Regulatory News - October 2020

Keep up to date with regulatory news from around the world with the Cell and Gene Therapy Catapult regulatory newsletter.


Medicines and Healthcare products Regulatory Agency (MHRA)

Updated MHRA Post-Transition Period Information and Guidance

The MHRA have updated Brexit post-transition guidance on their website. Key updates include:

  • How the MHRA will manage orphan medicinal products including incentives and the application process - The MHRA will be responsible for reviewing applications from companies for orphan designation at the time of a marketing authorisation application. There is no pre-marketing authorisation orphan designation
  • The conversion of community Marketing Authorisations (centrally authorised products to Great Britain Marketing Authorisations)
  • Guidance on new routes for assessment including an accelerated procedure and rolling review:
    • Procedures to prioritise access to new medicines - The MHRA is working with partner organisations in the UK to develop approaches to reduce the time to patient access for new medicines and technologies that will benefit patients.
    • An accelerated assessment procedure - From 1 January 2021, MHRA will introduce an accelerated procedure and will reach its opinion on approvability of marketing authorisation applications within 150 days of submission of a valid application.
    • A rolling review for marketing authorisation applications, intended to enhance development of novel medicines. It does this by offering on-going regulatory input and feedback enabling the applicants to ‘get it right first time’ and ensuring that applications can be approved as efficiently as possible. Applications for any new active substances including biological products that wish to obtain a marketing authorisation in GB based on submission of a ‘full dossier’ to MHRA are eligible for a rolling review.
  • Guidance on variations to Marketing Authorisations - The MHRA will also be responsible for reviewing pending and new variations to UK Marketing Authorisations.

The MHRA continue to update their guidance; see here for post-transition period guidance.

MHRA Issues Guidance on Registering to Make Submissions to the MHRA Post-Transition Period

The MHRA are making preparations to ensure developers can continue to submit regulatory and notification information to the UK from 1 January 2021.

Applications such as Marketing Authorisations will need to be submitted through a national portal. Developers and clinical trial sponsors wishing to make regulatory submissions will need to ensure they are registered in order to gain access to the new MHRA Gateway.

The MHRA has provided guidance on the user registration process. See herefor guidance.

UK MHRA Joins the Australia-Canada-Singapore-Switzerland (ACSS) Consortium

The UK Medicines regulator announced that they have joined the ACSS which will now be known as the Access Consortium.

The group aims to provide patients with timely access to high quality, safe and effective medicinal products. This is accomplished by Access partners working together to reduce regulatory duplication. Pharmaceutical companies that submit applications to some or all of the five Access countries will benefit from having their products evaluated for marketing in those countries simultaneously with reduced evaluation times.

The MHRA will commence working with the Access Consortium from 1 January 2021. Access will update and release guidance on how to apply for marketing authorisation of products across the five countries from the 1 January 2021.

See here for the press release.

MHRA Issues Draft Guidance on Randomised Controlled Trials Generating Real-World Evidence (RWE)

The MHRA has drafted new guidance on points to consider when planning a randomised clinical trial using real world data, with the intention of submitting this trial to gain regulatory approval - such as extending the use of a medication into a new indication or a broader patient population.

The guidance has been drafted alongside the Commission of Human Medicines Real-World Data ad hoc group and considers aspects related to clinical trial authorisation, clinical trial design and requirements in terms of database quality and inspection.

The consultation ends on 11 December 2020.

See here for the guidance document.


European Medicines Agency (EMA)

Orchard Therapeutics Gene Therapy Libmeldy Receives Positive Opinion from the Committee for Medicinal Products for Human Use (CHMP)

On 15 October 2020, Orchard Therapeutics received a positive opinion for Libmeldy from the CHMP for the treatment of Early-Onset Metachromatic Leukodystrophy (MLD) in children. The product was granted orphan designation in 2007 (EU/3/07/446)

The active substance of Libmeldy is autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene. Patients with MLD have a fault with the ARSA gene which leads to lack of the ARSA enzyme. This causes a build-up of sulfatides in the brain and nervous system. A one-time infusion of the gene therapy is expected to slow down the progression of the disease and improve the child’s quality of life. Libmeldy has shown to preserve cognitive and motor function in most patients.

See here for CHMP opinion.

Kite Pharma’s CAR T-cell Therapy Tecartus Receives Positive Opinion from the CHMP

The CHMP has recommended granting a conditional marketing authorisation for Tecartus (autologous anti-CD19-transduced CD3+ cells) for the treatment of adult patients with a rare cancer of white blood cells called mantle cell lymphoma (MCL). Furthermore, the indication is for the treatment of refractory/relapsed MCL. Tecartus is the third CAR-T cell therapy to be recommended approval in the EU. Tecartus was supported through EMA's PRIority MEdicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support to medicines that have a particular potential to address patients' unmet medical needs.

Tecartus contains the patient’s own T-cells, genetically engineering to have a chimeric antigen receptor that helps the body recognise and eliminate lymphoma cells.

An authorisation by the European Commision would be valid for a year but can be renewed annually. Additional data is required before full approval can be granted.

See here for CHMP opinion.

EMA Publishes Draft Guidance on Registry-Based Studies

The EMA has issued new guidance on registry-based studies. The EMA Patient Registry Initiative and the Cross-Committee Task Force on Registries have explored ways to improve the use of patient registries in the context of benefit-risk evaluation of medicinal products. A registry-based study is an investigation of a research question using the infrastructure of (a) new or (an) existing registry(-ies) for patient recruitment and data collection.

The guidance focuses on studies using disease registries as a data source, and primarily targets marketing authorisation applicants and holders. The aim is to provide recommendations on key methodological aspects of registry-based studies and the relevant legal basis and regulatory requirements for marketing authorisation applicants and holders. The guidance also applies to patients and to those involved in the funding, creation and management of registries, those participating in the collection and analysis of registry data, and those planning to use the registry information and infrastructure to perform registry based studies with a possible regulatory purpose.

The consultation ends on 31 December 2020.

See here for draft guidance and associated documents.


Food and Drug Administration (FDA)

FDA Discuss Impact of COVID-19 on Cell and Gene Therapies

FDA officials have expressed concern about the impact COVID-19 has had on the development of cell and gene therapies. Peter Marks, director of the FDA’s Centre for Biologics Evaluation and Research (CBER) said “For some of the studies that are ongoing there are some real challenges to overcome in terms of endpoints that may have been missed.” There have been reports of the pandemic disrupting global harmonisation of gene therapy programmes.

Prior to COVID, CBER were struggling with the influx of cell and gene therapy applications and with the shift in focus at the FDA to COVID-related activities, this will only cause more delays to clinical trials.

The pandemic has also had an effect on the FDA’s surveillance and preapproval inspections and the FDA are considering carrying virtual inspections for some sites.

See here for article.

Public Consultations








Guideline on registry-based studies

24 Sep 2020


31 Dec 2020

Public Consultation