Regulatory news - June 2016

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

EMA news

Improving safety of first-in-human clinical trials

EMA has started a review of the guidelines that describe first-in-human clinical trials and the data needed to enable their appropriate design and allow initiation. The aim is to agree a concept paper by July 2016 identifying areas for change and proposals to further minimise the risk of similar accidents to the one in France in January 2016. The concept paper will form the basis for an EU-wide review of the guidelines. This process will include targeted discussions with stakeholders and a public consultation on proposed changes later in 2016. Click here for details.

New cell-based therapy to support stem cell transplantation in patients with high-risk blood cancer

The European Medicines Agency (EMA) has recommended granting a conditional marketing authorisation for Zalmoninx, a new advanced therapy medicinal product (ATMP). Zalmoxis is allogeneic T cells genetically modified with a retroviral vector encoding for a truncated form of the human low affinity nerve growth factor receptor (ΔLNGFR) and the herpes simplex I virus thymidine kinase (HSV-TK Mut2). Zamonix has been recommended as an adjunctive, or add on, treatment for adult patients receiving a haploidentical haematopoietic stem cell transplant (HSCT) for various types of blood cancer to aid immune reconstitution and reduce the risk of graft-versus-host disease. The product is manufactured by MolMed. Click here for details.

Single, central platform now mandatory for all periodic safety update reports

As of 13 June 2016, all periodic safety update reports (PSURs) for human medicines authorised in the European Union must be submitted to the PSUR repository. The PSUR repository is a single, central platform for PSURs and related documents to be used by all regulatory authorities and pharmaceutical companies in the EU. It was introduced by the EU pharmacovigilance legislation to facilitate the exchange of information on the safety of authorised medicines between regulators and pharmaceutical companies. Click here for details.

First statistics on PRIME released

EMA is making available detailed information on the applications that have been granted or denied access to PRIME, including statistics on the type of applicants, the therapeutic areas represented and the data supporting the applications. Going forward, the Agency will release information on PRIME on a monthly basis.

18 applications for PRIME were received as of 6 April 2016 and subsequently assessed by EMA’s Scientific Advice Working Party, Committee for Advanced therapies (CAT) and Committee for Medicinal Products for Human Use (CHMP). Four medicines have been accepted for PRIME.

Since 6 April 2016, another 14 applications have been received and are being processed by EMA.

Click here for details.

Committee for Advanced Therapies (CAT) Monthly Report for May 2016 meeting

The CAT held its 82nd meeting on 18 – 20 May 2016 and the report published on the 27 May 2016 is in here.

The 83rd meeting of the CAT will be held on 16-17 June 2016.


Nothing significant for June 2016. Previous news letters are published here


The HRA has been receiving a far higher number of amendments than anticipated and are exploring the reasons for this. This may delay approvals for amendments planned in the next few months.


The Questions and Answers Document of the ICH S3A Guideline on Note for Guidance on Toxicokinetics: The Assessment of Systemic Exposure reached Step 2b of the ICH Process in May 2016 and now enters the consultation period.

European Commission News and Updates on Pharmaceuticals

The commission have launched a targeted stakeholder consultation on the draft Guidelines on Good Manufacturing Practice for Advanced Therapy Medicinal Products

The EC have now issued their GMP for ATMP document. It is intended this will be a stand-alone document distinct form Eudralex Volume 4. The consultation document has been targeted to stakeholders involved in the development, manufacture and/or commercialisation of ATMPs.

The consultation period runs from 28 June 2016 to 26 September 2016.

The consultation document can be downloaded here.

In addition, four public consultations on recommendations related to "Risk proportionate approaches in clinical trials" are open from 1 June 2016 to 31 August 2016. This is in preparation for the implementation of the new Clinical Trials Regulation (EU) No 536/2014. The main objective is to provide further information on how a risk proportionate approach can be implemented in clinical trials and also highlights the areas identified in the clinical trials Regulation which support and facilitate such adaptations. Further details are here.

Mapping of More Stringent Tissues and Cells Donor Testing Requirements

The Member State competent authorities and the European Commission, DG SANTE agreed to map the more stringent donor testing measures and to make the results of this mapping publicly available. The objective is to provide manufacturers and developers of medicinal products derived from tissues and cells greater clarity and transparency regarding the barriers for the sharing of tissues and cells between Member States. Further details are here.


The EDQM is organising a free webinar on the 7 July 2016 to explain in more depth how the Ph. Eur. and its groups of experts carry out their work and how to join the Ph. Eur. Network.

The 9th Edition of the European Pharmacopoeia, which is applicable from 1 January 2017 will be available in July 2016.


MHRA has produced draft guidance for medical devices including drug-device combination products. Deadline for comments: 5 August 2016 Details here. The guidance, Human Factors and Usability Engineering – Guidance for Medical Devices Including Drug-device Combination Products, is intended to be a useful resource on human factors and usability engineering principles, to clarify the expectations of the regulatory bodies i.e. notified bodies and competent authorities, around compliance with the current and future EU medical device legislation. It is aimed at manufacturers of all device classes who intend to market their device in the UK. The principles are also relevant to device components of drug-device combination products that are regulated as medicines.

FDA Recently Issued Guidance Documents

Charging for Investigational Drugs Under an IND —Questions and Answers. Concerns implementation of FDA’s regulation on charging for investigational drugs under an investigational new drug application (IND) for the purpose of either clinical trials or expanded access for treatment use (21 CFR 312.8).

FDA Categorization of Investigational Device Exemption (IDE) Devices to Assist the Centers for Medicare and Medicaid Services (CMS) with Coverage Decisions. Draft guidance that modifies the FDA’s current policy on categorizing IDE devices which assists the CMS in determining whether or not an IDE device should be covered (reimbursed) by CMS.

Currently Open Consultations

European Medicines Agency

Start Date



End Date

13 Apr 2016

Draft guideline on the sterilisation of the medicinal product, active substance, excipient and primary container

13 Oct 2016

European Commission

Start Date



End Date

1 Jun 2016

Public consultation on the revision of the "Definition of Investigational Medicinal Products (IMPs) and use of Auxiliary Medicinal Products (AMPs)" (previously called "Guidance on Investigational Medicinal Products (IMPS) and Non-Investigational Medicinal Products (NIMPs))

31 Aug 2016

1 Jun 2016

Public consultation on "Summary of Clinical Trial Results for Laypersons"

31 Aug 2016

1 Jun 2016

Public consultation on "Risk proportionate approaches in clinical trials"

31 Aug 2016

1 Jun 2016

Public consultation on the revision of "Ethical Considerations for Clinical Trials on Medicinal products conducted with Minors

31 Aug 2016

28 Jun 2016

Targeted stakeholder consultation on the draft Guidelines on Good Manufacturing Practice for Advanced Therapy Medicinal Products

26 Sep 2016