Regulatory News - May 2021

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)

MHRA Update Guidance for Medical Device Studies in Northern Ireland

The MHRA has updated its guidance on how to notify the MHRA about clinical investigations involving a medical device for studies with participating sites in Northern Ireland.

As of the 1st January 2021, Northern Ireland have aligned with EU legislation as required by the Northern Ireland Protocol. Therefore, in line with the EU’s implementation timeline, the Medical Device Regulation (EU) 2017/745 (MDR) and in vitro diagnostic Medical Device Regulation (EU) 2017/746 (IVDR) will apply in Northern Ireland from the 26th May 2021, and 26th May 2022 respectively.

See here for the updated MHRA guidance.


European Medicines Agency (EMA)

EU and Brazilian Regulatory Authorities Confidentiality Arrangement

The EMA and the EU Commissions Directorate General for Health and Food Safety (DG SANTE) have signed a confidentiality arrangement with the Brazilian regulatory authority, Agência Nacional de Vigilância Sanitária (ANVISA).

Signing of the arrangement is an important step towards mutual recognition and regulatory harmonisation, allowing participants to exchange sensitive information about medicinal products. Confidential information on the quality, safety and efficacy of human medicines can be shared, as well as regulatory guidance, legislation and information on inspections.

The confidentiality arrangement came into effect on 26th March 2021 and the agencies have released a guidance providing a framework under which the EMA, DG SANTE and ANVISA will develop their regulatory cooperation for the exchange of confidential information.

For further details on the confidentiality arrangement, see here.

EMA Recommend Gene Therapy for EU Marketing Authorisation

The EMA have recommended Skysona (elivaldogene autotemcel), an autologous gene therapy for treatment of children with cerebral adrenoleukodystrophy (CALD), for marketing authorisation in the EU.

CALD is a rare disease seen almost exclusively in males, caused by abnormalities in the ABCD1 gene, responsible for production of adrenoleukodystrophy protein (ALDP). This protein is required to break down very long chain fatty acids (VLCFA), meaning patients with CALD cannot break down VLCFA. The build-up of these fatty substances leads to inflammation and destruction of myelin sheath that insulates and improves the way that nerves function.

Skysona is manufactured from immature bone marrow cells taken from the patient, which are then modified by a lentivirus that contains a functional copy of the gene ABCD1 for the ALDP protein so that this gene is carried to the cells. Modified cells are administered back to the patient by intravenous infusion and are expected to spread through the body and develop into different types of healthy cells, including brain cells, that produce the ALDP protein.

Skysona has been developed under the PRIME scheme and assessed by the Committee for Advanced Therapies who provided a positive opinion to the EMA’s Committee for Medicinal Products for Human Use (CHMP) which led to the CHMP recommendation for approval of this medicine. The gene therapy is now pending decision from the European Commission on EU-wide marketing authorisation.

See here for the full article.

European Commission (EC)

EC Discuss Findings for the New Genomics Techniques Study

The EC has published a study on the development of New Genomic Techniques (NGTs) over the past two decades. NGTs are methods that can alter the genetic material of an organism and include a diverse group of techniques. These new techniques have the potential to bring many various benefits to the pharmaceutical sector, including in the development of vaccines and therapies for hereditary diseases and cancer.

The study investigated the status of NGTs under Union law following a judgement raised by the Court of Justice of the EU (CJEU) on the scope of GMO legislation. To provide clarity on NGTs, the study includes comprehensive information on their legal status, research and potential applications, safety, potential benefits, and concerns, in addition to addressing ethics and public awareness.

NGTs are shown to have potential for a variety of uses in the medicinal sector but indicates that GMO legislation is not fit to address some NGTs and their applications. As a result of this study, it is hoped that the work carried out by the EC will help policy makers decide whether any possible further action in this policy area should be taken.

Further information on the study is available here. See here for the full article.


Food and Drug Administration (FDA

FDA Flexibility for Data Gaps in Gene Therapy Trials

The Director of the FDA’s Office of Tissues and Advanced Therapies (OTAT) spoke at the American Society of Gene and Cell Therapy (ASGCT) about the impact of the pandemic on declining enrolment to gene therapy trials. Travel restrictions prompted by the pandemic have led to a decrease in enrolment rates for these trials as many gene therapies are for treatment of rare diseases, some affecting very few patients globally, requiring patients to travel to clinical trial sites.

In response to the lower enrolment rates, the FDA has received many requests from sponsors to change their primary and secondary end points, sample sizes, or study duration. The FDA are hesitant to grant these requests due to fears of compromising the integrity of the trials. However, the agency is willing to be flexible and discuss alternative approaches, including telemedicine and the use of wearable devices to monitor a patient’s reaction to therapy remotely.

For further information, see here.


World Health Organisation (WHO)

WHO and International Regulators Release Joint Statement on Public Access to Clinical Data

The International Coalition of Medicines Regulatory Authorities (ICMRA) and the WHO have released a joint statement requesting for pharmaceutical companies to publish clinical trial reports for new medicines and vaccines without redactions, ensuring all research results are publicly accessible.

The joint statement expresses that systematic transparency can increase trust in regulatory decision making, reassure public about data integrity and stimulate research and development. The statement comes because of the COVID-19 pandemic highlighting the need for improved transparency of medical research to build public confidence.

The ICMRA-WHO statement is available here.

Alliance for Regenerative Medicine (ARM)

ARM Release Joint Paper Calling for ATMP Exemption from EU GMO Legislation

In a joint paper ARM, the European Federation of Pharmaceutical Industries Associations (EFPIA), and the European Association of Bioindustries (EuropaBio) are calling for relaxation of EU regulations that treat advanced therapies as genetically modified organisms (GMO).

The paper and a press release statement made by the trade associations call for ATMPs to be exempt from EU GMO legislation. It is highlighted that treating these therapies as GMO products impacts Europe’s ability to attract clinical trials and ultimately delays patient access to transformative medicines. Application of GMO legislation is varied across the EU, with Member States taking diverse approaches for assessment of GMO therapies, causing significant trial delays despite findings that gene therapies pose a negligible risk to the environment.

As a result of the study on NGTs, the EC recognised that GMO requirements hinder the conduct of clinical trials and has called for GMO legislation to be made “fit for purpose” for medicinal products. ARM, EFPIA and EuropaBio are looking to engage with the EC and other stakeholders to resolve this and ensure the EU is a competitive destination for ATMP development.

See here for the ARM press release. The joint paper, published in Human Gene Therapy, is available here.

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