Paul Crozier, Author at HiTech Health

2024 ATMP Review

by Paul Crozier | Dec 11, | HiTech Health Latest News

Over the past decade, the regenerative medicine landscape has undergone an extraordinary evolution with the continuous growth of Advanced Therapeutic Medicinal Products (ATMPs). ATMPs are medicines for human use that are based on genes, tissues or cells. They treat the root cause of diseases and disorders by altering, augmenting, repairing, replacing, or regenerating organs, tissues, cells, genes and metabolic processes in the body. This means that they can offer potentially groundbreaking new opportunities to address unmet medical needs.

10 years ago, in 2014, the ATMP sector was starting to grow but several hurdles including development, manufacturing and regulatory challenges and uncertainties needed to be addressed. Back then the field was still in its infancy, with only a few approved therapies and a rapidly growing number of early stage development programmes. The FDA had approved a small number of cell therapies, including several cord blood-based products like Hemacord and Allocord. However, there were no FDA-approved gene therapies in 2014.¹

So much has changed in a positive way for patients. In 2024, the ATMP sector has expanded significantly, with therapies now available for a diverse range of medical conditions and they have become more accessible. There have been major improvements in the development, manufacturing and supply chains of ATMPs that have helped more patients receive these lifesaving and life changing medicines. There are now multiple gene therapies on the market for rare genetic disorders, such as Spinal Muscular Atrophy and Duchenne Muscular Dystrophy.

The sector has made significant advancements in 2024, driven by positive regulatory approvals, breakthroughs in scientific research, manufacturing optimisations and progress in market access and pricing. ATMPs have already saved and improved many lives, for example, a treatment for spinal muscular atrophy, Zolgensma, has been delivered to over 3,700 children globally. The sector has shown robustness and promising foundations for continued sector growth in 2025 and beyond. Developers are not only focused on proving that therapies can work but, are placing increased emphasis on refining their effectiveness, reducing side effects, expanding their indications and addressing market access and cost challenges.²

At the end of 2024, there were 39 cell and gene therapies approved in the United States by the FDA. Of the 6 new therapies approved by the FDA in 2024, 3 were gene therapies and 3 were cell therapies. In 2024 there have also been 2 new gene therapies approved by the European Medicines Agency (EMA). In addition to this, the FDA, EMA and other regulatory agencies are actively reviewing submissions made in 2024 and it is expected that a number of approval decisions will be made in the first quarter of 2025. The table below shows the therapies that were approved by the FDA and EMA in 2024.³

Therapy	Type	Indication	Developer	Approval Status
Lifileucel	Cell Therapy	Metastatic Melanoma	Iovance Biotherapeutics	FDA approved 16 February 2024
Lenmeldy	Gene Therapy	Metachromatic Leukodystrophy	Orchard Therapeutics	FDA approved 18 March 2024
Fidanacogene Elaparvovec	Gene Therapy	Hemophilia B	Pfizer	FDA approved 27 April 2024 EMA approved 25 July 2024
Tecelra	Cell Therapy	Advanced Synovial Sarcoma	Adaptimmune Therapeutics	FDA approved 1 August 2024
Obecabtagene Autoleucel	Cell Therapy	B-Cell Acute Lymphoblastic Leukaemia	Autolus Therapeutics	FDA approved 8 November 2024
Kebilidi	Gene Therapy	Aromatic L-Amino Acid Decarboxylase Deficiency	PTC Therapeutics	FDA approved 14 November 2024
Casgevy	Gene Therapy	Sickle cell Disease and Beta-Thalassemia	Vertex Pharmaceuticals and Crispr Therapeutics	EMA approved 13 February 2024 (Previously approved by the FDA in late 2023 with an expanded label approval on 16 January 2024)

Six years ago, in 2018, there were approximately 1000 clinical trials and 900 developers across the ATMP sector. By the end of the third quarter of 2024, the number of clinical trials has increased by approximately 97% in 6 years with an estimated 1,968 clinical trials. The number of developers has increased by approximately 230% during the same period to 2,981 developers. Of this estimated number of developers, 1,262 are based in North America, 1,036 are based in Asia Pacific, 587 are based in Europe and 96 are in other regions. Compared to the data in 2023, ‘other regions’ outside North America, Asia Pacific and Europe have seen the largest growth with an estimated yearly increase of 12.9% followed by Asia Pacific (12%), North America (6.6%) and Europe (3.3%).³

