Tower Mains Weekly Newsletter

Regulatory Updates

MHRA guidance updates

  • MHRA performance data for assessment of clinical trials and established medicines. On 15th August, the Medicines and Healthcare products Regulatory Agency (MHRA) published performance metrics for clinical trials and established medicines assessment. Click to view source
  • COVID-19 test validation approved products. On 15th August, published updated ‘COVID-19 test validation approved products’ data. Click to view source

FDA guidance updates

  • Formal Meetings Between the FDA and Sponsors or Applicants of BsUFA Products Guidance for Industry. On 9th August, the US Food and Drug Administration (FDA) published draft guidance providing recommendations on formal meetings between the FDA and sponsors or applicants relating to the development and review of biosimilar or interchangeable biological products regulated by the Center for Drug Evaluation and Research (CDER) or the Center for Biologics Evaluation and Research (CBER). Click to view source
  • Off-The-Shelf Software Use in Medical Devices. On 11th August, final guidance was published to provide information regarding the recommended documentation sponsors should include in a pre-market submission for the FDA’s evaluation of off-the-shelf (OTS) software used in a medical device. This guidance describes information that would be typically generated and documented during software development, verification, and validation. Click to view source
  • Informed Consent. On 15th August, final guidance was published to assist institutional review boards (IRBs), clinical investigators, and sponsors in complying with FDA’s informed consent regulations for clinical investigations. This guidance supersedes FDA’s guidance entitled “A Guide to Informed Consent,” issued in September 1998, and finalises FDA’s draft guidance entitled “Informed Consent Information Sheet,” issued in July 2014. Click to view source

Industry Updates

atai Life Sciences reveals data supporting further exploration of the potential of R-ketamine as a rapid-acting anti-depressant for home use

On 8th August, atai Life Sciences, a clinical-stage biopharmaceutical company aiming to transform the treatment of mental health disorders, announced results from Perception Neuroscience’s Phase 1 intravenous-to-subcutaneous bridging study of PCN-101 (R-ketamine). The study was designed to inform dosing regimens of the new subcutaneous formulation that may optimize the therapeutic index—the balance of safety, tolerability, and efficacy—of PCN-101 in future studies, thereby supporting further exploration of the potential of R-ketamine as a rapid-acting anti-depressant for home use.

Click to view source

 CNX Therapeutics expands hospital-injectable portfolio by acquiring four cancer support therapies from Clinigen

On 10th August, CNX Therapeutics Limited, a speciality pharmaceutical company with a focus on improving access to medicines, is pleased to announce that it has completed the acquisition of four cancer support products from Clinigen Limited, a global pharmaceutical services company. The four therapies are Cardioxane® (dexrazoxane), Savene® (dexrazoxane), Totect® (dexrazoxane) and Ethyol® (amifostine), all of which help mitigate the side effects patients may experience when treated with other cancer therapies.

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CDER Conversation: FDA’s work with ICH to support global drug development

On 10th August, the FDA published a CDER Conversation where Theresa Mullin, PhD, Associate Center Director for CDER’s Strategic Initiatives, talks about CDER’s important work in the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH).  A major part of the FDA’s regulatory harmonisation effort is undertaken through our work as a Founding Regulatory Member of the ICH providing leadership of key committees and expert working groups, developing harmonised guidelines and related training, and providing expert feedback on guideline development.

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First-and-only dual-action tablet for the treatment of BRCA-positive mCRPC approved in US

On 11th August, the Janssen Pharmaceutical Companies of Johnson & Johnson announced that the FDA approved AKEEGA™ (niraparib and abiraterone acetate), the first-and-only dual-action tablet combining a PARP inhibitor with abiraterone acetate, given with prednisone, for the treatment of adult patients with deleterious or suspected deleterious BRCA-positive mCRPC. Approval is based on the Phase 3 MAGNITUDE study, a prospectively designed precision medicine study including the largest population of BRCA-positive patients in combination trials to date with metastatic castration-resistant prostate cancer (mCRPC)

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WHO convenes first high-level global summit on traditional medicine

The World Health Organisation (WHO) is convening the Traditional Medicine Global Summit on 17th and 18th August 2023​ in Gandhinagar, Gujarat, India. Co-hosted by the Government of India, the Summit will explore ways to scale up scientific advances and realise the potential of evidence-based knowledge in the use of traditional medicine for people’s health and well-being around the world. Scientists and other experts will lead technical discussions on research, evidence, and learning; policy, data, and regulation; innovation and digital health; and biodiversity, equity, and Indigenous knowledge.

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Pfizer’s ELREXFIO™ receives accelerated approval for relapsed or refractory multiple myeloma

On 14th August, Pfizer Inc. announced the FDA has granted accelerated approval to ELREXFIO™ for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. ELREXFIO is a subcutaneously delivered B-cell maturation antigen (BCMA)-CD3-directed bispecific antibody (BsAb) immunotherapy that binds to BCMA on myeloma cells and CD3 on T-cells, bringing them together and activating the T-cells to kill myeloma cells.

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Use of UK plasma for the manufacture of albumins and vCJD risk

On 16th August, the MHRA published research and analysis from a further review of the safety of albumins manufactured from UK plasma concerning vCJD.

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Issue Number: WN00125

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