Tower Mains Weekly Newsletter
Regulatory Updates
MHRA guidance updates
- Adrenaline auto-injectors: new guidance and resources for safe use. On 27th June, the Medicines and Healthcare products Regulatory Agency (MHRA) launched new guidance to highlight the latest safety advice on the steps to take during anaphylaxis. Click to view source
- Blood regulation and safety. On 28th June, information on licences, regulations and safety related to human blood and blood components was published on the MHRA website. Click to view source
EMA publishes report supporting the use of real-world data in regulatory decision making
On 23rd June, the European Medicines Agency (EMA) published a report on the experience the agency has gained in conducting studies with real-world data (RWD), highlighting that it can support both pre-authorisation and post-approval assessments of its scientific committees, working parties and national competent authorities. The report is part of the EMA’s efforts, alongside the European Medicines Regulatory Network (EMRN), to enable the use of RWD in regulatory decision-making.
FDA guidance updates
- Alternative procedures for the manufacture of cold-stored platelets. On 23rd June, the US Food and Drug Administration (FDA) published final guidance on alternative procedures for the manufacture of cold-stored platelets intended to treat active bleeding when conventional platelets are unavailable or their use is not practical. It provides notice of exceptions and alternatives to certain requirements in Title 21 of the Code of Federal Regulations (CFR) regarding blood and blood components. Click to view source
- Formal dispute resolution and administrative hearings of final administrative orders under section 505g of the Food, Drug, and Cosmetic Act. On 23rd June, draft guidance was published to provide recommendations on the formal dispute resolution and administrative hearings procedures for resolving scientific and/or medical disputes between the Center for Drug Evaluation and Research (CDER) and requestors and sponsors of drugs that will be subject to a final administrative order (final order) under section 505G of the Food, Drug, and Cosmetic Act (FD&C Act)(21 U.S.C. 355h). Click to view source
- Psychedelic Drugs: Considerations for Clinical Investigations. On 23rd June, draft guidance was published to provide general considerations to sponsors developing psychedelic drugs for the treatment of medical conditions (e.g., psychiatric disorders, substance use disorders). Click to view source
Industry Updates
FDA approves talazoparib with enzalutamide for HRR gene-mutated mCRPC
On 20th June, the FDA approved Pfizer’s talazoparib with enzalutamide for the treatment of adult patients with homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (mCRPC). This represents the first approval of this class of drugs for patients without prior treatment for mCRPC with mutations in genes other than BRCA and is based on a 55% reduction in the risk of radiographic progression-free survival (rPFS) compared to placebo with enzalutamide in the TALAPRO-2 study that enrolled 399 patients with HRR gene-mutated mCRPC.
FDA announces open period for applications to develop evidence-based, clinical practice guidelines for the management of acute low back pain and acute postoperative pain
The FDA has announced an open period for applications to support the development of evidence-based, clinical practice guidelines for the management of acute low back pain, and acute postoperative pain in patients who have undergone diagnostic and procedural laparoscopic abdominal surgeries.
Alchemab Therapeutics reveals Alzheimer’s candidate
On 22nd June, Alchemab Therapeutics, a company focusing on identifying naturally occurring antibodies, unveiled data on its newly-discovered pre-clinical Alzheimer’s candidate at the Antibody Industrial Symposium in Tours, France. After identifying common antibodies unique to individuals resilient to Alzheimer’s disease, Alchemab identified the target as CD33, a cell surface protein which is thought to have a key role in the disease. Studies have found that higher expression of CD33 is associated with more advanced cognitive decline and worsening disease status as high levels of CD33 inhibit the normal function of brain-resident immune cells called microglia, which maintain neural networks and repair damage.
First gene therapy for treatment of certain patients with Duchenne muscular dystrophy approved in US
On 22nd June, the FDA approved Elevidys, the first gene therapy for the treatment of pediatric patients from four to five years of age with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who do not have a pre-existing medical reason preventing treatment with this therapy. A clinical benefit of Elevidys, including improved motor function, has not been established. As a condition of approval, the FDA is requiring the company to complete a clinical study to confirm the drug’s clinical benefit.
Blood Cancer UK extends collaboration with RareCan to accelerate research into rare cancers
On 23rd June, Blood Cancer UK announced that it has renewed its partnership with RareCan, a digital health company, to support the growth of RareCan’s patient membership and the rapid delivery of clinical trials focussed on the prevention, diagnosis, or treatment of rare cancers. The partnership will continue to enable individuals living with rare blood cancers, including types of leukaemia, lymphoma, and myeloma, to volunteer to participate in potentially lifesaving clinical research, by providing tissue and blood samples, and genetic data. Members with multiple myeloma who are already part of the RareCan community have also had the opportunity to be screened for possible clinical trials and more recently have been invited to join the Myeloma Research Panel run by Pfizer.
Ban lifted on the use of UK plasma to manufacture life-saving albumin treatments
On 26th June, the MHRA revealed leading scientists at the independent Commission on Human Medicines (CHM) have confirmed that albumin, a critically important medicine for the NHS, can now be safely derived from UK plasma donors. Following a further review of the evidence, the CHM has also recommended lifting the ban on treating patients with UK albumin.
MHRA issues Drug Safety Update newsletter for June 2023
On 27th June, the MHRA published the June issue of its monthly Drug Safety Update newsletter from the agency and its independent advisor the Commission on Human Medicines (CHM).
Click to view source View PDF newsletter
New collaboration between WHO International Classification of Diseases (ICD) and MedDRA launched
On 27th June, the World Health Organisation (WHO) and the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) announced a new collaboration to enhance registry and sharing of regulatory information on medical products worldwide. By connecting the WHO International Classification of Diseases (ICD-11) with the ICH Medical Dictionary for Regulatory Activities (MedDRA), this common language will enhance the analysis of adverse events and outcomes, such as insurance claims databases, mortality and morbidity statistics, clinical trials, and observational studies.
Issue Number: WN00118
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