Tower Mains Weekly Newsletter
EMA launches the Regulatory Science Research Needs initiative
On 15th December, the European Medicines Agency (EMA) announced it had issued a Regulatory Science Research Needs list of around one hundred topics that need further research to close gaps and improve medicine development and evaluation to enable access to innovative medicines for patients. These topics, and the initiative itself, emerged from the stakeholder consultations which underpinned the development of the Regulatory Science Strategy to 2025.
EMA announces regulatory science research webinar
On 15th December, the EMA announced it is hosting an online webinar seeking to stimulate researchers and funding organisations to consider addressing these needs in their research work and funding programmes, respectively. The event to launch the Regulatory Science Research Needs initiative will inform the public and stakeholders about these needs and how stakeholders can engage with the regulatory science research needs. The webinar will be held on the 18th of January 2022, 10 – 12pm (CET).
FDA approves first drug to prevent graft versus host disease
On 15th December, the US Food and Drug Administration (FDA) approved Orencia for the prevention of acute graft versus host disease (aGVHD), a condition that occurs when donor bone marrow or stem cells attack the graft recipient, in combination with certain immunosuppressants. Orencia may be used in adults and paediatric patients two years of age or older undergoing bone marrow transplantation from an unrelated donor.
MHRA publishes outcome of randomised controlled trials consultation
On 16th December, the Medicines and Healthcare products Regulatory Agency (MHRA) published the outcome of the consultation on randomised controlled trials generating real-world evidence to support regulatory decisions. This includes two draft guidance documents: MHRA Guidance on the use of Real-World Data in Clinical Studies to Support Regulatory Decisions and MHRA Guideline on Randomised Controlled Trials using Real-World Data to Support Regulatory Decisions. This guidance provides points to consider when planning a randomised clinical trial using real world data, with the intention of submitting this trial to gain a regulatory approval – such as extending the use of a medication into a new indication or a broader patient population.
MHRA publishes pharmacovigilance inspection metrics for 2021
On 16th December, the MHRA published its GPvP Inspection Metrics report for April 2020 to March 2021.
EMA recommends granting marketing authorisation for a new treatment for sickle cell disease
On 17th December, the EMA announced it had recommended granting a marketing authorisation in the EU for Oxbryta for the treatment of haemolytic anaemia due to sickle cell disease in patients 12 years of age and older. The treatment is supported through EMA’s Priority Medicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support for promising medicines with the potential to address unmet medical needs. The opinion adopted by the CHMP is an intermediary step on Oxbryta’s path to patient access.
European Commission ensures a continued supply of medicines to NI, as well as Cyprus, Ireland, and Malta
On 17th December, the European Commission put forward proposals to ensure the continued long-term supply of medicines from Great Britain to Northern Ireland, meaning that the same medicines will continue to be available in Northern Ireland at the same time as in the rest of the United Kingdom, while specific conditions ensure that UK-authorised medicines do not enter the Single Market.
It also put forward proposals to address the outstanding supply concerns in Cyprus, Ireland, and Malta. For example, during this period in these three countries, importers of medicines from the UK will not need to hold manufacturing authorisations, nor will these medicines need to be batch tested again if they have already been tested in the UK.
EMA Management Board meeting, December 2021
On 17th December, the EMA issued highlights from its Management Board meeting. These include the EMA budget for 2022, establishing accelerating Clinical Trials in the EU (ACT EU), and the preparation for the implementation of the Veterinary Medicinal Products Regulation.
FDA approves new treatment for Myasthenia Gravis
On 17th December, the FDA approved Vyvgart for the treatment of generalized myasthenia gravis (gMG) in adults who test positive for the anti-acetylcholine receptor (AChR) antibody. The treatment is an antibody fragment that binds to the neonatal Fc receptor (FcRn), preventing FcRn from recycling immunoglobulin G (IgG) back into the blood. The medication causes a reduction in overall levels of IgG, including the abnormal AChR antibodies that are present in myasthenia gravis. The FDA granted this application Fast Track and Orphan Drug designations.
European Commission publishes annual report on the application of the Regulation on medicines for children
On 20th December, publishes the latest annual report on the application of the Regulation on medicines for children. This report covers 2020 and lists the companies benefiting from and infringing the regulation.
