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Tower Mains Weekly Newsletter 8th August 2024
Regulatory Updates
Health Canada announces consultation on expanded access to clinical trials
On 2nd August, Health Canada launched a consultation on draft guidance on expanded access clinical trials. Expanded access clinical trials are a type of clinical trial that provides access to investigational drugs that have the potential to treat people living with medical conditions who do not qualify for other clinical trials or are not able to participate in one. The consultation will close on the 31st of October 2024.
MHRA updates
- Register medical devices to place on the market. On 2nd August, the Medicines and Healthcare products Regulatory Agency (MHRA) updated the ‘Account Management Reference Guide’ & ‘Device Registration Reference Guide’ to reflect changes to the registration system. Click to view source
- Export medical devices. On 2nd August, updated ‘Certificates of Free Sale for Medical Devices’ to reflect changes to the registration and Certificates of Free Sale system. Click to view source
European Commission launches call for expressions of interest for the EMA PRAC Committee
On 2nd August, the European Commission announced the launch of a selection procedure to appoint Independent Scientific Experts to the Pharmacovigilance Risk Assessment Committee (PRAC) of the European Medicines Agency (EMA) representing healthcare professionals and patient organisations. The successful candidates will be appointed for a three-year mandate from 2nd May 2025.
ICH to release RFI on Pharmaceutical Quality Knowledge Management
On 7th August, the International Council for Harmonisation (ICH) announced it would be releasing a Request for Information (RFI) to determine interest and potential approaches to support the ICH Pharmaceutical Quality Knowledge Management (PQKM) Task Force’s initial assessment for a secure standardised regulatory platform. The PQKM aims to enhance quality management practices in the pharmaceutical industry by leveraging knowledge management techniques and will focus on developing guidelines and frameworks to support this effort.
FDA updates
- M12 Drug Interaction Studies. On 8th August, the US Food & Drug Administration (FDA) published final guidance providing general recommendations on evaluating the enzyme and transporter-mediated pharmacokinetic drug-drug interaction potential for investigational drugs and the supplemental questions and answers document provides clarity to some concepts related to the evaluation of drug interactions covered in the guidance. Click to view source
Industry Updates
£28.5m in funding for Human Functional Genomics Initiative
On 1ST August, it was announced that the Medical Research Council (MRC), in collaboration with the Biotechnology and Biological Sciences Research Council (BBSRC), has provided £28.5 million in funding to establish the Human Functional Genomics Initiative programme. The initiative aims to advance our understanding of how genomic variation influences human physiology and how it changes over time and in disease.
Professor Steffen Petersen to become interim Director of the BHF Data Science Centre
On 1st August, the British Heart Foundation (BHF) announced that Professor Steffen Petersen is to become interim Director of the BHF Data Science Centre at Health Data Research UK. Professor Petersen takes over from founding Director Professor Cathie Sudlow, who is stepping down to lead a major new study.
EMA launches pilot programme to support orphan medical devices
On 2nd August, the European Medicines Agency (EMA) announced the launch of a pilot programme for expert panels to support the development and assessment of orphan medical devices in the EU. The pilot offers free advice from the medical device expert panels to selected manufacturers and notified bodies on the orphan device status and the data needed for their clinical evaluation and whilst it is scheduled to run until the end of 2025, the aim is to establish a long-term process for orphan device support.
FDA approves first gene therapy to treat adults with metastatic synovial sarcoma
On 2nd August, the FDA approved Tecelra, a gene therapy indicated for the treatment of adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are HLA antigen(s) A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive, and whose tumour expresses the MAGE-A4 antigen as determined by FDA authorised companion diagnostic devices.
FDA’s DHCoE shares latest health advancements to protect and promote public health
On 5th August, the FDA’s Digital Health Center of Excellence (DHCoE) published its summer newsletter sharing the latest digital health advancements to protect and promote public health. In this issue, DHCoE explores concepts and potential approaches for addressing questions on how to support the proper integration of Artificial Intelligence (AI) across the healthcare ecosystem and how to help AI reach its full potential in healthcare while reducing the risks and challenges presented by new technology.
CDRH launches discussion paper on advancing health equity in the context of medical devices
On 5th August, the FDA’s Center for Devices and Radiological Health (CDRH) released a Discussion Paper: Health Equity for Medical Devices to obtain feedback about ways to help ensure that all patients have timely access to safe, effective, and high-quality medical devices and safe radiation-emitting products. This paper requests feedback on possible considerations that may help inform the design of a clinical study that adequately reflects the intended use population for a particular medical device. The last day to submit comments is the 4th of October 2024.
First nalmefene hydrochloride auto-injector to reverse opioid overdose approved in US
On 7th August, the FDA approved Zurnai, the first nalmefene hydrochloride auto-injector for the emergency treatment of known or suspected opioid overdose in adults and paediatric patients 12 years of age and older. The approval of Zurnai is supported by safety and pharmacokinetic studies, as well as a study in healthy individuals who use opioids recreationally, to assess how quickly the product works. The agency approved the first nasal spray formulation of nalmefene in May 2023.
Ground breaking nipocalimab published in The New England Journal of Medicine
On 7th August, Johnson & Johnson announced the results from the Phase 2 open-label UNITY study of nipocalimab for the treatment of alloimmunizeda pregnant individuals at risk of early-onset severe (EOS) HDFN have been published in The New England Journal of Medicine (NEJM). The UNITY study met its primary endpoint with 54 per cent of individuals receiving nipocalimab achieving a live birth at or after 32 weeks gestational age (GA) without the need for IUT.1.
NHS England to roll out Vertex/CRISPR gene therapy for rare blood disorder
On 7th August, Vertex Pharmaceuticals announced an agreement with NHS England for eligible transfusion-dependent beta-thalassemia (TDT) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY™. The administration of the therapy requires experience in stem cell transplantation and the management of hemoglobinopathies; therefore, Vertex is engaging with experienced hospitals throughout England to establish a network of independently operated authorised treatment centres (ATCs).
Issue Number: WN00174
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