Regulatory news - March 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)

Importing Medicines into Northern Ireland Before 31st December 2021

The MHRA released guidance in line with the EU Commission notice on the Application of the Union’s pharmaceutical acquis in markets historically dependent on medicines supply from or through Great Britain after the end of the transition period, which places a reporting obligation on industry to notify the MHRA for medicines imported into Northern Ireland:

“In order to make use of the derogation foreseen in Article 20(b) of Directive 2001/83/EC for human medicinal products and Article 24(b) of Directive 2001/82/EC for veterinary medicinal products, marketing authorisation holders should notify the competent authority that granted the marketing authorisation of the product concerned (Cyprus, Ireland, Malta or Northern Ireland), specifying that – and why in their view - the above criteria of a ‘justifiable case’ in the sense of Article 20 (b) of Directive 2001/83, and of Article 24 (b) of Directive 2001/82, are fulfilled. For medicinal products to be placed on the market in Northern Ireland (NI), the competent authority is the MHRA. For centrally authorised products, the competent authority is European Medicines Authority (EMA).”

The EU Commission Notice means that, for all UK licenced medicines within a company’s portfolio, companies will make use of the flexibilities referenced in the statement above, unless the MHRA is informed otherwise.

For further information and the full MHRA guidance, see here.

On-Site Inspections Resume

The MHRA have announced that in-person inspections will return from 29th March 2021. The MHRA Inspectorate will use a combination of remote and on-site inspection approaches, following a risk-based approach to prioritise in-person inspections.

Guidance including points to consider for returning to on-site inspections, to allow the MHRA to fulfil their public health duties, whilst following the current government guidelines and minimising risk to personal safety.

See here for further details.

Innovative Licensing and Access Pathway

The MHRA have released guidance for industry on the new Innovative Licensing and Access Pathway (ILAP) and the Innovation Passport, which is the new medicine designation awarded to innovative products successfully submitted to the ILAP.

The ILAP aims to accelerate the time to market, facilitating patient access to medicines and providing enhanced regulatory and stakeholder input. The permanent partners of this scheme are the MHRA, National Institute for Health and Care Excellence (NICE), and Scottish Medicines Consortium (SMC). The supporting partners include NHS England (NHSE) and NHS Improvement, Health Research Authority (HRA), and National Institute for Health Research (NIHR).

The Innovation Passport application is the first step of the ILAP and is open to developers at the pre-clinical trial stage through to the mid-development programme point, although to maximise the benefits, applicants are encouraged to apply early in the development of their products. Upon successful Innovation Passport designation, the MHRA and partners create a ‘Target Development Profile’ (TDP) document, which sets out a product-specific roadmap towards patient access in the UK healthcare system.

Further information on the ILAP is available here.


Food and Drug Administration (FDA)

FDA Approves First Cell-Based Gene Therapy for Adult Patients with Multiple Myeloma

The FDA have approved Abecma (idecabtagene vicleucel), a cell-based gene therapy for the treatment of adult patients with multiple myeloma, who have not responded to, or whose disease has returned after, at least four prior types of therapy.

Abecma is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy. This is the first cell-based gene therapy approved by the FDA for this indication.

For more information, see here.