Si prevede che il mercato della terapia enzimatica sostitutiva crescerà da 9.673,39 milioni di dollari nel 2021 a 15.184,70 milioni di dollari entro il 2028; si stima che crescerà a un CAGR del 6,8% dal 2022 al 2028.
La terapia enzimatica sostitutiva (ERT) viene eseguita per trattare le carenze enzimatiche congenite utilizzando enzimi o proteine con attività enzimatica. Gli enzimi animali, umani e ingegnerizzati ricombinanti sono alcune fonti di enzimi necessari per il trattamento. La terapia viene generalmente applicata nelle malattie rare e da accumulo lisosomiale, tra cui la malattia di Pompe, la malattia di Fabry, la malattia di Gaucher, la malattia di Tay-Sachs e la sindrome di Hurler. Il metodo ERT più comune è l'infusione endovenosa, in cui l'enzima sostitutivo viene somministrato direttamente nel flusso sanguigno attraverso un gocciolamento controllato di liquidi. La crescita del mercato globale della terapia enzimatica sostitutiva è attribuita alla crescente prevalenza delle malattie da accumulo lisosomiale (LSD) e alla rapida approvazione normativa con altri vantaggi di marketing per i farmaci con designazione di farmaco orfano. Tuttavia, la concorrenza spietata tra gli operatori del mercato ostacola la crescita del mercato.
Il rapporto offre approfondimenti e analisi approfondite del mercato globale della terapia enzimatica sostitutiva, sottolineando vari parametri tra cui tendenze del mercato, progressi tecnologici, dinamiche di mercato, e analisi del panorama competitivo dei principali attori del mercato globale. Comprende anche l’impatto della pandemia COVID-19 sul mercato in tutte le regioni. A causa della pandemia di COVID-19, molte autorità sanitarie si sono concentrate sulle cure legate alla pandemia. Inoltre, molti pazienti che necessitano di terapia enzimatica sostitutiva hanno saltato le infusioni nel marzo 2020 a causa dell’inadeguatezza dei servizi ospedalieri che affrontano il peso dei pazienti affetti da COVID-19. I pazienti che necessitano di ERT hanno affermato di sentirsi ansiosi durante i ricoveri ospedalieri o riguardo alla fornitura di farmaci. Inoltre, gli studi hanno rivelato che alcuni pazienti soffrivano di depressione e di un aumento del livello di ansia. Pertanto, le sessioni programmate di terapia enzimatica sostitutiva (ERT) sono state interrotte durante la pandemia di COVID-19, con un impatto negativo sul mercato.
Approfondimenti strategici
Approfondimenti basati sulla geografia
In base all'area geografica, il mercato globale della terapia enzimatica sostitutiva è segmentato in Nord America (Stati Uniti, Canada e Messico), Europa (Francia, Germania, Regno Unito, Spagna, Italia e resto d'Europa), Asia Pacifico ( Cina, India, Giappone, Australia, Corea del Sud e resto dell'APAC), Medio Oriente e altri paesi. Africa (Arabia Saudita, Emirati Arabi Uniti, Sud Africa e resto del MEA) e Sud & America centrale (Brasile, Argentina e il resto dell'America meridionale e centrale).
