The advanced therapies market - Italy (2024)

1.2 The global market

In recent years, innovative therapeutic technologies have been developed based on DNA, RNA, cells, and tissues instead of chemically synthesized molecules. This broad set of therapeutic solutions is classified as Advanced Therapeutic Medicinal Products (***). ATMPs are true medicines derived from the remarkable advances made in biotechnology and genetic engineering over the past ** years. They aim to target diseases at their genetic or cellular root, redefining the therapeutic approach toward personalization targeted to the individual patient.

The value of the global advanced therapies market shows significant growth from **** to ****, with a compound annual growth rate (***) of **.** percent. Starting from $**.* billion in ****, it is expected to reach $**.* billion in ****. This represents an increase of ***% over the period.

Global market value of advanced therapies World, ****-****, in US$ billion GrandViewResearch

The emergence of advanced therapy medicinal products (***) has transformed the pharmaceutical industry and the landscape of disease treatment. It has opened new avenues for the treatment of incurable diseases and different types of cancer. The success of products such as Kymriah, Zolgensma and others is the key factor driving the market. The COVID-** pandemic sparked the application of advanced therapies in virology research. Although the market for advanced therapy drugs is currently in a ...

1.3 The European market

To date, ** Advanced Therapies have been approved in Europe - * of which have since been withdrawn from the market - mainly for monogenic diseases, hematologic malignancies, and tissue injury. It is estimated that at least ** Advanced Therapies will be on the way by ****, with nearly ***,*** patients potentially treatable with a gene or cell therapy. The burden of cancer is increasing in the EU. The estimated number of new cases diagnosed in **** was *.* million, and *.* million people lost their lives to it. It is the second leading cause of mortality after cardiovascular disease. However, it still remains an area of high unmet medical need in Europe. In ****, EMA launched a new initiative to help develop cancer medicines and build valuable expertise in line with the goals of the European Cancer Plan.

The value of the European advanced therapiesmarket shows steady growth from **** to ****. Starting at $*.* billion in ****, it is expected to reach $**.* billion in ****. This represents a ***% increase over the period, with a CAGR of **.*%.

European advanced therapies market value Europe, ****-****, in US$ billion PharmiWeb

The growing demand for innovative therapies underscores a paradigm shift in healthcare, in which patients and healthcare providers are increasingly prioritizing personalized and transformative treatments. Drugs ...

1.4 The Italian market

Technological innovation in biotechnology is leading to significant developments in advanced therapies, including gene therapies, cell therapies, and regenerative medicine. These advances offer new possibilities for the treatment of previously difficult-to-treat diseases, attracting investment and interest from both the public and private sectors. As for our country, * Atmps are reimbursed and on the market, and * more are under evaluation.

In addition, the increasing prevalence of chronic and rare diseases stimulates demand for more effective and personalized therapies, further driving market growth. Improved research and development infrastructure, along with favorable government policies and tax incentives, supports biotechnology companies in their development. Biotechnology is closely linked to advanced therapies because it provides the scientific and technical foundation needed to develop these innovative therapies. Biotechnology research contributes to the understanding of the biological mechanisms underlying diseases and the design of new treatment modalities. As a result, the growth of the biotechnology sector supports the expansion of the advanced therapies market, creating a virtuous circle of development and innovation.

Between **** and ****, the value of the Italian market under the full scenario is expected to increase significantly, from ***.* million euros to ****.* million euros. This represents a growth of ***% in five years. The most pronounced increase is ...

1.5 Inflation suffered by the sector

Inflation has increased production and research costs in advanced therapies, leading companies to review budgets and priorities. Despite rising drug prices and difficulties in accessing funding, the industry has responded by optimizing costs, seeking collaborations, and adopting automation technologies to maintain production efficiency. Companies have also intensified partnerships to share resources and accelerate the development of new therapies, ensuring innovation and accessibility of treatments.

Producer price index of the industry for the manufacture of medicines and pharmaceutical preparations [***] - monthly data - base ****=*** Italy, ****-****, index Istat

The producer price index of the industry for the manufacture of medicines and pharmaceutical preparations in Italy shows an upward trend from January **** to December ****. Starting from a value of ***.* in January ****, the index increases steadily until it reaches ***.* in December ****. This represents an increase of *.* percent over the period. After stabilizing in the middle of ****, the index resumes a steeper rise from the beginning of ****, signaling a continuous increase in producer prices in this sector.

2.1 Overview of demand

In recent years, innovative technologies based not on chemically synthesized molecules but on DNA, RNA, cells, and tissues have been developed for therapeutic purposes. This broad panel of therapeutic solutions is classified as Advanced Therapeutic Medicinal Products (***)-compatible donor.

