EMA

The European Medicines Agency (EMA) has granted PRIME (Priority Medicines) status to the gene therapy, GNT-0003, currently in clinical trials for Crigler-Najjar syndrome, a rare liver disease.   This status, granted only to drug candidates with major therapeutic potential, follows the successful completion of the first phases of the clinical trials.  The manufacturer, Genethon, is a […]

Robust digital workflows can reduce errors and time to compile data leading to greater transparency, faster reporting and help biopharmaceutical companies reach regulatory submission faster. Ken Forman, Senior Director of Product Strategy, IDBS explains. For many start-ups operating on limited investment capital, the ticking clock can be overwhelming. Teams must plan for post-approval activities while […]

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 […]

Greater data-centricity, supported by new technologies, is key to driving efficient product approvals and supply chains. Frits Stulp and Aida Demneri, partners at Deloitte (including Iperion – a Deloitte business), outline how life sciences companies can play their part in driving change. In the past two years, EU regulatory authorities accelerated processes so that vaccines […]

News round-up for 21-25 August by DDW Editor Reece Armstrong A treatment for Monkeypox, spider venom for drug discovery and funding for international start-ups are some of the stories in this week’s news round-up. Covid researchers launch Monkeypox study   A new study investigating a potential treatment for people who have been diagnosed with monkeypox has […]

The European Medicines Agency (EMA) has granted a Marketing Authorisation Application (MAA) for a combination treatment targeting adults with acute myeloid leukaemia (AML) who are not candidates for standard induction chemotherapy.  The MMA was granted to Otsuka Pharmaceutical and Astex Pharmaceuticals’ oral fixed-dose combination of decitabine and cedazuridine (ASTX727).   The current standard of care for […]

News round-up by DDW Editor Reece Armstrong for 15-19 August. Covid booster approvals, positive results for Malaria vaccines and new CEOs are some of the stories in this week’s news round-up.  MHRA approves Moderna’s bivalent Covid booster   The Medicines and Healthcare products Regulatory Agency (MHRA) has approved an updated version of Moderna’s Covid-19 booster vaccine […]

A gene therapy targeting a rare brain disorder has received a positive opinion by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). PTC Therapeutics’ one-time gene replacement therapy Upstaza was given a positive opinion for the treatment for aromatic L-amino acid decarboxylase (AADC) in patients 18 months or […]

The European Medicines Agency (EMA) has confirmed that the UNICOM’s DADI (Digital Application Dataset Integration) project will now serve as a ‘Plan B’ for the implementation of regulatory data submissions. Amplexor’s Renato Rjavec looks at the opportunities and challenges this presents for pharma companies. A more data-driven way of managing regulated product data, based on […]

Pfizer and BioNTech have submitted data supporting the vaccination of children 5 to 12 years of age with Comirnaty (Covid-19 mRNA vaccine) to the European Medicines Agency (EMA) for a variation of the Conditional Marketing Authorization (CMA) in the European Union (EU). According to The New York Times, federal regulators evaluated the safety and efficacy of this vaccine, […]