Tower Mains Weekly Newsletter
Company Updates
When things don’t go to plan in a laboratory, a good investigation is pivotal to understanding what went wrong and ensuring all necessary steps have been taken to identify the error, understand the root cause and prevent recurrence. Our new ‘Conducting Laboratory Investigations’ course is designed to equip you with the skills and knowledge to conduct a successful investigation. Our next open course date is the 22nd of November 2023 or if you are interested in this training for your organisation at a time of your choosing, please get in touch at training@towermains.com. For further information, click here.
Regulatory Updates
MHRA guidance updates
- MHRA phase I accreditation scheme. On 10th November, the Medicines and Healthcare products Regulatory Agency (MHRA) updated the list of accredited phase I units in the guidance on the scheme. Click to view source
- MHRA publishes performance data for assessment of clinical trials and established medicines. On 15th November, the MHRA published ‘Clinical Trials Performance Metrics: November 2022 – October 2023: Assessment of Clinical Trial Authorisation Applications, Clinical Investigations and Substantial Amendments‘ and ‘Performance Metrics January 2023 – October 2023: Assessment of New Marketing Authorisation Applications and Variations‘
FDA guidance updates
- Real-Time Oncology Review (RTOR). On 6th November, the US Food and Drug Administration (FDA) published final guidance providing recommendations to applicants on the process for submission of selected new drug applications (NDAs) and biologics license applications (BLAs) with oncology indications for review under Real-Time Oncology Review (RTOR). Click to view source
- COVID-19 container closure system and component changes: Glass vials and stoppers guidance for industry. On 8th November, final guidance was published providing technical specifications for the submission of clinical outcome assessment (COA) data that use Item Response Theory (IRT) and supplements the FDA Center for Drug Evaluation and Research (CDER) Patient-Focused Drug Development (PFDD) Methodological Guidance Series. Click to view source
Industry Updates
FDA approves first treatment for patients with rare inherited blood clotting disorder
On 9th November, the FDA approved Adzynma, the first recombinant protein product indicated for preventive or on demand enzyme replacement therapy (ERT) in adult and paediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), a rare and life-threatening blood clotting disorder.
Documentary highlights new targets to potentially treat motor neurone disease
On 10th November a new documentary, ‘Turning the Tide’ has highlighted the discovery of a promising new target for new treatments of motor neurone disease (MND) made by researchers at University College London (UCL). Funded as part of LifeArc’s MND Translational Fund and other MND charities, as well as the MND scientific community, provides insight into what it is like for those living with MND as well as their hopes for ongoing research in the field.
FDA approves first vaccine to prevent disease caused by chikungunya virus
On 9th November, the FDA approved Ixchiq, the first chikungunya vaccine. Ixchiq is approved for individuals 18 years of age and older who are at increased risk of exposure to chikungunya virus and is administered as a single dose by injection into the muscle. It contains a live, weakened version of the chikungunya virus and may cause symptoms in the vaccine recipient like those experienced by people who have chikungunya disease.
BBSRC to invest £12m into 62 UK bioscience research projects
On 9th November, it was announced that the Biotechnology and Biological Sciences Research Council (BBSRC) will fund 62 visionary projects that could revolutionise our understanding of the rules of life. The researchers across the UK will receive a share of £12 million to pursue novel bioscience research, and by drawing upon unconventional thinking and approaches, they hope to make exciting discoveries with the potential to transform our understanding of the fundamental rules of life.
EMA issues latest CHMP meeting highlights
On 10th November, the European Medicines Agency (EMA) issued meeting highlights from the human medicines committee (CHMP), held on the 6th-9th November 2023. Highlights include eight new medicines recommended for approval, recommendations on extensions of therapeutic indication for nine medicines and CHMP statistics.
EMA issues latest CVMP meeting highlights
On 10th November, the EMA issued meeting highlights from the Veterinary Medicinal Products (CVMP), held on the 7th-9th November 2023. Highlights include the procedure for checking proposed (invented) names for veterinary medicinal products, the revised reflection paper on the environmental risk assessment of ectoparasiticidal veterinary medicinal products used in cats and dogs (EMA/CVMP/ERA/31905/2021) and the adoption of a concept paper on the revision of the guideline on the principles of regulatory acceptance of 3Rs testing approaches (EMA/452614/2023).
FDA grants marketing authorisation of first test for chlamydia and gonorrhoea with at-home sample collection
On 15th November, the FDA granted marketing authorisation to LetsGetChecked for the Simple 2 Test, the first FDA-authorised test with at-home sample collection for any sexually transmitted disease other than HIV. The test is available over the counter (OTC) and is intended for use in adult patients ages 18 years and older.
US Federal Court enters consent decree against Arizona-based company for distribution of unapproved contraceptive drugs
On 15th November, US District Court for the District of Arizona entered a consent decree of permanent injunction ordering Smart Women’s Choice, Inc., an Arizona-based company, and Jennifer A. Richard, its chief executive officer, to stop distributing drugs until the company complies with the Federal Food, Drug, and Cosmetic Act (FD&C Act) and other requirements listed in the consent decree.
Cystic fibrosis drugs Kaftrio and Kalydeco licensed for patients aged two to five years old
On 15th November, the MHRA extended the licence of the cystic fibrosis medicines Kaftrio (ivacaftor, tezacaftor and elexacaftor) and Kalydeco (ivacaftor) for children aged two to five years old. Kaftrio and Kalydeco are taken together by patients as a long-term treatment to manage their symptoms of cystic fibrosis and are used to treat the disease in patients with at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
MHRA authorises world-first gene therapy that aims to cure sickle-cell disease and transfusion-dependent β-thalassemia
On 16th November, the MHRA authorised a new treatment for sickle-cell disease and transfusion-dependent β-thalassemia for patients aged 12. Casgevy is the first medicine to be licensed that uses the innovative gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020. It is designed to work by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin.
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