18 years and older against Chikungunya. It is given as a single dose. Chikungunya (also called CHIK fever) is a viral disease caused by Chikungunya virus (CHIKV), a virus transmitted to humans by infected mosquitoes …

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ICH Announces the Availability of a Final Guideline and Questions and Answers Document on Drug Interaction Studies (M12)

On May 22, 2024, the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) announced the availability of a final guideline on Drug Interaction Studies (M12) as well as an M12 Question and Answers document (M12 Q&A). The guideline provides recommendations to promote a consistent approach in designing, conducting, and interpreting …

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FDA grants accelerated approval to selpercatinib for pediatric patients two years and older with RET-altered metastatic thyroid cancer or solid tumors

On May 29, 2024, the Food and Drug Administration granted accelerated approval to selpercatinib (Retevmo, Eli Lilly and Company) for pediatric patients two years of age and older with the following: advanced or metastatic medullary thyroid cancer (MTC) with a RET mutation, as detected by an FDA-approved test, who require systemic therapy; advanced or metastatic thyroid cancer …

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FDA Approves First Interchangeable Biosimilar for Two Rare Diseases

The U.S. Food and Drug Administration approved Bkemv (eculizumab-aeeb) as the first interchangeable biosimilar to Soliris (eculizumab) to treat certain rare diseases. Bkemv is approved for the following treatment indications, which are also currently approved for Soliris: the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis; and the treatment of patients with …

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