Our weekly newsletter provides you with the latest industry news, developments and regulatory agency updates.
Regulatory Updates
MHRA guidance updates
- Medicines that you cannot export from the UK or hoard. On 1st October, the Medicines and Healthcare products Regulatory Agency (MHRA) added folinic acid to the list of medicines that cannot be exported from the UK or hoarded because they are needed for UK patients. Click to view source
FDA guidance updates
- Clinical Pharmacology Considerations for Human Radiolabelled Mass Balance Studies. On 26th September, the US Food and Drug Administration (FDA) published final guidance with recommendations regarding clinical pharmacology considerations for conducting human radiolabelled mass balance studies of investigational drugs, including: (1) deciding whether and when to conduct the study, (2) designing the study, and (3) reporting results. Click to view source
- Clarification of Radiation Control Regulations for Manufacturers of Diagnostic X-Ray Equipment. On 27th September, guidance was published to provide clarification to industry and FDA staff of the Federal regulations that relate to diagnostic x-ray equipment. This guidance document only addresses the requirements that apply to diagnostic x-ray equipment under the electronic product provisions of the FD&C Act and the regulations implementing those provisions. Click to view source
- CVM GFI #116 (VICH GL23 (R2)) Studies to Evaluate the Safety of Residues of Veterinary Drugs in Human Food: Genotoxicity Testing (Revision 2). On 30th September, draft guidance was published aimed at ensuring international harmonisation of genotoxicity testing of veterinary drug residues. Click to view source
- Electronic Systems, Electronic Records, and Electronic Signatures in Clinical Investigations: Questions and Answers. On 1st October, the FDA published final guidance with information for sponsors, clinical investigators, institutional review boards, contract research organisations, and other interested parties on the use of electronic systems, electronic records, and electronic signatures in clinical investigations of foods, medical products, tobacco products, and new animal drugs. Click to view source
Initiative announced to improve efficiency of approval process for new medicines in the EU
On 2nd October, the European Medicines Agency (EMA) announced that alongside the European medicines regulatory network, it is working to further improve efficiency in the assessment and approval processes for new medicines in the EU. The initiative aims to better manage the use of the network’s expert resources, streamline assessment processes and encourage better and more comprehensive application dossiers from applicants at the time of initial submission.
Industry Updates
WHO and TikTok to collaborate to promote reliable science-based information
On 26th September, the World Health Organisation (WHO) and TikTok announced a year-long collaboration aimed at providing reliable, science-based health information. This reflects WHO’s commitment to leveraging multiple digital communication platforms to combat misinformation, to promote health literacy, healthy behaviours, and actions in an increasingly digitised world.
EMA’s CHMP recommends suspension of sickle cell disease medicine Oxbryta amid ongoing review
On 26th September, the EMA announced its Committee for Medicinal Products for Human Use (CHMP) has recommended suspending the marketing authorisation for Oxbryta (voxelotor), a treatment for sickle cell disease, as a precautionary measure due to ongoing review of emerging safety data.
FDA approves first-of-its-kind device to reduce coronary obstruction risk
On 30th September, Pi-Cardia Ltd., a global leader in the development of leaflet modification solutions for treating heart valves, announced that the FDA has granted market clearance for ShortCut™, a first-of-a-kind device intended to prepare previously placed bio prosthetic aortic valves that have failed in certain patients who are at risk of coronary obstruction.
FDA awards $17.2m in new grants for rare disease clinical trials
On 1st October, the FDA announced seven new clinical trial grants for fiscal year (FY) 2024 under the Orphan Products Grants Program to support research addressing the critical and often unmet needs of individuals affected by rare diseases. Over the next four years, these grants will provide approximately $17.2 million to clinical researchers. The newly funded projects include a treatment for Cushing’s syndrome, two therapies for blood disorders, two cancer treatments, a treatment for lymphatic malformations, and a gene therapy for an inherited eye disease that causes blindness.
FDA Awards $5.4M to support rare neurodegenerative disease research
On 1st October, the FDA announced over $5.4 million in FY 2024 funding through the Rare Neurodegenerative Disease (RNDD) Grants Program. This includes a new $1.3 million annual grant – totalling $5 million over four years – for developing retinal imaging biomarkers for CADASIL and $4.1 million for nine ongoing studies targeting Amyotrophic Lateral Sclerosis (ALS) and other rare neurodegenerative diseases like Niemann-Pick disease type C, myotonic dystrophy type 1, and familial dysautonomia.
Eli Lilly announces new $4.5 billion facility focused on advanced manufacturing and drug development
On 2nd October, Eli Lilly announced a $4.5 billion investment to create the Lilly Medicine Foundry, a new centre for advanced manufacturing and drug development. This facility will give Lilly the ability to research new ways of producing medicines, while also scaling up manufacturing of medicines for clinical trials. The first facility of its kind, combining research and manufacturing in a single location, it will be located at Indiana’s LEAP Research and Innovation District and will expand the company’s investment there to more than $13 billion.
Gilead signs royalty-free licensing agreements to enhance access to lenacapivir for HIV prevention
On 2nd October, Gilead Sciences announced that it has signed non-exclusive, royalty-free voluntary licensing agreements with six pharmaceutical manufacturers to make and sell generic lenacapavir, subject to required regulatory approvals, in high-incidence, resource-limited countries, which are primarily low- and lower-middle income countries. The agreements were signed in advance of any global regulatory submissions to enable these countries to quickly introduce generic versions of lenacapavir for HIV prevention, if approved.
IRLAB Therapeutics’ IRL757 shows promise in treating apathy in Parkinson’s patients
On 3rd October, IRLAB Therapeutics AB, a company discovering and developing novel treatments for Parkinson’s disease, announced successful results from the first part of its Phase I clinical trial for IRL757, a drug candidate targeting apathy in Parkinson’s patients. The results show that IRL757 is well absorbed, provides good exposure in the body, and has a good tolerability and safety profile. IRLAB has recently secured full funding for the project through clinical proof-of-concept studies.
Issue Number: WN00182
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