Similar to 2023, the majority of the clinical trials still involve cell-based therapies, accounting for approximately 70% of all trials. Oncology remains to be the most prevalent indication for ATMPs. Cancer patients, especially those who relapse, often must endure a series of arduous treatments using current standard of care chemotherapy and radiotherapy. CAR-T therapies have shown a reduction in collateral damage to healthy cells and can minimise side effects compared to traditional treatments. According to the Blood Advances Journal, 76% of all blood cancer patients who receive CAR-T treatments achieve remission. For B cell Lymphoma, overall survival is almost 9 times higher than the standard of care. Patients who receive Yescarta, a therapy used to treat B cell lymphoma, are 21% less likely to require subsequent treatment (insert reference: Vanderbilt University).^{3 4}

Looking into the investments made in the ATMP sector, there was an estimated $13.3 billion USD invested 6 years ago in 2018 (excluding mergers and acquisitions (M&A) and grants). In the first 9 months of 2024, there has been a total estimated investment of $14.2 billion. At the time of writing, the data for the final 3 months of 2024 is not yet available but it is projected that the total annual investment will surpass $17 billion USD. There have been fluctuations in total investment over the last several years but the sector has shown robustness and positive investor sentiment. Across all regions, the largest type of investment is in the form of venture financing. North America has consistently seen the highest levels of investment since the industry was first developed.³

In Europe, in January 2022, Regulation (EU) 2021/2282 on health technology assessment (HTA) came into effect. The regulation aims to be applied in EU Member States by next month on the 12^th January 2025. The HTA introduces an evidence‑based process that will allow the competent EU and national authorities to determine the effectiveness of new or existing health technologies, including cell and gene therapies. From 2025, all cell and gene therapies will undergo a single EU assessment of the value they add to patients and healthcare systems, aiming to end the need for 27 individual reviews. Companies will also meet jointly with the European Medicines Agency and Europe’s HTA coordinating group to discuss and align on the optimal clinical trial designs that deliver data, not only on safety and efficacy but also on added value to patients and healthcare systems.⁵

The regulation of ATMPs has progressed significantly in both the US, Europe and Asia Pacific regions over the last several years. In the US, the FDA has defined more transparent pathways for ATMPs, supported by the Regenerative Medicine Advanced Therapy designation, which facilitates expedited review and development support. The EMA has refined its guidelines for ATMPs, publishing a joint action plan with the European Commission to offer more streamlined processes for developers. There are opportunities for improvements in the regulatory pathways and we expect that the coming years will yield further changes. Developers are seeking more harmonised international regulations to help them navigate approvals across multiple regions more effectively. Notably, the regulatory agencies have also responded to the growing need for post-market surveillance, recognising the long-term nature of many ATMPs and the requirement for ongoing monitoring of their safety and efficacy.

2024 continues the trend of significant advances in the development and approval of new ATMPs which, ultimately, is great news for patients. The number of therapies receiving regulatory approval remains high with several applications still under review by the agencies. While there are challenges for ATMPs in manufacturing, pricing, and safety, the significant increase in the number of clinical trials demonstrates the potential of these medicines to enhance and transform patients’ lives. The outlook for the sector remains strong, with continued investment, technological innovation, and regulatory support propelling the industry forward in 2025 and beyond.

Hitech Health is a leading European CDMO and service provider for ATMPs. If you require support with the development and manufacturing of an ATMP, schedule a meeting with our team by emailing info@hitech-health.com.

References:

1 – https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products

2 – https://www.novartis.com/news/media-releases/novartis-presents-new-data-safety-and-efficacy-zolgensma-including-maintained-and-improved-motor-milestones-older-and-heavier-children-sma

3 – https://alliancerm.org/data/

4 – https://www.hematology.org/newsroom/press-releases/2023/a-promising-outlook-car-t-cells-improve-patient-quality-of-life

5 – https://health.ec.europa.eu/health-technology-assessment/implementation-regulation-health-technology-assessment/joint-clinical-assessments_en

Why Ireland is a proving ground for medicinal breakthroughs like cell and gene therapy

by Paul Crozier | Nov 19, | HiTech Health Latest News

IDA Ireland, the agency responsible for the attraction and retention of inward foreign direct investment (FDI) into Ireland, has recently published an article entitled ‘Why Ireland is a proving ground for medicinal breakthroughs like cell and gene therapy’. Hitech Health is delighted to be featured as part of Ireland’s exciting growth in the cell and gene therapy space. Hitech was the first commercial company to have a licence to manufacture these advanced medicines in Ireland. It works with some large global companies, but Managing Director Brian Harrison says most of the companies in the cell and gene therapy space are smaller companies including university spin outs.

