Tower Mains Weekly Newsletter
Regulatory Updates
MHRA guidance updates
- International Recognition Procedure. On 3rd October, the Medicines and Healthcare products Regulatory Agency (MHRA) published a webinar providing information on guidance on the international recognition route for medicines utilising pre-existing approvals from Australia, Canada, the European Union, Japan, Switzerland, Singapore, and the United States. Click to view source
Clinical Trials Regulation (EU) No 536/2014 Q&A
On 29th September, the European Commission published a questions and answers document on Clinical Trials Regulation (EU) No 536/2014, submitted for discussion to the Expert Group on Clinical Trials and through written procedure to the Clinical Trials Coordination and Advisory Group.
Health Canada implements ICH M7(R2): Assessment and Control of DNA Reactive (Mutagenic) Impurities in Pharmaceuticals to Limit Potential Carcinogenic Risk
On 29th September, Health Canada implemented the ICH M7(R2) guideline providing guidance for new drug substances and products during their clinical development and subsequent applications for marketing.
Defining ‘candy-like’ non-prescription drug products webinar
The US Food and Drug Administration (FDA), in partnership with the University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI), announced the public workshop “Defining ‘Candy-Like’ Non-prescription Drug Products.” The workshop aims to further define features of non-prescription drug products that could be considered ‘candy-like,’ and will bring together stakeholders Presentations and panel discussions will include various topics related to solid oral “candy-like” dosage forms, including formulation considerations for developing palatable drug products and implications of candy-like characteristics on medication adherence in paediatric and geriatric populations. The workshop will be held on Monday, 30th October from 8:30 am to 4pm (ET) both on-site at the FDA’s White Oak Campus (Silver Spring, MD) and virtually.
FDA guidance updates
- Electronic submission template for Medical Device 510(k) submissions. On 2nd October, final guidance was published, providing the standards for the submission of premarket notification (510(k)) submissions by electronic format, a timetable for the establishment of these standards, and criteria for waivers of and exemptions from the requirements to meet a statutory requirement. Click to view source
- Dose Banding for Drug and Biological Products. On 2nd October, final guidance was published, providing recommendations for incorporating dose banding information into the labelling of an injectable drug product that is seeking approval through a new drug application submitted under section 505(b) of the FD&C Act (21 U.S.C. 355(b)), a biologics license application submitted under section 351(a) of the PHS Act (42 U.S.C. 262(a)), or a supplement to one of these approved applications. Click to view source
- Developing drugs for treatment of stimulant use disorder. On 4th October, draft guidance was published providing the agency’s current thinking on the overall development program and clinical trial design to develop drugs and biologics to support the treatment of moderate to severe cocaine use disorder, methamphetamine use disorder and prescription stimulant use disorder. Click to view source
Industry Updates
FDA proposes rule aimed at ensuring the safety and effectiveness of Laboratory Developed Tests
On 29th September, the U.S. Food and Drug Administration (FDA) announced a proposed rule aimed at helping ensure the safety and effectiveness of Laboratory Developed Tests (LDTs), which are used in a growing number of healthcare decisions and for which concerns have been raised for many years. The rule seeks to amend the FDA’s regulations to make explicit that IVDs are devices under the Federal Food, Drug, and Cosmetic Act, including when the manufacturer of the IVD is a laboratory. Along with this amendment, the FDA is proposing a policy under which the agency intends to provide greater oversight of LDTs, through a phaseout of its general enforcement discretion approach to LDTs.
FDA launches pilot to accelerate the development of novel drug and biological products for rare diseases
On 29th September, the FDA announced the opportunity for a limited number of sponsors to participate in the Selected Participants of the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program. The pilot will allow participants to obtain frequent advice to address product-specific development issues, including, but not limited to, clinical study design, choice of control group and fine-tuning the choice of patient population.
FDA grants marketing authorisation for a DNA test to assess predisposition for certain cancer types
On 29th September, the FDA granted de novo marketing authorisation for the Invitae Common Hereditary Cancers Panel, an in vitro diagnostic test that can help detect hundreds of genetic variants associated with an elevated risk of developing certain cancers. The test can also help identify potentially cancer-associated hereditary variants in individuals with already-diagnosed cancer. The test, which is the first of its kind to be granted FDA marketing authorization, evaluates DNA extracted from a blood sample to identify variants in 47 genes known to be associated with an elevated risk of developing certain types of cancer.
MHRA authorises monoclonal antibody treatment for endometrial cancer
On 2nd October, the Medicines and Healthcare products Regulatory Agency (MHRA) announced the authorisation of a new indication for Jemperli, a treatment for some types of endometrial cancer in adults. Jemperli is now authorised to be used together with chemotherapy to slow the progression of these cancers, increasing life expectancy for patients. This is the first medicine licensed as a first-line treatment for primary advanced or recurrent endometrial cancer.
Scientists behind mRNA COVID vaccines awarded Nobel Prize in Medicine
On 2nd October, Dr. Kariko and Dr. Weissman were awarded the Nobel Prize in Physiology or Medicine for their discoveries concerning nucleoside base modifications that enabled the development of effective mRNA vaccines against COVID-19. Through their ground-breaking findings, the laureates contributed to the unprecedented rate of vaccine development during one of the greatest threats to human health in modern times.
NIHR invests £97.5 million in new Research Support Service
On 2nd October, the National Institute for Health and Care Research (NIHR) announced it is investing £97.5 million in a new Research Support Service. The RSS provides free and confidential support, advice, and expertise for all researchers in England working across the remit of the NIHR, and will provide support at every stage, from pre-application through to post-award delivery.
Capsida Biotherapeutics to collaborate with Kate Therapeutics to develop KateTx’s gene therapy medicines
On 3rd October, Capsida Biotherapeutics Inc. and Kate Therapeutics announced a collaboration to leverage Capsida’s expertise and adeno-associated virus (AAV) manufacturing capabilities to enable KateTx’s initial internal portfolio of muscle and heart disease programs. Under the terms of the agreement, Capsida will provide GMP manufacturing of KateTx’s gene therapies as the medicines advance through preclinical and clinical development, and Capsida will receive undisclosed funding for the term of the agreement.
Issue Number: WN00132
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