Regioni redditizie per il mercato globale della terapia enzimatica sostitutiva
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Approfondimenti di mercato
Crescente prevalenza delle malattie da accumulo lisosomiale (LSD)
Un lisosoma è un organello cellulare legato alla membrana che contiene enzimi digestivi. Un lisosoma è l'hub cellulare chiave per il catabolismo, il riciclaggio e la segnalazione delle macromolecole. Qualsiasi difetto in queste funzioni porta all'accumulo o allo stoccaggio di macromolecole nei lisosomi, con conseguente danno cellulare. La causa principale delle malattie da accumulo lisosomiale (LSD) sono le mutazioni nei geni responsabili della codifica di un enzima lisosomiale. Inoltre, gli LSD sono caratterizzati da un errore congenito del metabolismo che provoca l'assenza o la carenza di un enzima. I neonati e i bambini soffrono più gravemente rispetto agli adulti poiché i bambini possono ereditare il gene difettoso da uno o entrambi i genitori. Tuttavia, dall'ultimo decennio, il modello è cambiato e l'LSD è più comune negli adulti
Inoltre, l'LSD copre circa 70 malattie, tra cui la malattia di Gaucher, la malattia di Niemann-Pick, la malattia di Fabry, la malattia di Tay-Sachs malattia, malattie delle mucopolisaccaridosi (MPS) e malattia di Pompe. Questi disturbi sono rari individualmente, ma collettivamente sono significativamente prevalenti e colpiscono 1 su 5.000 nati vivi. Molti paesi hanno condotto indagini per comprendere il modello di prevalenza e la prevalenza complessiva era in aumento. Ad esempio, uno studio pubblicato su The Lancet Regional Health intitolato "Prevalenza dei disturbi da accumulo lisosomiale in Australia dal 2009 al 2020". Lo studio ha rivelato i seguenti risultati:
- La prevalenza nella popolazione australiana è 1,6 volte (1 su 4.800 nati vivi) , superiore alla prevalenza riportata nel 1996 (1 su 7.700 nati vivi).
- La malattia di Fabry era la più diffusa, rappresentando il 34% di tutte le diagnosi (fino al 2020) .
- L'LSD è più comune nell'età adulta che nell'infanzia
Pertanto, la crescente prevalenza dell'LSD e le terapie limitate per curarla la malattia sta guidando la crescita del mercato globale della terapia enzimatica sostitutiva
Approfondimenti basati sul tipo di enzima
In base al tipo di enzima, il mercato globale della terapia enzimatica sostitutiva è segmentato in alglucosidasi alfa, agalsidasi beta, imiglucerasi, idursulfasi, galsulfasi, velaglucerasi alfa e altri enzimi. Nel 2021, il segmento degli altri enzimi rappresentava la quota di mercato maggiore. Si prevede che lo stesso segmento registrerà il CAGR più elevato del mercato dal 2022 al 2028.
Mercato globale della terapia sostitutiva enzimatica, per tipo di enzima: 2021 e 2028
Approfondimenti basati sulle condizioni terapeutiche
In base alle condizioni terapeutiche, il mercato globale della terapia enzimatica sostitutiva è segmentato in malattia di Gaucher, malattia di Fabry, malattia di Pompe, SCID, MPS e altre condizioni terapeutiche. Il segmento della malattia di Gaucher ha detenuto la quota di mercato maggiore nel 2021 e si prevede che registrerà il CAGR più elevato durante il periodo di previsione.
Percorso di approfondimenti basati sull'amministrazione
Basato sul percorso di somministrazione, il mercato globale della terapia enzimatica sostitutiva è biforcato in parenterale e orale. Il segmento parenterale ha detenuto una quota di mercato maggiore nel 2021 e si prevede che registrerà un CAGR più elevato durante il periodo di previsione.
Approfondimenti basati sull'utente finale
Basati sull'utente finale, l'enzima globale Il mercato della terapia sostitutiva è segmentato in ospedali, centri di infusione e altri. Il segmento degli ospedali ha detenuto la quota di mercato maggiore nel 2021. Tuttavia, si prevede che il segmento dei centri di infusione registrerà il CAGR più elevato durante il periodo di previsione.
Gli attori globali del mercato della terapia enzimatica sostitutiva adottano strategie organiche, compreso il lancio del prodotto ed espansione, per espandere la propria presenza e il portafoglio di prodotti in tutto il mondo e soddisfare la crescente domanda. Alcuni dei principali attori che contribuiscono al mercato sono Takeda Pharmaceutical Company Limited; Sanofi SA; AbbVie Inc.; BioMarin Pharmaceutical Inc.; Amicus Therapeutics; Alexion Pharmaceuticals, Inc. (AstraZeneca); Janssen Pharmaceuticals (Johnson & Johnson Services, Inc.); Recordati SpA; Pfizer Inc.; e CHIESI Farmaceutici SpA
Report Coverage
Revenue forecast, Company Analysis, Industry landscape, Growth factors, and Trends
Segment Covered
This text is related
to segments covered.
Regional Scope
North America, Europe, Asia Pacific, Middle East & Africa, South & Central America
Country Scope
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to country scope.
