Tower Mains Weekly Newsletter
Regulatory Updates
MHRA guidance updates
- Project Orbis On 12th January, the Medicines and Healthcare products Regulatory Agency (MHRA) updated its guidance concerning Project Orbis and ILAP to express that it is no longer a requirement to obtain an Innovation Passport as part of the Project Orbis programme.’ Click to view source
- Notify the MHRA about a clinical investigation for a medical device. On 16th January, a template for submitting Quarterly Summary Reports was added to guidance on how to notify the MHRA of the intention to conduct a clinical investigation for medical devices. Click to view source
- Apply for a licence to market a medicine in the UK. On 17th January, the MHRA added further guidance on obtaining a PL number for applications. Click to view source
- Medicines: Marketing Authorisation Holders’ submission of Nitrosamine risk evaluation, risk assessment and confirmatory testing. On 17th January, additional guidance about lifecycle management and updated information following the passing of the deadline for the submission of steps 1, 2 and 3 were added to guidance on mitigating the risk of nitrosamine impurities being present in human medicinal products. Click to view source
FDA guidance updates
- Requests for reconsideration at the division level under GDUFA. On 10th January, the US Food & Drug Administration (FDA) published draft guidance providing recommendations on the procedures for applicants of abbreviated new drug applications (ANDAs) who wish to pursue a request for reconsideration within the review discipline at the division level or original signatory authority. This revision is being issued to reflect the most recent reauthorisation of the Generic Drug User Fee Amendments (GDUFA) and to clarify what matters are appropriate for requests for reconsideration. Click to view source
- Q5A(R2) viral safety evaluation of biotechnology products derived from cell lines of human or animal origin. On 10th January, final guidance was published intending to describe risk-based principles and mitigation strategies to assure the viral safety of biotechnology products, including the data necessary to submit in a marketing application. Click to view source
Good Clinical Practice & Pharmacovigilance Compliance Symposium
The FDA has published information on the joint Good Clinical Practice & Pharmacovigilance Compliance Symposium organised by the FDA, MHRA and Health Canada. From the 13th-15th of February 2024, the workshop will focus on Global Clinical Trials in Good Clinical Practice, Bioequivalence, and Pharmacovigilance in the post-pandemic world. Topics covered will include key updates to ICH E6(R3), the use of technology in clinical trials and clinical and bioanalytical challenges in bioequivalence studies.
FDA publishes report on public meeting on mitigating clinical study disruptions
On 16th January, the FDA announced it has published a report on topics discussed at the October 2023, public meeting, “Mitigating Clinical Study Disruptions During Disasters and Public Health Emergencies (PHEs).” The report provides a summary of the meeting and satisfies a mandate under Section 3605(b) of the Food and Drug Omnibus Reform Act of 2022 (FDORA) which requires the agency to publish a report within 90 days of the meeting on topics discussed at the meeting.
Industry Updates
Merck’s KEYTRUDA® receives US approval for FIGO 2014 Stage III-IVA cervical cancer
On 12th January, Merck, known as MSD outside of the US and Canada, announced that the FDA has approved KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with chemoradiotherapy (CRT) for the treatment of patients with FIGO (International Federation of Gynaecology and Obstetrics) 2014 Stage III-IVA cervical cancer.
Potential risk of neurodevelopmental disorders in children born to men treated with valproate medicines
On 12th January, the European Medicines Agency’s safety committee (PRAC) recommended precautionary measures for the treatment of male patients with valproate medicines. These measures are to address a potential increased risk of neurodevelopmental disorders in children born to men treated with valproate during the 3 months before conception. The PRAC recommends that valproate treatment in male patients is started and supervised by a specialist in the management of epilepsy, bipolar disorder, or migraine. The MHRA has subsequently announced an update on the new safety measures being introduced in UK for male and female patients.
FDA announces open period for Kratom study
On 16th January, the FDA announced an open period for applications to support the development, implementation, and evaluation of a human abuse potential (HAP) study on the use of botanical kratom. Addressing the use of botanical Kratom is a priority for the FDA as it works to characterise its abuse potential and subjective effects, given the absence of published literature and clinical evaluations despite increased human use.
Vertex announces US approval of CASGEVY™ for treatment of transfusion-dependent beta-thalassemia
On 16th January, Vertex Pharmaceuticals Incorporated announced that the FDA has approved CASGEVY™, a CRISPR/Cas9 gene-edited cell therapy, for the treatment of patients 12 years of age and older with transfusion-dependent beta-thalassemia. The approval signifies a second FDA-approved indication for the therapy, as it was also recently approved for the treatment of sickle cell disease in patients 12 years and older.
EMA publishes 2023 highlights
On 16th January, the EMA published its highlights of 2023. The overview includes figures on the authorisation of medicines and a selection of new treatments that represent significant progress in their therapeutic areas.
AbbVie-backed DISCO Pharmaceuticals launches with €20m in funding
On 16th January, DISCO Pharmaceuticals, a specialist biotech focused on unlocking the surfaceome of cancer cells at scale to identify new targets and develop first-in-class drugs, launched with €20 million in funding, which was backed by a world-leading investor syndicate, including Sofinnova Partners, Panakes Partners, M Ventures and AbbVie Ventures. The company has also announced the completion of the first-ever map of the surfaceome of a cancer type, Small Cell Lung Cancer (SCLC), and is developing proprietary antibody-based treatments for the cancer, which has historically been difficult to treat
ERS Genomics and StemSight announce collaboration
On 17th January, ERS Genomics Limited (ERS) – a CRISPR licensing company – and StemSight, a biotechnology company developing stem cell-based therapies for corneal blindness, announced a non-exclusive CRISPR/Cas9 license agreement allowing StemSight access to ERS’ CRISPR/Cas9 patent portfolio.
Synendos receives green light to begin a phase 1 study on its Endocannabinoid System modulator, SYT-510
On 18th January, Synendos Therapeutics AG, a world leader in innovative Endocannabinoid System (ECS) treatments, announced that has received approval from the European Medicines Agency (EMA) to commence the Phase 1 ‘first-in-human’ clinical trial of its lead asset, SYT-510, a first-in-class inhibitor that modulates a newly identified drug target in the Endocannabinoid System to restore healthy brain physiology.
Issue Number: WN00145
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