ATMPs are a rapidly growing therapeutic category, offering new opportunities for the treatment, prevention, and diagnosis of diseases considered incurable to date (***). At the same time, however, these complex and innovative treatments pose specific challenges to the design and conduct of preclinical and clinical studies, as well as to their market access.

Biologic drugs in this new frontier for personalized medicine are classified into four main categories:

Gene therapy: Gene therapy products contain a DNA or RNA molecule to regulate, repair, replace, add or delete a gene sequence. An attempt is made to combat the disease by directly targeting its genetic basis, either by providing the organism with a corrected copy of the defective gene, or another gene that can compensate for its malfunction in the cells affected by the mutation, or by using genomic editing, a procedure that allows a defective gene to be corrected without having to provide a healthy copy from outside. The procedure can take place:

In vivo: ...

2.2 Geographical distribution of demand

The geographical distribution of ATMP spending in Italy shows a strong concentration in some regions, with Lombardy leading the way with ** million, followed by Latium with *.* million and Emilia-Romagna with *.* million. Regions such as Piedmont, Veneto, Tuscany and Sicily show moderate spending between *.* and *.* million. Other regions such as Valle d'Aosta, Molise and Basilicata show no spending, while regions such as Trentino-Alto Adige, Abruzzo and Sardinia show very low spending of less than one million. This distribution shows a varied and uneven commitment to ATMP adoption among different Italian regions.

For per capita spending, Liguria and Lombardy lead the ranking with *.** and *.** euros respectively, followed by Umbria with *.** euros. In contrast, regions such as Valle d'Aosta, Molise and Basilicata show no spending at all. Trentino-Alto Adige and Sardinia show very low expenditures of *.** and *.** euros, respectively. Other regions such as Friuli-Venezia Giulia, Emilia-Romagna and Tuscany have expenditures between *.** and *.** euros, while regions such as Latium, Sicily and Calabria are between *.** and *.** euros. These figures reflect considerable variability in per capita investment in ATMP among different Italian regions.

2.3 Online interest in advanced therapies

Online searches for CAR-T and gene therapies in Italy from July **** to June **** show divergent trends and interesting dynamics. Searches on CAR-T therapies start from an index of **.* in July **** and increase significantly to **.* in June ****, an increase of ***%. The largest peaks are observed in June **** and January ****, showing increasing interest over time.

Gene therapy research begins with an index of **.* in July **** and fluctuates over the period, ending with **.* in June ****, representing a slight decrease of *.*%. The highest values are found in December **** and January ****, followed by a decline.

Overall, CAR-T therapies have seen much more growth than gene therapies, indicating a significant increase in interest in CAR-Ts over the period examined.

*The numbers represent research interest relative to the highest point on the graph in relation to the region and period shown. A value of *** indicates the highest frequency of search for the term; ** indicates half as many searches. A score of *, on the other hand, indicates that insufficient data were found for the term.

Trend of online searches for CAR-T and gene therapies Italy, ****-****, index Google Trends, Businesscoot elaboration

The geographic distribution of online searches for CAR-T in Italy shows a strong concentration of interest in ...

2.4 Trends of major diseases treated by advanced therapies

Between **** and **** in Italy, the leading causes of death for which advanced therapies are indicated show different trends. Malignancies are the predominant cause, with the number declining from ***,*** in **** to ***,*** in ****, a * percent decrease. Ischemic diseases of the heart also show a significant reduction, from **,*** to **,***, a decrease of **%. Chronic lower respiratory diseases see a **% decrease, from **,*** to **,***. On the other hand, deaths from diabetes mellitus increase by **%, from **,*** in **** to **,*** in ****, peaking at **,*** in ****. Diseases of the nervous system and sense organs show a * percent increase, from **,*** to **,***, peaking at **,*** in ****. These data show an overall reduction in deaths for some chronic diseases, while others such as diabetes mellitus and diseases of the nervous system show an opposite trend.

Main causes of death indicated for advanced therapies Italy, ****-****, in numbers Istat

In ****, deaths from malignancies in Italy show a strong correlation with age, increasing significantly in older age groups. In children and adolescents (***), deaths are relatively low, with a maximum of ** cases in the **-** age group. In the later age groups, the numbers gradually increase: *** deaths among **-** year olds and **** among **-** year olds. The number of deaths exceeds *,*** for the **-** age group and continues to ...

2.5 New trends in demand

New trends in the demand for advanced therapies, such as gene therapies, cell therapies, and RNA therapies, are influenced by various technological, regulatory, and market factors. Here is an overview of the main current trends:

*. Exponential Growth in Demand

Rare Disease Rise: There is a growing awareness and diagnosis of rare diseases, many of which are targets for advanced therapies. Aging Population: An increasing elderly population is driving demand for therapies for chronic and degenerative conditions.

