Telethon 003 Archives - European Industrial Pharmacists Group (EIPG)

The EU Parliament voted its position on the Unitary SPC


by Giuliana Miglierini The intersecting pathways of revision of the pharmaceutical and intellectual property legislations recently marked the adoption of the EU Parliament’s position on the new unitary Supplementary Protection Certificate (SPC) system, parallel to the recast of the current Read more

Reform of pharma legislation: the debate on regulatory data protection


by Giuliana Miglierini As the definition of the final contents of many new pieces of the overall revision of the pharmaceutical legislation is approaching, many voices commented the possible impact the new scheme for regulatory data protection (RDP) may have Read more

Environmental sustainability: the EIPG perspective


Piero Iamartino Although the impact of medicines on the environment has been highlighted since the 70s of the last century with the emergence of the first reports of pollution in surface waters, it is only since the beginning of the Read more

EMA’s pilot scheme for academic and non-profit development of ATMPs

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by Giuliana Miglierini

Advanced therapy medicinal products (ATMPs) are often developed by academic and non-profit organisations, because of their high level expertise in the biotechnological techniques that underpin many new therapeutic approaches. On the other hand, these organisations often lack sufficient understanding and experience to face the complexity of the regulatory development.

To improve the possibility for non-commercial developers to access regulatory and scientific support related to promising ATMPs addressing unmet medical needs, a pilot was launched by EMA in September 2022. Three projects have been already selected to participate to the pilot. ARI-0001, a chimeric antigen receptor (CAR) product based on patients’ own T-cells, developed by the Hospital Clínic de Barcelona, was the first project to access the pilot. The product was granted eligibility to the PRIME scheme in December 2021, and is targeted to treat patients older than 25 years with relapsed/refractory acute lymphoblastic leukaemia.

The second call closed in December 2023 and saw the participation of 11 candidates, among which two new academic organisations were selected. The Berlin Center for Advanced Therapies (BeCAT) – Charité is developing TregTacRes, a gene therapy based on modified T-cells, for use as add-on therapy after transplantation. Fondazione Telethon’s gene therapy Telethon 003 (etuvetidigene autotemcel) targets the Wiskott-Aldrich syndrome, a rare, life-threatening immunodeficiency.

The new phase of the project will now recruit a total of 5 new participants by the end of 2024. The first results of the pilot are expected in 2025.

How to apply
Interested academic organisations can find all information together with the ATMP Pilot Application Form on the dedicated EMA webpage; applications are open up to the end of April 2024. The email address [email protected] is also available to request more information or to express interest in participating. A guideline document on fee incentives for scientific advice, marketing authorisation (MA) applications and pre-authorisation inspections for academic participants is available, together with Q&As on the pilot.

Pre-selected candidates will be invited to a meeting with EMA’s Innovation Task Force (ITF) to provide further information on their projects before final selection. At this stage, interactions between EMA and applicants would mainly take place via the online platform IRIS. Therefore, interested organisations will need to register to the platform and request a research product identifier (RPI).

Requirements and procedure for the application
Academic developers active in the EU can apply to the pilot provided they have already generated some proof-of-principle data on the interested ATMP. The academic status of the organisation will be checked by EMA during the selection phase. Applicants may include public/not-forprofit hospitals or research organisations and hospitals, Higher Education Institutions (HEI), public-private partnerships/consortia, and international research organisations, provided they are establish in the EU. In case of projects comprehensive of non-EU participants, the principal investigator has to be located in the EU, and clinical trials must include EU patients. The academic sponsor must be free from operating agreements with any pharmaceutical company, and it can freely operate via intellectual property rights on the product.

The support provided by EMA aims to ensure that development activities would meet regulatory standards as for quality, safety and efficacy of the ATMP product. A smooth path towards the submission of the MA application based on existing regulatory procedures and tools should therefore be possible. The pilot also aims to identify potential gaps in existing tools and procedures, from the perspective of academic sector developers.

Key principles used to select the new participants are listed in the Q&As document. As for individual ATMPs under development, they must address an unmet medical need, represent a major therapeutic advantage over existing treatments, or offer a new option in orphan areas. Previous eligibility to the PRIME scheme is not a prerequisite to apply for the pilot. The Q&As also specify that there is no direct link between the product having received an hospital exemption (HE) and access to the academic pilot.

Preliminary clinical evidence in patients is needed to support the application, as well as information on the mechanism of action gained by non-clinical studies. A sufficiently mature quality development, to be assessed against the pharmaceutical process and the planned GMP manufacturing process, should be also available to better support later stage clinical development and/or a MA application in the EU.

The academic sponsor must also have full access to the data related to the development and manufacture of the product, e.g. control of critical starting materials. The knowledge needed to successfully interact with EMA may be ideally provided by a specific person (also a consultant) appointed by the sponsor and with experience in the field of product development and regulatory affairs.

Benefits and fee reductions
Selected academic organisations will benefit by a dedicated EMA’s point of contact in the relevant therapeutic area office. A EMA Support team may be also appointed to provide regulatory and scientific support depending on the stage of development and nature of the program. Activities to be part of the pilot may include preparatory teleconferences to check planning, identify potential needs for additional support and complement interaction mechanisms under existing tools. The optimisation of pre-submission meetings is another goal of the pilot, together with debriefings before and/or after regulatory interactions. A particular attention will be payed to the regular assessment of the level of maturity of the projects, including co-decisions and stopping points.

EMA will also provide financial support to the selected academic applicants for the activities concerning the five selected ATMPs. More in particular, the Agency will grant the same incentives as for micro-, small- and medium-sized enterprises, with respect to fees established by the Council Regulation (EC) No 297/95 and its Implementing Rules.

To qualify for the fee incentives, selected academic organisations must continue to fulfil all the above mentioned criteria for accessing the pilot also at the time of the request for a fee incentive related to a procedure or service to be provided as a part of the pilot itself. To this instance, applicants shall submit a declaration to EMA, inclusive of the fulfilment of requirements and establishment in the European economic area.

Incentives for academic organisations participating to the pilot include a 90% fee reduction for both initial scientific advice and follow-up, and pre-authorisation inspections. MA applications for designated orphan medicinal products for human use will benefit a 100% fee reduction, while MA applications not covered under this occurrence will see deferral of payment until the notification of the final decision on the MA for the concerned ATMP is issued.

The document on fee incentives specifies also that remuneration of national competent authorities for those activities shall not be reduced.

The granting of fee incentives will follow EMA’s verification of the documentation submitted by the applicants. After confirmation by the Agency the applicant qualifies for the fee reduction, participants to the pilot will have a six months period to submit their requests for scientific advice and/or marketing authorisation. Ex–post controls and prove of evidence confirming the fulfilment of criteria for the fee reduction may also be required at any time until the finalisation of the concerned procedure.