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  • Regulatory NewsRegulatory News

    Orphan product designation requests climb in the US, remain steady in the EU

    The number of new requests for orphan drug designation in the United States increased 41% from 2019 to 2020, bringing the number of requests to 753 for 2020, according to the U.S. Food and Drug Administration.   There was also a significant jump in rare pediatric disease designation requests, which rose 330% from 2019 to 2020, for a total of 284 requests in 2020. The agency received 20 humanitarian use device designation requests, similar to the number received in 2019...
  • Regulatory NewsRegulatory News

    EMA offers reassurance on AZ vaccine, while some European authorities pause

     On the same day that the European Medicines Agency granted conditional marketing authorization to Janssen's one-dose COVID-19 vaccine, administration of another authorized COVID-19 vaccine has been paused in scattered European countries.  A handful of European national health authorities have announced the pause for AstraZeneca's COVID-19 vaccine following reports of blood clots in the vaccine's recipients. Other regulators, reassured by the fact that pulmonary embol...
  • ReconRecon

    Recon: 2021's priciest drugs; A leaderless FDA?

    Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.   In Focus: US Novartis' canakinumab failed to improve survival in Phase III trial ( Reuters ) ( Endpoints ) Is a leaderless FDA clamping down on drug reviews? Not knowing is weighing heavy on biotech ( STAT ) Pandemic Forces FDA to Sharply Curtail Drug Company Inspections ( NYT ) A Survey of Survival Outcomes for Targeted Cancer Drugs Approved by the US Food and Drug Admini...
  • Regulatory NewsRegulatory News

    In new pilot, EC probes uneven oncology product launches

    A pilot program launched this month by the European Medicines Agency (EMA) and the European Commission (EC) aims to reduce the time to market launch of certain oncology products by providing the regulator with an “early warning system” for sponsors’ impending marketing plans. The pilot also seeks to aid understanding of the reasons behind uneven launch of some products across European Union (EU) countries.   The project, which is planned to run through August 2022, sup...
  • Regulatory NewsRegulatory News

    Vaccine makers call for alternative study designs for COVID vaccines

    Regulators need to facilitate new ways of clinically validating COVID-19 vaccines now that effective shots are being given around the world, Thomas Triomphe, executive vice president of Sanofi Pasteur, testified at a joint meeting of the European Parliament’s Committee on Industry, Research and Energy (ITRE) and the Committee on the Environment, Public Health and Food Safety (ENVI).   With over a dozen vaccines authorized or approved around the world, a significant n...
  • Regulatory NewsRegulatory News

    EMA sets 12 March J&J vaccine meeting; Celltrion's mAb gets Article 5(3) review

    The European Medicines Agency (EMA) has announced an extraordinary meeting of its human medicines committee (CHMP) to evaluate the COVID-19 vaccine developed by Janssen, a Johnson & Johnson division. The aim of the 11 March meeting will be to “conclude the evaluation, if possible.”   The US Food and Drug Administration issued an emergency use authorization for the one-shot adenovirus vaccine on 27 February after its vaccines advisory committee voted unanimously that ...
  • Regulatory NewsRegulatory News

    EMA, Health Canada publish Moderna data package

    Making good on an earlier commitment to transparency in regulatory decision-making during the COVID-19 pandemic, the European Medicines Agency (EMA), in collaboration with Health Canada, has published the full clinical data package upon which the regulators based their approvals of the COVID-19 vaccine jointly developed by Moderna and the US National Institutes of Health.   The Tuesday announcement of the availability of the data package “highlights the shared commitme...
  • Feature ArticlesFeature Articles

    February's Regulatory Focus: Global and regional harmonization and alignment

    Feature articles during February included a range of topics focused on harmonization and alignment across global and regional regulatory entities, including electronic submissions through the European Medicines Agency (EMA), tools for updating the EU In Vitro Diagnostic Regulation (EU IVDR) template, China’s new PAC regulation, Project Orbis and improving patients access to new therapies, as well as a report on medtech companies’ preparation for EU Medical Device Regulatio...
  • Regulatory NewsRegulatory News

    CHMP’s February recommendations: Evrysdi, Jemperli, and a generic for prostate cancer

    The European Medicines Agency’s Committee for Medicinal Products for Human Use (EMA’s CHMP) recommended six new medicines for approval when it met in February, including two biosimilars and one generic medicine.   The committee recommended marketing authorization for Roche’s Evrysdi (risdiplam) for the treatment of certain types of spinal muscular atrophy (SMA). It is the first oral treatment for this condition and was reviewed under the EMA’s accelerated assessment pr...
  • Regulatory NewsRegulatory News

    EMA releases guidance for COVID variant vaccine development

    The European Medicines Agency (EMA) has published a reflection paper laying out what data are needed to support approval of vaccines that protect against variants of SARS-CoV-2, the virus that causes COVID-19.   “[I]t is an urgent public health priority to define an expedited regulatory process for the adaptation of vaccines to protect against current or future variants,” wrote the agency in announcing the new guidance. The document comes less than 2 weeks after a vari...
  • Regulatory NewsRegulatory News

    EMA plots earlier engagement with patient groups

    The European Medicines Agency’s (EMA) on Friday announced it will pilot an early engagement program to bring patient and consumer groups to the table with its human medicines committee earlier in the evaluation process.   In its proposal for the pilot, EMA says there are “missed opportunities” for its Committee for Medicinal Products for Human Use (CHMP) to incorporate patient perspectives into its reviews, as patient input is typically sought late in the process and o...
  • Regulatory NewsRegulatory News

    EMA: Lentiviral thalassemia treatment on hold pending cancer investigation

    The European Medicines Agency (EMA) was notified by a gene therapy firm that the company has suspended marketing of its beta thalassemia treatment Zynteglo (betibeglogene autotemcel, bluebird bio) as a precautionary measure.   In a press release , the agency related that bluebird bio’s actions came after a recipient of the related bb1111 gene therapy for sickle cell disease developed acute myeloid leukemia (AML). Another bb1111 recipient has also developed myelodyspla...