*. Technological Progress

CRISPR and Genomic Editing: New gene editing technologies, such as CRISPR, are accelerating the development of personalized therapies for a wide range of diseases. RNA-Based Therapies: Following the success of mRNA vaccines against COVID-**, there is growing interest in RNA-based therapies for other diseases.

*. Personalization of Therapies

Personalized Medicine: Advanced therapies are often customized to fit the patient's genetic profile, improving efficacy and reducing side effects. Biomarkers and Advanced Diagnostics: The use of biomarkers to identify patients who may benefit most from advanced therapies is becoming increasingly common.

*. Access and Reimbursability

ReimbursabilityPolicies: Governments and insurance companies are beginning to develop specific reimbursement policies for advanced therapies, despite their high costs. Access in Developing Countries: There is a growing effort ...

3.1 Italian market structure

From **** to ****, the number of companies active in the field of biotechnology for health in Italy increased steadily from *** to ***. This represents an increase of **.* percent over the period. **** saw a significant increase, with a jump from *** to ***, while in **** there was a slight decline to ***, followed by continued recovery and growth through ****. This positive trend indicates an expanding sector, despite the slight decline in ****.

Number of firms active in biotechnology research and experimental development [***] Italy, ****-****, in number Istat

From **** to ****, the number of enterprises active in research and development in health biotechnology in Italy has shown a fluctuating trend. Starting from *** in ****, the number dropped to *** in ****, then rose again to *** in **** and reached *** in ****. However, there was a significant decrease to *** in ****, stabilizing at that value in **** as well. Overall, the number of businesses decreased by **.* percent. Number Firms active in research and development in health biotechnology Italy, ****-****, in number Assobiotec, Businesscoot elaboration

From **** to ****, the number of people working in the health field of biotechnology in Italy showed an initial growth from **** to **** in ****, followed by a slight decrease, stabilizing around **** in ****. Overall, there was an increase of *.* percent over the period. The peak in **** was ...

3.2 Value Chain

*. Research and Development (***)

Discovery and Innovation:

Identification of diseases and unmet clinical needs. Development of advanced technologies and methodologies such as viral vectors, CRISPR, and scaffolds for tissue-based therapies.

*. Preclinical Experimentation

Preliminary Testing:

Conducting in vitro and in vivo studies to evaluate safety and initial efficacy. Optimization of doses and methods of administration.

*. Clinical Trials

Clinical Studies:

Phase I: Safety evaluation on a small number of volunteers or patients. Phase II: Efficacy studies on a larger number of patients and further safety evaluation. Phase III: Confirmation of efficacy and safety on a large scale to support regulatory approval.

*. Manufacturing and Production

Manufacturing and Quality Control:

Large-scale manufacturing of vectors and therapeutic cells. Implementation of rigorous quality controls to ensure product purity, efficacy and safety.

*. Regulatory Approval

Marketing Authorization:

Submission of clinical trial data to regulatory authorities (***). Review and approval of therapies by regulatory agencies.

*. Distribution and Administration

Logistics and Processing:

Cold chain management for therapies requiring controlled temperatures. Distribution of therapies to clinical centers and administration to patients, with post-administration monitoring.

*. Post-Marketing Monitoring and Access

Pharmacovigilance and Market Access:

Continuous monitoring of real-world safety and efficacy. Negotiation of price and reimbursement arrangements to ensure accessibility ...

3.3 The main players in the market

The Italian companies that have advanced therapies approved in Europe and in Italy, that is, that hold the AIC, marketing authorization, are as follows for gene therapy:

Telethon Foundation: Related to the product Strimvelis, is an organization dedicated to scientific research on rare genetic diseases. Established in May ****, it collaborated on the development of Strimvelis, a retrovirus-based ex vivo gene therapy designed to treat ADA-SCID in the absence of consanguineous donors. It uses a cell fraction enriched with genetically engineered autologous CD**+ cells containing cDNA encoding for human ADA. The Telethon Foundation was crucial in achieving this medical breakthrough, contributing to its approval as an orphan drug in Europe and Italy.

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Novartis: It is a globally operating pharmaceutical company. Specializing in the research, development and commercialization of innovative treatments, it has created Luxturna, an in vivo gene therapy for inherited retinal dystrophy caused by biallelic mutations in the RPE** gene. This treatment uses an adeno-associated virus to deliver a functional copy of the RPE** gene directly to retinal cells. Luxturna ...

