European Commission

A label expansion for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) has been approved in the European Union (EU). The European Commission has extended use of the therapy to include children with CF aged two through five years old who have at least one F508del mutation in […]

The European Commission (EC) has granted marketing authorisation for YORVIPATH (palopegteriparatide) as replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism.   YORVIPATH is Ascendis Pharma’s prodrug of parathyroid hormone (PTH 1-34), administered once daily. Ascendis plans its first EU launch of YORVIPATH in January 2024 in Germany.  “Each patient living with chronic hypoparathyroidism […]

The European Commission (EC) has approved INAQOVI (oral decitabine and cedazuridine) as monotherapy for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) who are ineligible for standard induction chemotherapy. INAQOVI, developed by Otsuka Pharmaceutical and Astex Pharmaceuticals, is the first oral hypomethylating agent to be licensed in the EEA in this […]

The European Commission (EC) has granted marketing authorisation for Jazz Pharmaceuticals’ Enrylaze as part of a multi-agent regimen for acute lymphoblastic leukaemia (ALL) and lymphoblastic lymphoma (LBL). The treatment is approved in adult and paediatric patients who developed hypersensitivity or silent inactivation to E. coli-derived asparaginase. Enrylaze, approved as Rylaze in the United States and […]

The European Commission (EC) has granted conditional marketing authorisation (CMA) for Janssen’s Talvey (talquetamab) as monotherapy for relapsed and refractory multiple myeloma (RRMM). It is approved in adult patients who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the […]

On 31 January 2020, the United Kingdom (UK) withdrew from the European Union (EU). Alongside a number of repercussions, the most impactful to the drug discovery sector arguably relates to regulatory affairs on account of adjusting from the European Medicines Agency (EMA) to the Medicines and Healthcare products Regulatory Agency (MHRA). DDW’s Megan Thomas evaluates […]

On 31 January 2020, the United Kingdom (UK) withdrew from the European Union (EU). Alongside a number of repercussions, the most impactful to the drug discovery sector arguably relates to regulatory affairs on account of adjusting from the European Medicines Agency (EMA) to the Medicines and Healthcare products Regulatory Agency (MHRA). DDW’s Megan Thomas evaluates […]

On 31 January 2020, the United Kingdom (UK) withdrew from the European Union (EU). Alongside a number of repercussions, the most impactful to the drug discovery sector arguably relates to regulatory affairs on account of adjusting from the European Medicines Agency (EMA) to the Medicines and Healthcare products Regulatory Agency (MHRA). This is the second […]

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the marketing authorisation of JZP458 (a recombinant Erwinia asparaginase or crisantaspase). It has been recommended for use as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma […]

The Janssen Pharmaceutical Companies of Johnson & Johnson have submitted a Type II variation application to the European Medicines Agency (EMA) seeking approval of Carvykti (ciltacabtagene autoleucel; cilta-cel) for relapsed and lenalidomide-refractory multiple myeloma. The application is supported by data from the Phase III CARTITUDE-4 study, which evaluated the safety profile and efficacy of cilta-cel […]

DDW’s Diana Spencer explores the European Commission’s proposed changes to EU pharmaceutical legislation, the reaction from industry and possible impact on smaller companies. The European Commission (EC) has proposed the largest reform to the EU’s pharmaceutical legislation in over 20 years. The EC says the revisions will make the system more agile, flexible, and adapted to the needs […]

The European Commission (EC) has granted marketing authorisation for Akeega (niraparib and abiraterone acetate [AA]) for adults with metastatic castration-resistant prostate cancer (mCRPC) and BRCA1/2 mutations (germline and/or somatic). The drug is given in the form of a dual action tablet (DAT), with prednisone or prednisolone, and is approved in patients for whom chemotherapy is […]

On January 31 2023, an internally circulated version of the European Commission’s overhaul of the EU’s pharma legislation was leaked, and published by Politico1. Politico published a summary of the key propositions outlined in the draft2, and DDW’s Multimedia Editor Megan Thomas investigates their intention, and observes how the industry has responded.   Key proposals   Approved […]

The European Commission and the UK Government have reached a political agreement that they hope will address the challenges faced by citizens and businesses in Northern Ireland (NI) following Brexit. The Windsor Framework, which focuses on a variety of issues, including supply of medicines, constitutes a comprehensive set of joint solutions aimed to ensure lasting […]

The European Commission has granted conditional marketing authorisation for CSL’s Hemgenix (etranacogene dezaparvovec), the first one-time gene therapy for severe and moderately severe haemophilia B (congenital Factor IX deficiency) in adults without a history of Factor IX inhibitors.   In an ongoing clinical trial, the drug reduced the rate of annual bleeds with a single infusion […]

UCB’s Fintepla (fenfluramine) oral solution has been approved in the European Union (EU) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS).  It has received authorisation as an add-on therapy to other anti-epileptic medicines for patients two years of age and older.  The approval was based on safety and efficacy data from a global, […]