Issue 24: Pharmaceutical regulatory roundup

BY DR TIM SANDLE  | 29 August 2024

 

Catch up with the latest news from around the pharmaceutical industry with issue 24 of our regulatory review, curated by Dr Tim Sandle.

 

  

 

New monographs on anti-interleukin monoclonal antibody 

 

The European Pharmacopoeia (Ph. Eur.) has published two new draft monographs, Ustekinumab concentrated solution (3165) and Ustekinumab injection (3188), for public comment in this quarter’s issue of Pharmeuropa (36.3).

Following the adoption and publication of the monographs for Etanercept (2895), Infliximab concentrated solution (2928) and Golimumab concentrated solution (3103), as well as the ongoing elaboration of a new monograph for Adalimumab concentrated solution (3147), the European Pharmacopoeia Commission (EPC) has continued to build upon its work in the field of therapeutic monoclonal antibodies (mAbs). To date, this has predominantly focused on TNF-alpha inhibitors. Under the spotlight now is the development of standards for a mAb that targets another type of cytokines (i.e. interleukins (IL)), thereby addressing different complexities. Two new draft monographs for ustekinumab, a first-in-class therapeutic human IgG1 kappa mAb that binds to IL-12 and IL-23, have been established using the single-source approach and reflect the considerations outlined in the EDQM’s scientific publication on Elaborating European Pharmacopoeia monographs for biotherapeutic proteins using substances from a single source. A first wave of biosimilar product approvals for ustekinumab has been recorded, with more biosimilars under development.

 

All interested parties are invited to review the drafts and submit comments on their technical content, via the appropriate channel. Stakeholder input will be key to refining and evolving the proposed monograph specifications, ensuring that they are and remain fit-for-purpose in a rapidly evolving multi-product market.

 

The same issue of Pharmeuropa contains another new draft monograph, Golimumab injection (3187), the first text on a medicinal product comprising the active substance described in Golimumab concentrated solution (3103), recently published in Ph. Eur. Supplement 11.6. Ustekinumab concentrated solution (3165) and Ustekinumab injection (3188) therefore form the second such ‘pair’ of monographs for application in this dynamic field.

 

For details, see Pharmeuropa (requires registration): https://www.edqm.eu/en/comment-on-drafts-pharmeuropa- 

 

 

 

Ph. Eur. seeks feedback on the use of plastic additive 18

 

Plastic additive 18 (P-EPQ) is a phenolic antioxidant that may be used as a stabiliser in the processing of plastic materials. It consists of seven components, all of which contribute to the efficiency of the additive. However, it is a product-by-process (reaction product) rather than a physical mixture. It comprises one main component and the isomers of that component.

 

The members of European Pharmacopoeia (Ph. Eur.) Group 16 (the group responsible for texts on plastic materials and containers) are not certain if this additive – which is only mentioned in Ph. Eur. general chapters Polyolefins (3.1.3) and Plastic additives (3.1.13) – is still in use. In addition, from an analytical point of view, detection and quantification of the components of the additive is complex. This is especially true in the presence of other plastic additives, with some of the components being prone to degradation (oxidation). This makes it difficult to establish reasonable quality criteria that can be measured with a standardised compendial method for products-by-process.

 

Feedback and supporting data can be sent to epd@edqm.eu

 

 

 

Ph. Eur. 11.6 now available 

 

The European Pharmacopoeia (Ph. Eur.) Supplement 11.6 is now available and will be applicable in 39 European countries as of 1 January 2025.

 

This volume is included in the 2025 subscription (11.6, 11.7 and 11.8) to the 11th Edition of the Ph. Eur.

 

 

 

USP approves endotoxin testing using non-animal derived reagents

 

The USP Microbiology Expert Committee has approved the inclusion of Chapter <86> ‘Bacterial Endotoxins Test Using Recombinant Reagents’, which permits the use of non-animal-derived reagents for endotoxin testing, to the United States Pharmacopeia–National Formulary (USP–NF). The final text of the chapter will be published for early adoption in November 2024 and will become official in May 2025.

