Regulatory news - May 2019

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


European Commission (EC)

Quality of Medicines: Korean Active Substances in line with EU standards

The EC has confirmed the Republic of Korea will join the list of third countries recognised as having a regulatory framework applicable to active substances is capable of ensuring a level of protection of public health equivalent to that of the EU. The Ministry of Food and Drug Safety (MFDS) of the Republic of Korea requested the assessment from the Commission to assess their regulatory framework in 2015 and after two audits conducted in 2016 and 2018, the assessment outcome was successful. The Republic of Korea will join Australia, Brazil, Israel, Japan, Switzerland and the United States of America.

For more information, see here. The Commission Implementing Decision is available here.

European Medicines Agency (EMA)

Working Together for Safe Medicines in the EU

The EMA has launched a social media campaign to highlight how the European Medicines Regulatory Network keeps medicines safe and effective. As part of the campaign, the EMA will start sharing a series of info-cards on Twitter and LinkedIn describing the added value in EU cooperation in keeping medicines safe.

‘Keeping medicines safe’ is a priority for the EMA and they encourage stakeholders to support the campaign by sharing the info-cards on social media.

For more information and to see the info-cards, see here.

United Kingdom

Medicines and Healthcare products Regulatory Agency (MHRA)

MHRA Offers Guidance on Written Confirmations for export of Active Substances Manufactured in the UK in a No-Deal Scenario

The MHRA has released updated guidance on export of active substances manufactured in the UK to the EEA in the case that the UK leaves the EU without a deal. Written Confirmation confirms that, for a third country exporting Active Substances to the EEA; the standards of GMP are equivalent to those in the EEA, the manufacturing plant is subject to regular inspections and that significant non-compliance events would be communicated to the EEA without delay.

Manufacturers of biological active substances do not require Written Confirmation to export to the EEA if they hold an MIA which includes manufacture of biological active substances and the associated GMP certificate, as this confirms what is required in the Written Confirmation.

To read the full article, see here (MHRA’s guidance here).

UK Launch Combined Ways of Working Pilot

The MHRA and Health Research Authority (HRA) have been jointly running a pilot programme to streamline the Clinical Trials of Investigational Medicinal Products (CTIMPs) submission and review process in preparation for the future of clinical trials under the EU Clinical Trials Regulation 536/214.

Currently in the UK, separate Clinical Trial Authorisation (CTA) and Research Ethics Committee (REC) submissions to the MHRA and HRA are carried out prior to initiating a clinical trial. Since April 2018, the pilot has been trialing a single CTIMP application process, which is submitted for both the CTA and REC opinion and has dealt with more than 40 applications.

In the program, the reviews are carried out independently, but the outcome of the review is a coordinated communication ensuring any requests for information or changes to documentation are compatible. Applicants receive a single coordinated communication from the authorities for requests for further information and final decision.

The pilot is currently running with a limited number of organisations, however the Agencies hope to increase the number involved as the pilot progresses allowing more applicants to benefit from trialing the process.

For more information, see here. For an update on the progress of the pilot to date, see here.


Food and Drug Administration (FDA)

White Paper Released Seeking More FDA Flexibility in Early Cell Therapy Development

The Friends of Cancer Research (FOCR) and the Parker Institute for Cancer Immunotherapy have released a white paper requesting the FDA to increase flexibility in clinical and manufacturing requirements for anti-cancer cell therapies.

With the increase in number of cell therapies, including engineered T-cell receptor (TCR) and chimeric antigen receptor (CAR) T-cell therapies showing promise in treating a wide range of cancers over recent years, the paper calls for FDA to revise its 2006 guidance on exploratory investigational new drug (IND) studies to include recommendations for studies of cell therapies.

The white paper acknowledges that updated guidance is needed addressing the unique aspects of cell therapies and to provide clarity on GMP requirements for early investigational production of cell therapies.

To read the full article, see here. The white paper is available here.

FDA Approves Gene Therapy to Treat Paediatric Patients with Spinal Muscular Atrophy

On the 24th May 2019, the US FDA granted the approval of Zolgensma (onasemnogene abeparvovec-xioi), a gene therapy intended to treat children less than two years of age with spinal muscular atrophy (SMA). SMA is a rare genetic disorder caused by mutation in the survival motor neuron 1 (SMN1) gene. This gene encodes for the survival motor neuron (SMN) protein, which is critical for the maintenance and function of motor neurons in the brain and spinal cord, controlling muscle movement throughout the body. This product is the first gene therapy to be approved for the treatment of SMA.

Zolgensma is an adeno-associated viral vector-based gene therapy to treat the genetic mutation in the SMN1 gene. The vector delivers a fully functional copy of the human SMN gene to the target motor neuron cells. One-time intravenous administration of Zolgensma results in the expression of the SNM protein in motor neurons, which improves function and movement of muscle, and survival of a child with SMA.

To read the full article, see here.


International Coalition of Medicines Regulatory Authorities (ICMRA)

Regulators Look to Harmonise how they Tackle Innovation

The ICMRA released a report on how regulators worldwide are working together to better identify and address future regulatory challenges posed by new categories of therapeutics, like cell and gene therapies, and new tools for drug development such as artificial intelligence (AI).

The report is part of a wider effort to reduce duplicative work and increase harmonisation among drug regulators from the US, Europe, Japan and elsewhere, and includes the topic of horizon scanning for new innovations, which for most regulators is still in its infancy. In addition, the report addresses novel regulatory pathways, such as expedited and other early engagement pathways with stakeholders such as the Health Technology Assessment agencies.

To read the full article, see here. The report is available here.

Public consultations




Consultation period


ICH guideline E8 (R1) on general considerations for clinical studies

10 May to 30 Sept 2019

Public consultation