Our weekly newsletter provides you with the latest industry news, developments and regulatory agency updates.
Regulatory Updates
MHRA guidance updates
- Pharmacovigilance following agreement of the Windsor Framework. On 30th August, the Medicines and Healthcare products Regulatory Agency (MHRA) published guidance on the implementation of changes to pharmacovigilance for medicines authorised in the UK following the agreement of the Windsor Framework. Click to view source
- UK-wide licensing for human medicines. On 30th August, the MHRA updated Information on the implementation of changes to the licensing of medicines for human use in the UK following the agreement of the Windsor Framework. Click to view source
MHRA issues key advice on children’s medicines and medical devices
The MHRA has issued tips for children taking medicines or using medical devices to coincide with the new school year. This includes reassurance that childhood vaccinations are safe and effective against disease and safeguarding around anaphylaxis.
FDA guidance updates
- Bioresearch Monitoring Technical Conformance Guide. On 3rd September, the US Food and Drug Administration (FDA) published the Bioresearch Monitoring Technical Conformance Guide. This guide outlines current FDA specifications, recommendations, and general considerations for preparing and submitting Clinical Study-Level Information, Subject-Level Data Line Listings by Clinical Site, and a Summary-Level Clinical Site Dataset that are used by the Center for Drug Evaluation and Research (CDER) for planning of Bioresearch Monitoring (BIMO) inspections in electronic format for new drug applications (NDAs), biologics license applications (BLAs), and NDA or BLA supplemental applications containing clinical data that are regulated by CDER. It also applies when these data and information are submitted under certain investigational new drug applications (INDs) in advance of a planned NDA, BLA, or supplemental submission. Click to view source
- Control of Nitrosamine Impurities in Human Drugs. On 5th September, final guidance was published with recommendations on steps manufacturers of active pharmaceutical ingredients (APIs) and drug products should take to detect and prevent unacceptable levels of nitrosamine impurities in pharmaceutical products. It also describes two general structural classes of nitrosamine impurities: small-molecule nitrosamine impurities (i.e., nitrosamine impurities that do not share structural similarity to the API), and nitrosamine drug substance-related impurities (NDSRIs), which share structural similarity to the API and are generally unique to each API. Click to view source
Industry Updates
Appointments of four MHRA Non-Executive Directors extended until August 2025
On 29th August, the MHRA announced that Dr Junaid Bajwa, Professor Graham Cooke, Dr Paul Goldsmith and Rajakumari Long have had their appointments as Non-Executive Directors (NEDs) extended from 1st September 2024 until 31st August 2025.
Emergent’s ACAM2000 vaccine approved in US for mpox prevention in high-risk individuals
On 29th August, the FDA approved a new indication for Emergent’s ACAM2000, a live replicating vaccinia virus vaccine, to include preventing mpox disease in individuals at high risk for mpox infection. ACAM2000 has been approved since 2007 for the prevention of smallpox disease in individuals determined to be at high risk for smallpox infection.
FDA grants EUA for updated Novavax COVID-19 vaccine targeting current variants
On 30th August, the FDA granted an emergency use authorisation (EUA) for an updated version of the Novavax COVID-19 vaccine that more closely targets currently circulating variants to provide better protection against serious consequences of COVID-19, including hospitalisation and death. The updated vaccine is authorised for use in individuals aged 12 and older and contains a monovalent component specific to the Omicron variant JN.1 strain of SARS-CoV-2.
Merck’s KEYTRUDA® plus Padcev® approved as first-line treatment of advanced urothelial carcinoma in EU
On 3rd September, Merck announced that the European Commission has approved KEYTRUDA® (pembrolizumab) in combination with Padcev® (enfortumab vedotin-ejfv) as a first-line treatment for adults with unresectable or metastatic urothelial carcinoma. This decision is based on clinical guidelines from the European Society for Medical Oncology and the European Association of Urology, recommending the combination therapy for these patients, regardless of their eligibility for platinum-based treatments.
BlueRock receives FDA clearance for IND application of OpCT-001 cell therapy for photoreceptor diseases
On 3rd September, BlueRock Therapeutics, a clinical-stage cell therapy company and wholly owned, independently operated subsidiary of Bayer AG, announced the clearance of its Investigational New Drug (IND) application by the FDA for OpCT-001, an investigational induced pluripotent stem cell (iPSC)-derived cell therapy for the treatment of primary photoreceptor diseases. OpCT-001 is the first iPSC-derived investigational cell therapy to be clinically tested for treating primary photoreceptor diseases, a subgroup of inherited retinal disorders that affect the structure and function of the photoreceptor cells in the retina, leading to irreversible vision loss in children and adults. BlueRock plans on conducting a Phase 1/2a clinical study.
Valneva and Pfizer report positive Phase 2 data for Lyme disease vaccine candidate
On 3rd September, Valneva SE and Pfizer Inc. announced positive immunogenicity and safety data from their VLA15-221 Phase 2 study. The immune response and safety profile of VLA15 one month after receiving the second booster dose were like those reported after receiving the first booster dose, showing compatibility with the anticipated benefit of a booster vaccination before each Lyme season. There are currently no approved human vaccines for Lyme disease, and VLA15 is the Lyme disease vaccine candidate which has advanced the furthest along the clinical development timeline, with two Phase 3 trials in progress.
AMLo Biosciences’ prognostic melanoma biomarker receives UKCA Mark
On 3rd September, AMLo Biosciences Ltd announced the successful receipt of the UKCA mark for AMBLor, a histopathological biomarker test for the personal identification of early-stage melanomas at low risk of progression, post-staging diagnosis. It is the first in vitro diagnostic (IVD) of its kind that can reliably stratify non-ulcerated AJCC stage I-II melanomas that are unlikely to metastasise, potentially segmenting a group of patients that could be spared further diagnostic procedures and lengthy follow-up schedules.
MHRA finds evidence does not support link between GLP-1 receptor agonists and suicidal and self-injurious thoughts and actions
On 4th September, the MHRA announced that current evidence does not establish a causal link between Glucagon-Like Peptide-1 (GLP-1) receptor agonists and suicidal behaviour, suicidal thoughts, self-injury, or depression. The MHRA’s evaluation of post-marketing data in the UK aligns with the findings of a European regulatory review, which examined data from various sources, including post-marketing reports, clinical trials, epidemiological studies, and scientific literature. The conclusion is that the available data does not support an association between GLP-1 receptor agonists and these mental health issues, and therefore, no updates to the product information are currently necessary.
Issue Number: WN00178
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