Tower Mains Weekly Newsletter
Regulatory Updates
MHRA launches pathway for innovative technologies
On 19th September, the Medicines and Healthcare products Regulatory Agency (MHRA) announced the launch of the Innovative Devices Access Pathway (IDAP), a program designed to accelerate the development of cost-effective medical devices and their integration into the UK market. The IDAP, supported by £10 million of Government funding, has the potential to accelerate UK patient access to innovative technologies by providing innovators and manufacturers with multi-partner support and targeted scientific advice. Guidance was subsequently released, providing further information on the pilot.
Further MHRA guidance updates
- Medical devices: EU regulations for MDR and IVDR (Northern Ireland). On 21st September, guidance was updated on the EU Regulations for medical devices (MDR) and in vitro diagnostic medical devices (IVDR). Click to view source
- Medicines, medical devices and blood regulation and safety: Manufacturing, wholesaling, importing and exporting medicines. On 21st September, a collection of guidance was published on applications and licences for manufacturers & wholesalers, suspended and revoked licences, registrations, exporting medicines, defective medicinal products and fees. Click to view source
- Medicines, medical devices and blood regulation and safety: Clinical trials and investigations. On 21st September, a collection of guidance was published providing information on clinical trials for medicines, how to apply for authorisation in the UK, how to manage your authorisation, reporting safety issues and details about the MHRA phase I accreditation scheme. Click to view source
EMA issues Q&A for biological medicinal products
The European Medicines Agency (EMA) has published questions and answers for biological medicinal products. Topics include reprocessing process validation and biosimilar medicinal products. This has been developed and is maintained by the EMA’s Committee for Medicinal Products for Human Use (CHMP) Biologics Working Party (BWP) and provides agreed positions by the Biologics Working Party position on issues that can be subject to different interpretations or require clarification, typically arising from discussions or correspondence during assessment procedures of biological human medicinal products.
FDA guidance updates
- CVM GFI #282 Informed Consent Forms for Studies that Enrol Client-Owned Companion Animals. On 15th September, the US Food and Drug Administration (FDA) published draft guidance detailing recommendations on ICFs used for studies that enrol client-owned companion animals (dogs, cats, and horses). CVM recommends all studies conducted with client-owned companion animals use an ICF and be conducted per Good Clinical Practice guidelines. Click to view source
- Labelling for Biosimilar and Interchangeable Biosimilar Products. On 18th September, draft guidance was published intended to help applicants develop draft labelling for proposed biosimilar and interchangeable biosimilar products for submission in an application under section 351(k) of the Public Health Service Act (PHS Act) (42 U.S.C. 262(k)) (351(k) application). Click to view source
- Demonstrating Substantial Evidence of Effectiveness Based on One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence. On 19th September, draft guidance was published providing recommendations for sponsors to consider when planning a drug development program. This guidance emphasises the importance of early engagement with the agency for sponsors that intend to establish substantial evidence of effectiveness with one adequate and well-controlled clinical investigation and confirmatory evidence. Click to view source
- Alternative Tools: Assessing Drug Manufacturing Facilities Identified in Pending Applications On 20th September, draft guidance was published to provide information to applicants on how the FDA intends to use alternative tools to assess manufacturing facilities identified in a marketing application (i.e., a new drug application (NDA), an abbreviated new drug application (ANDA), a biologics license application (BLA), or a supplement to any of these types of applications). Click to view source
Industry Updates
Nona Biosciences and BeiGene expand antibody discovery collaboration
On 14th September, Nona Biosciences, a company committed to cutting-edge antibody technology innovations, announced an agreement with BeiGene, Ltd. to expand the companies’ discovery strategic collaboration leveraging Nona’s proprietary Harbour Mice® H2L2 platform. The collaboration will allow BeiGene to utilise Nona’s platform to further improve therapeutic antibody discovery efficiency and flexibility.
Astellas Pharma to invest more than €330 million in new state-of-the-art facility in Ireland
On 14th September, Astellas Pharma Inc. announced its intent to submit a planning application to build a new state-of-the-art facility, at an approximate cost of €330 million in Tralee, Co. Kerry. This investment will expand the company’s capacity and capabilities for aseptic drug products, reinforce stable production for global supply and accelerate the development and commercialisation of innovative antibody drugs and other new products.