 

Endotoxin testing is a critical step in ensuring the quality and safety of many sterile pharmaceutical products. Chapter <86> provides techniques for bacterial endotoxin testing, using non-animal derived reagents. The bacterial endotoxins tests described in the new chapter are additional techniques to the current tests described in Chapter <85> ‘Bacterial Endotoxins’. 

 

The new chapter includes methods that use both recombinant cascade (rCR) and recombinant Factor C (rFC) reagents and provides information for manufacturers of new and existing pharmaceutical products on how to incorporate them into their quality testing.


Manufacturers that currently use limulus amebocyte lysate (LAL) for endotoxin testing can continue to do so - Chapter <86> has no impact on them.

 

The addition of Chapter <86> to the US Pharmacopeia is in line with USP’s commitment to expanding the use of animal-free methods and materials.

 

 

 

ISPE Good Practice Guide addresses primary container ID challenges 

 

ISPE has brought together industry stakeholders, to develop the ‘Good Practice Guide: Unique Identification of Glass Primary Containers in Pharmaceutical Fill and Finish Operations’. The aim of this is to help ensure uniformity and process interoperability, enabling the industry to interact with regulatory agencies and to influence emerging standards for primary container serialisation. Team leaders Tod Urquhart and Alessandro Pelizzi explain some of the challenges with primary packaging and how the Good Practice Guide addresses them.

 

See: https://www.bioprocessonline.com/doc/should-you-be-using-unique-container-identification-0001 

 

 

 

FDA issues draft guidance on medical device, LDT modification 

 

The FDA released draft guidance highlighting recommended policies on predetermined change control plans for medical devices, including laboratory-developed tests. The guidance includes three examples of devices and the modifications that are and are not appropriate for PCCP inclusion.

 

See: https://www.mwe.com/insights/fda-issues-final-rule-regulating-many-laboratory-developed-tests-as-medical-devices/  

 

 

 

Chemistry of new active substances

 

Control of active substances (chemical entities) present in a medicinal product is fundamental for ensuring both the efficacy of the medicine and patient safety. Many drug products include multiple chemical transformation steps to separate the starting material from the final active substance. 

 

To improve the new drug submission process in relation to the active substance, the European Medicines Agency (EMA) has issued a new draft guideline, titled ‘Guideline on the chemistry of active substances’.

 

The new document aims to bring together new and existing active substances into one guideline. The draft is open for public consultation, until January 31, 2025.    

 

To access see: https://www.ema.europa.eu/en/documents/scientific-guideline/draft-guideline-chemistry-active-substances-revision-1_en.pdf   

 

 

 

Real world data for real world evidence

 

The role of real-world data (RWD) and real-world evidence (RWE) in supporting the evaluation of 
medicines across the different stages of their development and lifecycle is evolving.

 

ICH has released a reflection paper on ‘pursuing opportunities for harmonisation in using real-world data to generate real-world evidence, with a focus on effectiveness of medicines’. The aim is to further enable the integration of RWE into regulatory submissions and timely regulatory decision-making.

 

The paper can be reviewed here: https://www.ema.europa.eu/en/documents/scientific-guideline/ich-reflection-paper-pursuing-opportunities-harmonisation-using-real-world-data-generate-realworld-evidence-focus-effectiveness-medicines_en.pdf 

 

 

 

Good pharmacovigilance practices

 

The EMA has updated its definitions pertaining to good pharmacovigilance practices. The document was first issued in 2012 and it has been periodically updated. 

 

The updated definitions are detailed in ‘Guideline on good pharmacovigilance practices (GVP)’.

 

The update can be accessed here: https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-good-pharmacovigilance-practices-annex-i-definitions-rev-5_en.pdf 

 

 

 


Chemical and biological warfare

 

The European Medicines Agency has published two updated guidances for the treatment and prevention of biological and chemical agents, as might be used by terrorists or in time of war. The scope is with listing and describing medicines and regimens that might be used in the case of an attack with each biological agent. 

