Tower Mains Weekly Newsletter
Regulatory Updates
Timeframe for accepting CE-marked medical devices in Great Britain extended
On 1st July, the Medicines and Healthcare products Regulatory Agency (MHRA) announced regulations (The Medical Devices (Amendment) (Great Britain) Regulations 2023) that enable CE-marked medical devices to be accepted in Great Britain for defined periods beyond 30th June 2023. This aims to support the ongoing and safe supply of medical devices and facilitate a smooth transition towards a future strengthened regulatory framework for medical devices.
ICMRA issues statement on the safety of COVID-19 vaccines
On 5th July, the International Coalition of Medicines Regulatory Authorities (ICMRA) issued a statement providing important information regarding the safety of COVID-19 vaccines, which have now been in use for more than two years. It also highlights that vaccines reduce the impact of long COVID based on several real-world data studies and that there is no safety signal from the very large data set held by international regulators suggesting that this condition is a possible side effect of COVID-19 vaccination.
MHRA updates guidance on how to register medical devices to place on the market
On 6th July, the MHRA updated its guidance on how to register medical devices to place on the markets in the UK and Northern Ireland to reflect an increase in registration fees, effective immediately.
FDA guidance updates
- Patient-matched guides to orthopaedic implants. On 27th June, the US Food and Drug Administration (FDA) published draft guidance providing recommendations on information to support premarket submissions for patient-matched guides to orthopaedic implants. The recommendations reflect current review practices and are intended to promote consistency and facilitate efficient review of submissions for patient-matched guides to orthopaedic implants. The guidance also provides recommendations that manufacturers should consider when developing their design process for these device types. Click to view source
- Chronic Rhinosinusitis with Nasal Polyps: Developing Drugs for Treatment.On 29th June, final guidance was published to assist sponsors in the development of drugs or biological products for the treatment of chronic rhinosinusitis with nasal polyps (CRSwNP). Specifically, this guidance addresses the FDA’s current thinking regarding trial population and design, effectiveness, statistical analysis, and safety for drugs being developed for the treatment of CRSwNP. Click to view source
Industry Updates
Reflection Paper on Harmonisation of RWE Terminology available for public consultation
On 30th June, the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Reflection Paper on Harmonisation of Real-World Evidence (RWE) Terminology was made available for public consultation until thee 30th of September 2023
FDA approves first cellular therapy to treat patients with type 1 diabetes
On 28th June, the FDA approved Lantidra, the first allogeneic pancreatic islet cellular therapy made from deceased donor pancreatic cells for the treatment of type 1 diabetes. Lantidra is approved for the treatment of adults with type 1 diabetes who are unable to approach target glycated hemoglobin because of current repeated episodes of severe hypoglycemia (low blood sugar) despite intensive diabetes management and education.
FDA approves first gene therapy for adults with severe haemophilia A
On 29th June, the FDA approved Roctavian, an adeno-associated virus vector-based gene therapy for the treatment of adults with severe haemophilia A without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test. Roctavian is a one-time gene therapy product administered as a single dose by intravenous infusion and consists of a viral vector carrying a gene for clotting Factor VIII.
Novartis publishes new five-year efficacy data on Kesimpta® for the treatment of relapsing multiple sclerosis
On 30th June, Novartis revealed pivotal long-term data from its ALITHIOS open-label extension study at the European Academy of Neurology (EAN) Annual Meeting held in Hungary on 1st-4th July 2023. The data demonstrated the sustained efficacy of continuous Kesimpta® (ofatumumab) treatment over five years in patients with relapsing forms of multiple sclerosis (RMS) and patients treated with Kesimpta experienced profoundly suppressed relapse rates, reduced MRI lesions and high rates of no evidence of disease activity (NEDA-3).
WHO launches the TRACK initiative for capacity building and knowledge sharing on advanced HIV disease
On 1st July, the World Health Organisation (WHO) announced the launch of “TRACK”, a new digital learning series for capacity building and knowledge sharing on advanced HIV disease (AHD). This is an integral part of a broader WHO response, in collaboration with partners, for AHD activities that will include in-person workshops, development of AHD-related disease estimates, creation of an AHD innovation pipeline for prioritisation, considerations for ART in the context of AHD, broadening the role of palliative care in the management of AHD, as well as considerations for the update of the guidelines for AHD in 2024.
Supporting patient access to innovation at the heart of MHRA’s Corporate Plan 2023 to 2026
On 4th July, the MHRA published its Corporate Plan 2023 to 2023, setting out the central priorities for the agency over the next three years so that it can deliver on this core purpose. Patients remain at the heart of the agency’s focus, with plans to further embed meaningful patient involvement across the agency’s regulatory pathways and to develop efficacy and safety information that better meets the needs of all patients. The priorities in the Corporate Plan are to:
- Maintain public trust through transparency and proactive communication.
- Enable healthcare access to safe and effective medical products.
- Deliver scientific and regulatory excellence through strategic partnerships.
- Become an agency where people flourish alongside a responsive customer service culture.
EAMS scientific opinions
- Glofitamab in the treatment of diffuse large B-cell lymphoma (DLBCL). On 5th July, Early Access to medicines scheme (EAMS) scientific opinion was issued to Roche Products Limited for glofitamab as monotherapy in the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). Click to view source
- Dostarlimab in the treatment of endometrial cancer. On 5th July, EAMS scientific opinion was issued to GlaxoSmithKline UK Limited for dostarlimab in combination with platinum-containing chemotherapy for the treatment of adult patients with mismatch repair deficient (dMMR) / microsatellite instability-high (MSI-H) primary advanced or recurrent endometrial cancer and who are candidates for systemic therapy. Click to view source
GeneCode receives record funding for Parkinson’s treatment
On 5th July, GeneCode, a company focused on the development of disease-modifying therapeutics to combat neurodegeneration, announced that the European Innovation Council (EIC) Accelerator is providing €16 million to support the development of a drug and treatment to slow the progression of Parkinson’s disease.
Issue Number: WN00119
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