FDA approves manufacturing change for Comirnaty COVID-19 vaccine
On 15th December, the FDA approved a manufacturing change for Comirnaty COVID-19 vaccine to include a formulation that uses a different buffer. This new formulation is more stable at refrigerated temperatures for longer periods, permitting greater flexibility for vaccination providers. This formulation of Comirnaty does not need to be diluted before use, thus, vaccination providers can more readily prepare and deliver appropriate doses.
EMA recommends approval for use of Kineret in adults with COVID-19
On 16th December, the EMA’s human medicines committee (CHMP) recommended extending the indication of Kineret to include treatment of COVID-19 in adult patients with pneumonia requiring supplemental oxygen and who are at risk of developing severe respiratory failure, as determined by blood levels of a protein called soluble urokinase plasminogen activator receptor (suPAR ) of at least 6 ng per ml. The medicine is considered to reduce the inflammation associated with COVID-19 and thus decrease lower airway damage, preventing the development of severe respiratory failure.
EMA recommends approval for use of Kineret in adults with COVID-19
On 16th December, the EMA’s CHMP recommended authorising the monoclonal antibody Xevudy for the treatment of COVID-19. Xevudy is the third monoclonal antibody recommended in the EU for treating COVID-19 and follows the approval of Region and Ronapreve in November.
EMA issues advice on use of Paxlovid for the treatment of COVID-19
On 16th December, the EMA’s CHMP issued advice on the use of Paxlovid (PF-07321332 and ritonavir) for the treatment of COVID-19. The medicine, which is not yet authorised in the EU, can be used to treat adults with COVID-19 who do not require supplemental oxygen and who are at increased risk of progressing to severe disease. Paxlovid should be administered as soon as possible after a diagnosis of COVID-19 and within 5 days of the start of symptoms. In parallel to the provision of this advice, a more rolling review started on 13th December 2021 ahead of a possible application for a marketing authorisation.
MHRA updates guidance how on to get fast-track approval of medical devices during COVID-19
On 17th December, the MHRA updated its guidance on exemptions from devices regulations during the COVID-19 outbreak with information regarding software products that read lateral flow tests.
MHRA updates advice for users of software or apps being used during COVID-19 pandemic
On 17th December, the MHRA updated advice for manufacturers, members of the public and professional users of software or apps being used during the COVID-19 pandemic. Information on software specifically intended to read lateral flow tests was added.
WHO issues an emergency use listing for Corovax COVID-19 vaccine
On 17th December, the World Health Organisation (WHO) issued an emergency use listing (EUL) for Covovax COVID-19 vaccine. The vaccine is produced by the Serum Institute of India under licence from Novavax and is part of the COVAX facility portfolio, boosting the ongoing efforts to increase access to vaccination in lower-income countries.
EMA recommends Nuvaxovid for authorisation in the EU
On 20th December, the EMA announced it had recommended granting a conditional marketing authorisation for Novavax’s COVID-19 vaccine Nuvaxovid (also known as NVX-CoV2373) to prevent COVID-19 in people from 18 years of age. The original strain of SARS-CoV-2 and some variants of concern such as Alpha and Beta were the most common strains circulating when the studies were ongoing, therefore there is currently limited data on the efficacy of Nuvaxovid against other variants of concern, including Omicron.
MHRA publishes Public Assessment Report for Lagevrio
On 20th December, the MHRA published the Public Assessment Report for Lagevrio (molnupiravir).
FDA grants marketing authorisation for inferior vena cava filter removal device
On 21st December, the FDA authorised marketing of the first laser-based device for the removal of Inferior Vena Cava (IVC) filters. The device is designed for patients who have an IVC filter, a small cage-like device inserted into the largest vein in the body to capture blood clots and prevent them from traveling to the lungs. The new device, called the Philips CavaClear Laser Sheath, is intended for the removal of tissue to facilitate the detachment of an IVC filter during retrieval when previous methods of removal have failed.
FDA funding new project to enhance the understanding of nonclinical model responses to SARS-CoV-2
The FDA, Australia’s national science agency Commonwealth Scientific and Industrial Research Organisation (CSIRO), and its global partners will use systems biology and machine learning approaches to enhance the understanding of nonclinical model responses to SARS-CoV-2. The new project aims to develop the underpinning regulatory science needed to help the FDA evaluate nonclinical model data for assessing potential vaccines and therapeutics for COVID-19, including the impact of omicron, and future SARS-CoV-2 variants of concern.
Issue Number: WN00042