Frequently Asked Questions
Enzyme replacement therapy (ERT) is a treatment that replaces enzymes that are not present or are deficient in the body. This is usually done by giving the patient an intravenous (IV) infusion or oral solution in which the enzyme is present. ERT is available for various lysosomal storage diseases which include Fabry’s disease, Gaucher’s disease, Pompe’s disease, MPS, and others. The ERT only increases the concentration of the enzymes which is lacking in the patient body and it does not correct any underlying genetic defect. ERT has also been used to treat patients with severe combined immunodeficiency (SCID) resulting from an adenosine deaminase deficiency (ADA-SCID).
Rising prevalence of lysosomal storage diseases (LSDS) and rapid regulatory approval with other benefits for drug with orphan drug designation are the most significant factors responsible for the overall market growth.
Based on enzyme type, other enzymes segment took the forefront lead in the worldwide market by accounting largest share in 2021 and is expected to continue to do so till the forecast period. Other enzymes include taliglucerase, laronidase, agalsidase alfa, eliglustat, cipaglucosidase alfa, miglustat, elosulfase alfa, elapegademase, velmanase alfa, pancreatic enzymes, pegademase, sebelipase alfa, cerliponase alfa, among others.
Based on therapeutic conditions, Gaucher's disease segment took the forefront lead in the worldwide market by accounting largest share in 2021 and is expected to continue to do so till the forecast period.
Based on route of administration, parenteral route of administration segment took the forefront lead in the worldwide market by accounting largest share in 2021 and is expected to continue to do so till the forecast period.
The hospitals segment dominated the global enzyme replacement therapy market and accounted for the largest market share of 49.00% in 2021.
Takeda Pharmaceutical Company Limited, Sanofi, AbbVie Inc., BioMarin Pharmaceutical Inc., Amicus Therapeutics, Alexion Pharmaceuticals, Inc.(AstraZeneca), Janssen Pharmaceuticals (Johnson & Johnson Services, Inc.), Recordati S.p.A., Pfizer Inc., and CHIESI Farmaceutici S.p.A. are among the leading companies operating in the enzyme replacement therapy market
Global enzyme replacement therapy market is segmented by region into North America, Europe, Asia Pacific, Middle East & Africa, and South & Central America. In North America, the U.S. is the largest market for enzyme replacement therapy market. A rise in the prevalence of lysosomal storage diseases such as Gaucher’s, Pompe’s, and Fabry’s diseases, increasing regulatory approval, favorable regulatory support by the government, and the availability of various ERT products will drive enzyme replacement therapy market growth in North America. However, the growing government funding for rare disease treatment, increasing clinical trials, an increasing number of product approvals, and expanding partnerships among key players are the key factors responsible for the Asia-Pacific regional growth of the enzyme replacement therapy market accounting fastest growth in the region during the coming years.
The List of Companies - Enzyme Replacement Therapy Market
- Sanofi
- BioMarin Pharmaceutical Inc.
- Takeda Pharmaceutical Company Limited
- AbbVie Inc.
- Janssen Pharmaceuticals (Johnson & Johnson Services, Inc.)
- Alexion Pharmaceuticals, Inc (AstraZeneca)
- Amicus Therapeutics
- Recordati S.p.A.
- CHIESI Farmaceutici S.p.A.
- Pfizer Inc.
The Insight Partners performs research in 4 major stages: Data Collection & Secondary Research, Primary Research, Data Analysis and Data Triangulation & Final Review.
- Data Collection and Secondary Research:
As a market research and consulting firm operating from a decade, we have published and advised several client across the globe. First step for any study will start with an assessment of currently available data and insights from existing reports. Further, historical and current market information is collected from Investor Presentations, Annual Reports, SEC Filings, etc., and other information related to company’s performance and market positioning are gathered from Paid Databases (Factiva, Hoovers, and Reuters) and various other publications available in public domain.
Several associations trade associates, technical forums, institutes, societies and organization are accessed to gain technical as well as market related insights through their publications such as research papers, blogs and press releases related to the studies are referred to get cues about the market. Further, white papers, journals, magazines, and other news articles published in last 3 years are scrutinized and analyzed to understand the current market trends.