3.4 Innovative start-ups

In the field of advanced therapies, technological and scientific innovations are revolutionizing the treatment of many diseases, particularly cancer diseases. Cell and gene therapies, biologics, and antibody-drug conjugates are some of the areas that are seeing significant developments. These advanced therapies aim not only to improve the efficacy of treatments, but also to reduce side effects and improve patients' quality of life. Listed below are some of the most innovative start-ups that are contributing promising solutions to this field.

Ankyra Therapeutics: This US-based start-up develops cytokine suppression-based therapies for cancer immunotherapy. Their platform aims to improve activation of the immune system against tumors, reducing toxicity and increasing the safety of treatments(***).

OXvax: British start-up developing a dendritic cell-based cancer vaccine platform. They use induced pluripotent stem cells to generate a new subtype of dendritic cells with a potent anti-cancer response(***).

Catamaran Bio: Located in the United States, Catamaran Bio offers proprietary technology to engineer and produce NK cells with chimeric antigen receptors (***).

Creative Destruction Lab (***).

StemBond Technologies: Based in the United Kingdom, StemBond develops a hydrogel-based tissue culture platform that enables precise control over cell fate and function. This technology facilitates scalable cell culture and tissue engineering for regenerative medicine(***).

THT ...

4.1 Supply analysis

Below is a detailed description of the main types of advanced therapy offerings:

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Source: ****

4.2 Market prices

Data from the Consumer Price Index for Pharmaceuticals in Italy show a general downward trend from **** to ****. The index fell from **.* in **** to **.* in ****, an overall decrease of about *.* percent. However, the trend has been uneven: the index hit its lowest point in **** at **.*, and then rose slightly in subsequent years, reaching **.* in ****. This indicates some stability and a slight recovery in recent years after the initial decline. For the advanced therapy market, this recent stability suggests a relatively stable price environment, which is useful for planning entry or expansion strategies.

Consumer price index for pharmaceuticals [***] for the whole community (***) - annual averages Italy, ****-****, index Istat

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Pricing of ATMP products:

4.3 New supply trends

The market for advanced therapies is evolving rapidly with several emerging trends. Here are the main ones:

Personalization of Treatments: Personalized medicine continues to grow, especially in the context of gene and cell therapies. For example, CRISPR gene editing has seen significant approval in the United Kingdom for the treatment of sickle cell disease and beta thalassemia (***). This personalized approach is becoming increasingly common, allowing treatments tailored to specific genetic profiles of patients.

Expansion of CAR-T Therapies: They are seeing significant developments to address current challenges. For example, combining CAR-T therapies with specific vaccines to maintain an effective CAR-T cell population and using gene-editing tools to reduce toxicity are new strategies being explored (***).

Reducing Costs and Increasing Affordability: Innovations in payment models, such as outcomes-based contracts and copay assistance programs, are making advanced therapies more affordable despite high costs. For example, AscellaHealth is implementing innovative financial solutions to help patients cover the costs of expensive gene therapies (***).

Multi-Target and Combined Treat ments: New therapies are beginning to target multiple molecular markers simultaneously, increasing efficacy against different forms of disease. In addition, there is growing interest in combination therapies, which use multiple therapeutic approaches together (***) to improve outcomes.

Adoption of CRISPR Technology: ...

5.1 Regulation of advanced therapies

In Italy, the regulation of advanced therapies is a complex system involving numerous laws and regulations to ensure the safety, efficacy and quality of products. Advanced therapies include gene therapy medicinal products, somatic cell therapy medicinal products, and tissue engineering products. This regulatory process follows guidelines established by the European Union, supplemented by specific Italian regulations.

The European regulatory framework for advanced therapies is mainly governed by Regulation (***) No. ***/****. This regulation defines the requirements for marketing authorization, pharmacovigilance, traceability and quality of advanced therapy products. Directive ****/**/EC, on the Community code relating to medicinal products for human use, provides the general framework for safety, efficacy and quality requirements for all medicinal products, including advanced therapy medicinal products.

As with any medicinal product, the development of ATMPs must also comply with Directive ****/**/EC regarding the application of good clinical practice in the conduct of clinical trials on medicinal products for human use(***), which provides specific rules for this type of medicinal product precisely because of its complexity.

In Italy, the main body responsible for the regulation of medicines is the Agenzia Italiana del Farmaco(***). The authorization procedure for advanced therapies involves several steps:

For preclinical and clinical evaluation, advanced therapy products must ...

6.1 Market segmentation

• Takeda Pharma
• BioMarin Pharmaceutical
• Kite Pharma
• Novartis
• Bristol Myers Squibb
• PTC Therapeutics
• Orchard Therapeutics
• CO.DON AG
• Fondazione Telethon
• Holostem