 

The two new guidance documents are:

 

‘EMA guidance document on the use of medicinal products  for treatment in case of exposure to chemical agents used as weapons of terrorism, crime or warfare’: https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/ema-guidance-use-medicinal-products-treatment-case-exposure-chemical-agents-used-weapons-terrorism-crime-or-warfare_en.pdf 

 

‘EMA guidance document on the use of medicinal products for treatment and prophylaxis in case of exposure to biological agents used as weapons of terrorism, crime or warfare’: EMA guidance document on the use of medicinal products for treatment and prophylaxis in case of exposure to biological agents used as weapons of terrorism, crime or warfare (europa.eu)

 

 

 

Mutal inspections

 

The document describing the mutual recognition agreement between the European Union and the US (EMA and FDA regulations and inspections) – ‘Questions & Answers on the impact of Mutual Recognition Agreement (MRA) between the European Union and the  United States as of 31 May 2023’ – has been revised during August 2024.

 

The document can be accessed here: https://www.ema.europa.eu/en/documents/other/questions-answers-impact-mutual-recognition-agreement-between-european-union-united-states-7-august-2024_en.pdf 

 

 

 

WHO/EDQM establishment study for Prekallikrein activator in albumin reference standards

 

The details of the new European Pharmacopoeia (Ph. Eur.) ‘Biological Reference Preparation (BRP) for Prekallikrein activator in albumin batch 7’ has been published in the scientific journal Pharmeuropa Bio & Scientific Notes.

 

The study (BSP153) was organised jointly by the National Institute for Biological Standards and Control (NIBSC, now MHRA) and the EDQM, under the auspices of the World Health Organization (WHO) and the Biological Standardisation Programme (BSP). The objective of the joint study was to establish both the WHO 3rd International Standard and the Ph. Eur. BRP batch 7 thereby ensuring harmonised standards and maximising laboratory resources.

 

Here is the link: https://pbiosn.edqm.eu/app/pbiosn/content/pbiosn/2024-6_Establishment-of-PKA-in-albumin-references.pdf 

 

 

 

Concept paper for the development of a guideline on the demonstration of therapeutic equivalence for nasal products

 

The EMA has issued a new draft guideline for consultation on the pharmaceutical quality of inhalation and nasal medicinal products (EMEA/CHMP/QWP/49313/2005 Corr). This document covers both orally inhaled products (OIPs) and nasal products. 

 

The guideline includes the requirements for demonstration of therapeutic equivalence (TE) between two inhaled products for use in the treatment of asthma and chronic obstructive pulmonary disease (COPD) in adults and for use in the treatment of asthma in children and adolescents (CPMP/EWP/4151/00 Rev. 1).

 

To access, see: https://www.ema.europa.eu/en/documents/scientific-guideline/concept-paper-development-guideline-demonstration-therapeutic-equivalence-nasal-products_en.pdf 

 

 

 

Combination products

 

The Food and Drug Administration has issued a draft guidance for industry and FDA staff entitled ‘Purpose and Content of Use-Related Risk Analyses for Drugs, Biological Products, and Combination Products’. 

 

This document provides guidance on the purpose and content of a use-related risk analysis (URRA) and how it, along with other information, can be used to determine human factors (HF) data needs during product development and to support a marketing application.

 

The document can be found here: https://www.fda.gov/media/179858/download

 

 

 

Container-closure systems

 

The U.S. FDA has issued guidance to collate recommendations for appropriate reporting categories and the content of post-approval change submissions across numerous FDA guidance documents. 

 

This guidance conveys recommendations to holders of approved new drug applications (NDAs), biologics license applications (BLAs) and abbreviated new drug applications (ANDAs) regarding the reporting and implementation of changes to container closure system (CCS) components consisting of glass vials and stoppers for approved sterile drug products, (including biological products) administered parenterally. 

 

The guidance also discusses pathways available to application holders to obtain Agency feedback. 

 

Additionally, this guidance discusses risk-based tools available to facilitate the implementation of changes to CCSs consisting of glass vials and stoppers. This guidance does not apply to CCS types other than glass vials and stoppers.

 

To access, see: https://www.fda.gov/media/167925/download 

 

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