Our weekly newsletter provides you with the latest industry news, developments and regulatory agency updates.
Regulatory Updates
FDA guidance updates
- Drugs for the Treatment of Partial Onset Seizures: Extrapolation of Efficacy from Adults to Pediatric Patients 1 Month of Age and Older. On 15th July, the US Food & Drug Administration (FDA) published final guidance providing recommendations to sponsors on the clinical development of drugs for the treatment of partial-onset seizures (POS) in paediatric patients. Specifically, this guidance addresses the FDA’s current thinking regarding clinical development programs that can support extrapolation of the efficacy of drugs approved for the treatment of POS in adults to paediatric patients of one month of age and older. Click to view source
- Application User Fees for Combination Products. On 16th July, final guidance was published with information on application user fees for combination products as defined under 21 CFR 3.2(e). It also addresses how the FDA applies user fees for combination products when separate applications are submitted for the constituent parts. Click to view source
- Pediatric Inflammatory Bowel Disease: Developing Drugs for Treatment. On 16th July, the FDA announced the availability of draft guidance on the agency’s recommendations of necessary attributes of clinical studies for drugs being developed for the treatment of paediatric ulcerative colitis or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
- Blood Pressure and Pulse Donor Eligibility Requirements – Compliance Policy. On 17th July, the FDA issued final guidance addressing the regulatory requirements for determining donor eligibility that apply to establishments that collect blood and blood components for transfusion or for further manufacturing use, including Source Plasma. Click to view source
Industry Updates
WHO prequalifies the first self-test for hepatitis C virus
On 10th July, the World Health Organisation (WHO) announced the prequalification of the first self-test for hepatitis C, the OraQuick HCV self-test. This self-test, designed for lay users, complements existing HCV testing services and aims to boost diagnosis and treatment, particularly in low- and middle-income countries. The prequalification supports global efforts to eliminate hepatitis C by providing safe, effective, and stigma-free testing options.
NIHR and Diabetes UK partner to address diabetes distress
On 10th July, the National Institute for Health and Care Research (NIHR) announced that in partnership with Diabetes UK, it is investing nearly £3m to develop research to treat and prevent type 1 diabetes distress. The initiative entitled ‘D-Stress’ aims to improve the quality of life for individuals living with diabetes by exploring new interventions and support strategies to manage the emotional and psychological challenges associated with the condition.
Requested re-examination of non-renewal of authorisation of Duchenne muscular dystrophy medicine
On 11th July, the European Medicines Agency (EMA) announced that the company for Translarna has requested a re-examination of the agency’s June 2024 decision not to renew the marketing authorisation, which was based on a review of new data showing insufficient demonstration of Translarna’s clinical benefit in treating Duchenne muscular dystrophy (DMD). The EMA will re-examine its opinion upon receiving the grounds of this request and subsequently issue a final recommendation.
KCL leads first guideline on immune monitoring in patients with hard-to-treat blood cancer
On 12th July, King’s College London (KCL) announced the publication of first-in-their-kind guidelines that aim to standardise how clinicians measure the immune response of patients who have Myelodysplastic syndromes (MDS), a type of hard-to-treat blood cancer. The new guidelines give recommendations around flow cytometry and recommendations for seven panels for measuring immune response in MDS.
MHRA announces approvals
- On 12th July, ocrelizumab was approved as an injection treatment for adults with relapsing forms of multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS). The approval marks a significant advancement in treatment options for MS patients in the UK. Click to view source
- On 17th July, capivasertib (Truqap) was approved for patients with advanced hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative breast cancer with specific genetic mutations unresponsive to other therapies. Capivasertib, an AKT inhibitor, is combined with fulvestrant to block cancer cell growth and spread, showing significant efficacy in clinical trials by delaying disease progression. Click to view source
- On 17th July, vibegron (Obgemsa) was approved for treating overactive bladder syndrome (OAB) in adults. Vibegron, a beta-3 adrenergic receptor agonist, reduces bladder muscle activity and associated symptoms such as urgency, frequent urination, and urinary incontinence. Approval was based on a phase 3 trial demonstrating its effectiveness over 12 weeks. Click to view source
CDRH announces organisational changes to increase agility and advance public health mission
On 15th July, the FDA announced organisational changes within the Center for Devices and Radiological Health (CDRH) to help strengthen and better position the Center to meet the FDA’s mission to protect and promote public health.
FDA issues warning to MIT over non-compliance
On 16th July, the FDA issued a warning letter to the Massachusetts Institute of Technology (MIT) for non-compliance issues within its Institutional Review Board (IRB). Violations include inadequate informed consent forms, insufficient documentation of IRB activities, and repeated failures to ensure compliance with federal regulations, despite previous warnings. The FDA demands corrective actions and detailed responses from MIT to address these issues and ensure future compliance.
Repurposed drug improves outcomes for patients with severe COVID-19 pneumonia
On 16th July, the University College London (UCL) announced its researchers, along with collaborators from the University College London Hospitals NHS Foundation Trust (UCLH) and the Francis Crick Institute, have conducted a clinical trial that yielded promising results for treating severe COVID-19 pneumonia. The outcome indicated that dornase alfa – commonly used to treat cystic fibrosis – reduces hyper-inflammation and systemic inflammation, aiding recovery and reducing hospital stays. Further large-scale clinical trials are planned to confirm its effectiveness and explore its potential for treating other respiratory conditions.
Issue Number: WN00171
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