In many cases, these advanced medicine companies have products in development, and need expertise to manufacture pre-clinical materials as is required for toxicology studies. Hitech Health can also manufacture clinical trial materials for these advanced medicine companies once the potential treatment has been approved by regulators to be used in patients . In some cases, large pharma companies outsource these advanced medicine products to contractors and they continue to use Hitech for support.

Read the full article here:https://www.idaireland.com/latest-news/insights/why-ireland-is-a-proving-ground-for-medicinal-breakthroughs

Five Key Considerations When Selecting a Cell and Gene Therapy CDMO

by Paul Crozier | Jul 28, | HiTech Health Latest News

Many Cell and Gene Therapy (CGT) companies outsource product development and manufacturing activities to a Contract Development and Manufacturing Organisation (CDMO). It is critical to ensure that an informed decision is made when selecting a CDMO to prevent future disruption to the intricate supply chains of cell and gene therapies and ultimately patients. HiTech Health explores 5 key considerations for a cross-functional team to examine when partaking in the CDMO selection process.

Does your CDMO have an experienced CGT team?

The complex and time-sensitive nature of CGT manufacturing processes makes it imperative to select a CDMO with a team who have deep knowledge of cell and gene therapies. The technical and regulatory landscapes in the CGT industry are changing frequently and ensuring your CDMO has in-depth expertise and flexibility is crucial to success. Assessing the CGT experience of the proposed CDMO team could reduce the risk of knowledge gaps later. A CDMO may have a strong track record of developing and manufacturing small molecule products and biologics but may have limited experience of CGTs. Are sufficient resources in place now and into the future?

Is there an integrated project management approach defined by the CDMO?

If you have shortlisted potential CDMO partners, have you enquired about their project management approach? How do the proposed approaches differ among the potential partners? Having an integrated project management approach that ensures seamless alignment between production, supply chain, QC testing personnel and many other functions is paramount given the stringent timeframe for supplying CGTs to patients. Understanding how frequently you will meet with the assigned CDMO project manager and how they will communicate with you should be clearly defined from the beginning.

Does the CDMO practice effective Quality by Design (QbD)?

Considering if a CDMO practices Quality by Design (QbD) is an important part of the due diligence process. The CDMO team may work closely with you to measure, analyse and report on the quality attributes of the CGT product. The team members who develop your upstream regulatory strategy and initial CMC plan need to be able to work closely with a trusted CDMO partner who can ensure quality and compliance goals are met. Have you considered an on-site audit of the CDMO before the final selection?

How flexible is the potential CDMO partner?

During the due diligence assessment, look at the CDMO’s internal systems and workflows for a true indication of their fit as a potential partner. The CDMO should be experienced and agile to help you navigate unforeseen challenges, open and transparent to help you mitigate risks and control your costs as well as being fully committed to the ultimate goal of providing CGTs to the patients who vitally need them. These are the characteristics of a resilient and long-lasting partnership, and the indicators of future success.

From the start of a project, the right CDMO will encourage an ongoing dialogue about the quality, pace and cost considerations of your project. They will focus on developing a robust and scalable approach that will work for the entire lifecycle of your project.

Is there a Business Continuity Plan (BCP) that has been implemented and managed effectively by the CDMO?

The CDMO’s ability to maintain essential functions during and after an unforeseen event has occurred is very important and should be assessed prior to signing a contract. Business continuity planning, in this case, relates to establishing risk management processes and procedures that aim to prevent interruptions to manufacturing and supplying CGTs. If an unforeseen event occurs, such as critical components not being available, how will the CDMO mitigate or manage the issues to ensure the effects on operations are minimised. Have you requested a copy of your shortlisted CDMO’s business continuity plan?

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Cell and Gene at the Abbey 2021 – Galway, Ireland

by Paul Crozier | Dec 3, | HiTech Health Latest News

From 10^th to 12^th November, HiTech Health hosted ‘Cell and Gene at the Abbey’, a world-leading conference focusing on Production, Scale-up and Globalisation of cell and gene therapies. We would like to sincerely thank all the speakers, sponsors and attendees of this year’s conference and are already looking forward to hosting the event in 2022.