MHRA approves adapted Spikevax COVID-19 vaccine that targets Omicron XBB.1.5
On 15th September, the MHRA announced the approval of an adapted Spikevax COVID-19 vaccine that targets the Omicron XBB 1.5 subvariant after it was found to meet the UK regulator’s standards of safety, quality and effectiveness.
EMA recommends non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna
On 15th September, the EMA’s CHMP recommended not renewing the marketing authorisation for Translarna, a medicine for treating patients with Duchenne muscular dystrophy whose disease is caused by a type of genetic defect called a ‘nonsense mutation’ in the dystrophin gene and who can walk. The recommendation follows the full re-evaluation of the benefits and risks of the medicine during the renewal of its marketing authorisation, including the results of a new study which failed to confirm Translarna’s effectiveness.
EMA recommends non-renewal of authorisation of multiple myeloma medicine Blenrep
On 15th September, the EMA’s CHMP recommended not renewing the conditional marketing authorisation for Blenrep, a medicine used to treat multiple myeloma. Blenrep is given to adults who have received at least four previous treatments and whose disease does not respond to certain other types of cancer treatment, and whose cancer has worsened since receiving the last treatment.
EMA and EUnetHTA consortium ceases operations
On 15th September, it was announced that the EMA and European Network for Health Technology Assessment (EUnetHTA 21) consortium will cease operations but preparations will continue for the implementation of the Regulation, under the direction of the HTA Coordination Group. EMA’s collaboration with EUnetHTA began in 2010 as a project to test out whether early engagement between regulators and HTA bodies could bring tangible benefits for patient access to medicines and most recently, it delivered several milestones to prepare the EU for entry into application of the Regulation on Health Technology Assessment. More information on the consortium’s achievements is available here.
FDA issues Digital Health FAQs
On 15th September, the FDA issued FAQs on digital health. Digital health technologies are advancing rapidly and the FDA encourages digital health developers to contact the Center for Devices and Radiological Health’s (CDRH) Digital Health Center of Excellence (DHCoE) throughout the development process with questions about digital health regulatory considerations.
FDA to take further steps to modernise its approach to innovative animal and veterinary products
On 15th September, the FDA announced it is taking new steps to further modernise its approach to evaluating and supporting the development of innovative animal and veterinary products, to increase regulatory flexibility, predictability and efficiency. The Animal and Veterinary Innovation Agenda details objectives and supporting actions by the agency’s Center for Veterinary Medicine (CVM) to foster product development and implement smart, risk-based approaches to regulating modern animal and veterinary products. These actions will further position the agency for continued scientific and technological evolution while helping to bring safe and effective products to the market.
Evaxion announces new vaccine project in collaboration with a leading pharmaceutical company
On 18th September, Evaxion Biotech A/S, a clinical-stage biotechnology company specialising in the development of AI-powered immunotherapies, announced the initiation of a novel pipeline program, EVX-B3, an AI-designed vaccine candidate against an undisclosed bacterial pathogen. The new vaccine project will be a collaboration between Evaxion and an undisclosed leading pharmaceutical company, leveraging unique assets and knowledge from both organisations.
FDA’s IT Strategy: Unlocking Potential, Leading Transformation
On 19th September, the FDA published the FDA Voices: “FDA’s IT Strategy: Unlocking Potential, Leading Transformation,” by Vid Desai, Chief Information Officer, and Jessica N. Berrellez, Executive Officer. The agency considers the new IT Strategy more than a roadmap: it’s a shared vision that unites the FDA’s IT portfolio and will foster innovation, enhance public health outcomes, and lead the FDA into a new era of scientific and technological advancement.
Cancer Research UK announces largest-ever investment in Scotland
On 20th September, Cancer Research UK (CRUK) announced its largest-ever investment in Scotland of up to £123m as part of a seven-year commitment to the Cancer Research UK Scotland Institute (formerly known as the Cancer Research UK Beatson Institute). The institute, which is facilitated in partnership with the University of Glasgow, will use the investment to support research into new and improved cancer treatments and diagnosis, as well as bolster Scotland as a major global hub for cancer research.
Issue Number: WN00130
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