Our weekly newsletter provides you with the latest industry news, developments and regulatory agency updates.
Regulatory Updates
FDA updates
- Pediatric Inflammatory Bowel Disease: Developing Drugs for Treatment. On 22nd July, the US Food & Drug Administration (FDA) published draft guidance providing its recommendations regarding the necessary attributes of clinical studies for drugs being developed for the treatment of paediatric ulcerative colitis or paediatric Crohn’s disease, including study population, study design, efficacy considerations, and safety assessments. Click to view source
- Laboratory Developed Tests FAQs. On 22nd July, the FDA published common questions received related to laboratory-developed tests (LDTs). To ensure transparency, the FDA intends to respond to generally applicable questions, as appropriate, in a public manner, such as in webinars, guidances, FAQ page, and other resources throughout the phaseout period for the general enforcement discretion to help assure the safety and effectiveness of in vitro diagnostic products offered as LDTs. It also intends to update the FAQ page periodically. Click to view source
MHRA updates
- Good clinical practice for clinical trials. On 22nd July, the Medicines and Healthcare products Regulatory Agency (MHRA) added an infringement notice for Celixir to its guidance on how to show MHRA you’re meeting good clinical practice (GCP) standards and what to expect from an inspection. Click to view source
EMA launches event on the guideline on good pharmacovigilance practices Module XVI on risk minimisation measures and its Addendum II
The European Medicines Agency (EMA) announced an online event to launch the revised guideline on good pharmacovigilance practices (GVP) Module XVI and its Addendum II. The event will provide an overview of how stakeholder comments from the public consultation on these documents have been addressed and will be followed by a questions and answers session for answering audience questions.
Industry Updates
FDA to launch rare disease innovation hub to advance outcomes for patients
On 17th July, the FDA published the FDA Voices: “FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients,” by Patrizia Cavazzoni, M.D., director of the Center for Drug Evaluation and Research and Peter Marks, M.D., Ph.D., director of the Center for Biologics Evaluation and Research. The FDA plans to establish a Rare Disease Innovation Hub, which will work across rare diseases but will particularly focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood.
First patient recruited to innovative rare liver disease clinical trial in UK
On 18th July, the University of Birmingham announced the first patient has been recruited to the FARGO clinical trial, which is testing a new treatment to improve quality of life for patients living with primary sclerosing cholangitis (PSC), a rare liver disease. The trial, funded by LifeArc and PSC Support and run by the University of Birmingham, will investigate the potential of faecal microbiota transplantation (FMT) to correct the imbalance of the gut microbiome in people with PSC, with the hope that this could slow or even reverse the progression of the disease.
Workshop announced to discuss expansion of the ASCA program
On 18th July, the FDA announced a public workshop titled Accreditation Scheme for Conformity Assessment (ASCA) and Use of Chemical Analysis to Support Biocompatibility of Medical Devices that will be held on Wednesday, 6th November 2024 at the FDA’s White Oak campus in Silver Spring, Md., and via webcast. Its purpose is to discuss the possible expansion of the Accreditation Scheme for Conformity Assessment (ASCA) program to include chemical analysis to support the biocompatibility of medical devices.
MHRA approves the first generic raltegravir medicines to treat adult and paediatric patients infected by HIV
On 19th July, the MHRA approved the first generic raltegravir medicines to treat adult and paediatric Human Immunodeficiency Virus (HIV) patients who weigh at least 40kg. HIV produces an enzyme called HIV integrase which enables multiplication of the virus in cells within the body and raltegravir stops this enzyme from working. When used with other medicines it may reduce the amount of HIV in the patient’s blood and increase the patient’s CD4-cell count.
EMA releases highlights from latest CVMP meetings
On 19th July, the EMA issued meeting highlights from the Committee for Veterinary Medicinal Products (CVMP), which was held on the 16th-18th of July. Topics include opinions on veterinary medicinal products, new safety information for healthcare professionals and legislation.
Agilent to acquire CDMO Biovectra
On 22nd July, Agilent Technologies Inc. announced it has signed a definitive agreement to acquire Biovectra, a leading specialised contract development and manufacturing organisation, for $925 million. The acquisition expands Agilent’s end-to-end biopharma solutions that accelerate drug discovery, development, and manufacturing.
MHRA publishes latest Drug Safety Update
On 23rd July, the MHRA published the July edition of the Drug Safety Update newsletter from MHRA and its independent advisor, the Commission on Human Medicines.
MHRA approves semaglutide to reduce risk of serious heart problems in obese or overweight adults
On 23rd July, the MHRA approved a new indication for semaglutide to reduce the risk of overweight and obese adults suffering serious heart problems or strokes. The approval means that semaglutide is the first weight loss drug to be prescribed to prevent cardiovascular events, such as cardiovascular death, non-fatal heart attack and non-fatal stroke, in people with established cardiovascular disease and a Body Mass Index (BMI) higher or equal to 27 kg/m2.
Study suggests targeting amyloid beta production could lead to promising treatments for Alzheimer’s
On 23rd July, it was announced that a study, led by the UK Dementia Research Institute (UK DRI) and University College London (UCL), has suggested targeting amyloid beta production in one cell type in the brain could improve early disease outcomes, with fewer side effects, in Alzheimer’s disease (AD). The research could lead to new specific treatments for the condition that exhibit fewer side effects.
Merck announces late-stage study success for RSV antibody
On 23rd July, Merck announced positive topline results from its Phase 2b/3 clinical trial (MK-1654-004) evaluating clesrovimab (MK-1654), the company’s investigational prophylactic monoclonal antibody designed to protect infants from respiratory syncytial virus (RSV) disease. Detailed results will be presented at an upcoming scientific congress, and Merck plans to submit the data to global regulatory authorities.
UKRI launches eight research networks to combat antimicrobial resistance
On 23rd July, the UK Research and Innovation (UKRI) announced the establishment of eight new research networks to combat antimicrobial resistance (AMR). These networks, funded with £4.8 million, bring together diverse experts to develop innovative, cross-disciplinary solutions. Key areas of focus include diagnostics, fungal resistance, behavioural changes in antibiotic use, and the impact of climate change on AMR. The initiative aims to enhance the UK’s ability to tackle infectious diseases and AMR.
Pfizer announces positive topline results from phase 3 study of haemophilia a gene therapy candidate
On 24th July, Pfizer Inc. announced positive topline results from the Phase 3 AFFINE study (NCT04370054) evaluating giroctocogene fitelparvovec, an investigational gene therapy for the treatment of adults with moderately severe to severe haemophilia A.
Issue Number: WN00172
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