Marketing approval authorisation

The Janssen Pharmaceutical Companies of Johnson & Johnson has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) seeking approval of erdafitinib for the treatment of adult patients with locally advanced unresectable or metastatic urothelial carcinoma (UC), harbouring susceptible fibroblast growth factor receptor 3 (FGFR3) genetic alterations, with disease progression during or […]

The EU’s Committee for Medicinal Products for Human Use (CHMP) recommended granting a marketing authorisation for 14 new medicines in its July meeting. The following novel medicines and biosimilars each received a positive opinion: Abrysvo (bivalent, recombinant), a vaccine to protect small infants and older people against lower respiratory tract disease caused by respiratory syncytial […]

The US Food and Drug Administration (FDA) has granted marketing authorisations to several new therapeutics over the last month that will have a significant impact on areas of unmet need and the evolution of future treatments. Elevidys, Sarepta Therapeutics Elevidys became the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in […]

The European Federation of Pharmaceutical Industries and Associations (EFPIA) has published a series of reports about the availability of new medicines throughout Europe. EFPIA’s 2022 Patient WAIT Indicator tracks the availability of new medicines and length of time it takes patients to get them in 37 countries in the EU and EEA. Figures show that, […]

The Food and Drug Administration (FDA) in the US has made several key drug decisions over the last few weeks. Here’s a summary. Orserdu (elacestrant), Stemline Therapeutics The first and only treatment specifically indicated for patients with ESR1 mutations in ER+, HER2- advanced or metastatic breast cancer was approved in the US in February. The […]

The Scottish Medicines Consortium (SMC) has become the first UK health authority to permit routine NHS use of Gilead Sciences’ Hepcludex (bulevirtide) for the treatment of chronic hepatitis delta virus (HDV) infection. The drug has been accepted for the treatment of chronic HDV infection in adult patients with evidence of significant fibrosis, whose disease has […]

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 […]

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended not to grant marketing authorisation for Ipsen’s investigational palovarotene as a treatment for fibrodysplasia ossificans progressiva (FOP).   The company says it will be requesting a re-examination of the CHMP opinion, based on scientific data available from the existing […]

Janssen has submitted a marketing authorisation application (MAA) seeking approval of talquetamab in Europe for the treatment of patients with relapsed or refractory multiple myeloma (RRMM).   Talquetamab is an investigational, off-the-shelf, bispecific T-cell engager antibody targeting both GPRC5D, a novel drug target that is on some normal cells but overexpressed on myeloma cells, and CD3 […]

AstraZeneca’s Evusheld (tixagevimab co-packaged with cilgavimab) has been authorised by the Medicines and Healthcare products Regulatory Agency (MHRA) and is the first antibody combination for pre-exposure prophylaxis (PrEP) against Covid-19 licensed in Great Britain.   The use of this medicine is for adults who are not currently infected with (or know exposure to) the Covid-19 virus […]

The National Institute for Health and Care Excellence (NICE) has updated Technology Appraisal Guidance (TAG) TA599 for AstraZeneca’s Lokelma (sodium zirconium cyclosilicate [SZC]) in the treatment of adult patients with hyperkalaemia in England, Wales, and Northern Ireland.   Context   This guidance update follows the removal of the previous commercial arrangement, meaning that access to SZC for the treatment of persistent hyperkalaemia is […]

The Pfizer/BioNTech COVID-19 vaccine has been authorised for emergency use by the Medicines and Healthcare Products Regulatory Authority (MHRA). Pfizer and BioNTech recently announced final efficacy analysis in their Phase III study for their mRNA-based COVID-19 vaccine candidate BNT162b2, which showed an efficacy rate of 95% in participants without prior SARS-CoV-2 infection. The vaccine will […]

Vanessa Fachada Oliveira, Pharmacovigilance Manager & EU QPPV at Arriello, discusses 10 common issues identified in risk-based audits, and pinpoints implications for drug developers as well as marketing authorisation holders (MAHs). In EU markets, pharmacovigilance (PV) provisions are closely monitored to ensure that patients can have maximum confidence in the safety of the products they take. […]

Diamond Pharma Services has provided EU regulatory, pharmacovigilance, quality and compliance support to GenSight Biologics including the preparation, authoring support, agency communication and submission of GenSight’s first marketing authorisation application (MAA) to the European Medicines Agency (EMA), for its novel ocular gene therapy LUMEVOQ.  The EMA decision is expected in H2 2021. LUMEVOQ is a […]