Over the course of 3 days at the beautiful setting of the Glenlo Abbey in Galway, there were amazing insights from global leaders in the cell and gene therapy space. Overcoming production and supply chain challenges was a key focus with speakers from around the world attending the event. The exciting list of speakers included Power List 2020 in Advanced Medicines featured experts Bruce Levine, Massimo Dominici and Qasim Rafiq who spoke about development and regulatory approvals, challenges in scaling up, supply chain and manufacturing and the opportunities to solve them. An update on the European regulatory environment for Advanced Medicines from Niamh Curran of the HPRA was of benefit to all.

Attendees were able to see first-hand some of the latest technologies in cell processing technologies from many of the event’s sponsors Cellexus, ChemoMetec, Miltenyi Biotec, Sexton Biotechnologies, Terumo, VivaBioCel, AccuScience and MeiraGTx. Cell and Gene therapies are at the forefront of regenerative medicine and HiTech Health are continuing to grow rapidly in this sector. Find out how we can support you on your development and commercialisation journey by contacting us today.

Brexit Deal: Ensuring Continuity of Drug Supply

by Paul Crozier | Jan 12, | HiTech Health Latest News

Overview

On the 24^th December 2020, a Trade and Cooperation Agreement (TCA) was struck between the EU and UK. This is welcomed by the pharmaceutical industry as it helps with maintaining continuity of supply and flow of medicinal products as well as other products between EU and the UK.

From a high-level perspective there will be significant changes to customs procedures which came into effect from the 01^st January 2021. The most catastrophic disruption of a no deal scenario has been avoided and companies can now begin to invest in long-term future supply plans. One of the challenges companies now face is that they have had little time to adjust to the conditions of the deal which may cause initial delays and errors in certification, importation/exportation documentation, and transportation of medical products between the UK and EU and vice versa. The trade deal has significant consequences for the movement of medicinal products both to and from the EU. Let us have a look at these in more detail.

UK companies supplying product to the EU

The three primary areas where businesses should keep an eye out for changes include Transportation and Shipping, Regulatory and Quality and Compliance. Here is what you need to know.

Shipments of medicinal products from the UK to the EU no longer fall under the free movement of goods and therefore are categorised as a cross-border activity. There will be a requirement for additional paperwork such as customs declarations. Companies may need to use brokerage service providers which may result in an increased end user price of the product. Fortunately, the deal means there will be no tariffs placed on any products. UK companies will need to engage EU businesses to act as the Importer of Record (IoR) for products moving from the UK to the EU. It will be extremely important to ensure the correct paperwork is in order for shipments going forward, especially for products with a very short shelf that are being transport under strict time and temperature constraints.

From a regulatory perspective, there are two important things to note. Marketing Authorisation (MA) holders, if held in the UK, will have to be transferred to an EU or EEA country. Secondly, for products that are being imported from the UK to the EU, the EU site of batch release must be named on the MA.

Each batch of UK manufactured drug product intended for the EU/EEA market will need to be imported to a site of physical importation and released by a QP operating under an MIA within the EU/EEA. Until a Mutual Recognition Agreement is in place between the EU and UK, each batch of finished medicinal product must undergo, in an EU Member State, a full qualitative analysis, a quantitative analysis of all the active substances. Pharmacovigilance of drug products placed on the EU/EEA market will need to be managed from a location within the EU/EEA. This means the Qualified Person Responsible for Pharmacovigilance (QPPV) must also reside and operate in the EU/EEA from 1st January 2021.

EU companies supplying product to the UK

Transportation and shipping changes for EU companies supplying product to the UK looks similar to what was mentioned above with additional paperwork including customs declarations being the critical requirement going forward.

If EU companies wish to conduct clinical trials in the UK, the UK regulatory authority will require that exported product to the UK have a certification by a UK QP before it can be administered to a patient (1).

If you are the Sponsor of a UK clinical trial using IMPs imported from into the UK from countries on the ‘approved country for import’ list (initially, all EU and EEA countries) you will require a UK Manufacturing and Import Authorisation (MIA (IMP)) holder to put in place the assurance system to check these IMPs have been certified by a Qualified Person (QP) in a listed country, before being they are used in a clinical trial setting (1). IMPs will not require recertification, however. A wholesaler may only import certified medicines from the EU if the appropriate checks are made by the UK Responsible Person (RP). The RP must reside in the UK, as well as a number of other factors which can be found on the MHRA website (2). It is therefore important to engage with the right personnel in order to successfully put in place the infrastructure necessary to continue to supply medicinal products between the EU and the UK.

For more information on QP batch certification and importation, Importer of Record (IoR) and Responsible Person services, please contact the author Robert Hanly at rhanly@hitech-health.com or email info@hitech-health.com.

References:

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