- Primary Research:
The primarily interview analysis comprise of data obtained from industry participants interview and answers to survey questions gathered by in-house primary team.
For primary research, interviews are conducted with industry experts/CEOs/Marketing Managers/VPs/Subject Matter Experts from both demand and supply side to get a 360-degree view of the market. The primary team conducts several interviews based on the complexity of the markets to understand the various market trends and dynamics which makes research more credible and precise.
A typical research interview fulfils the following functions:
- Provides first-hand information on the market size, market trends, growth trends, competitive landscape, and outlook
- Validates and strengthens in-house secondary research findings
- Develops the analysis team’s expertise and market understanding
Primary research involves email interactions and telephone interviews for each market, category, segment, and sub-segment across geographies. The participants who typically take part in such a process include, but are not limited to:
- Industry participants: VPs, business development managers, market intelligence managers and national sales managers
- Outside experts: Valuation experts, research analysts and key opinion leaders specializing in the electronics and semiconductor industry.
Below is the breakup of our primary respondents by company, designation, and region:
Once we receive the confirmation from primary research sources or primary respondents, we finalize the base year market estimation and forecast the data as per the macroeconomic and microeconomic factors assessed during data collection.
- Data Analysis:
Once data is validated through both secondary as well as primary respondents, we finalize the market estimations by hypothesis formulation and factor analysis at regional and country level.
- Macro-Economic Factor Analysis:
We analyse macroeconomic indicators such the gross domestic product (GDP), increase in the demand for goods and services across industries, technological advancement, regional economic growth, governmental policies, the influence of COVID-19, PEST analysis, and other aspects. This analysis aids in setting benchmarks for various nations/regions and approximating market splits. Additionally, the general trend of the aforementioned components aid in determining the market's development possibilities.
- Country Level Data:
Various factors that are especially aligned to the country are taken into account to determine the market size for a certain area and country, including the presence of vendors, such as headquarters and offices, the country's GDP, demand patterns, and industry growth. To comprehend the market dynamics for the nation, a number of growth variables, inhibitors, application areas, and current market trends are researched. The aforementioned elements aid in determining the country's overall market's growth potential.
- Company Profile:
The “Table of Contents” is formulated by listing and analyzing more than 25 - 30 companies operating in the market ecosystem across geographies. However, we profile only 10 companies as a standard practice in our syndicate reports. These 10 companies comprise leading, emerging, and regional players. Nonetheless, our analysis is not restricted to the 10 listed companies, we also analyze other companies present in the market to develop a holistic view and understand the prevailing trends. The “Company Profiles” section in the report covers key facts, business description, products & services, financial information, SWOT analysis, and key developments. The financial information presented is extracted from the annual reports and official documents of the publicly listed companies. Upon collecting the information for the sections of respective companies, we verify them via various primary sources and then compile the data in respective company profiles. The company level information helps us in deriving the base number as well as in forecasting the market size.
- Developing Base Number:
Aggregation of sales statistics (2020-2022) and macro-economic factor, and other secondary and primary research insights are utilized to arrive at base number and related market shares for 2022. The data gaps are identified in this step and relevant market data is analyzed, collected from paid primary interviews or databases. On finalizing the base year market size, forecasts are developed on the basis of macro-economic, industry and market growth factors and company level analysis.
- Data Triangulation and Final Review:
The market findings and base year market size calculations are validated from supply as well as demand side. Demand side validations are based on macro-economic factor analysis and benchmarks for respective regions and countries. In case of supply side validations, revenues of major companies are estimated (in case not available) based on industry benchmark, approximate number of employees, product portfolio, and primary interviews revenues are gathered. Further revenue from target product/service segment is assessed to avoid overshooting of market statistics. In case of heavy deviations between supply and demand side values, all thes steps are repeated to achieve synchronization.
We follow an iterative model, wherein we share our research findings with Subject Matter Experts (SME’s) and Key Opinion Leaders (KOLs) until consensus view of the market is not formulated – this model negates any drastic deviation in the opinions of experts. Only validated and universally acceptable research findings are quoted in our reports.
We have important check points that we use to validate our research findings – which we call – data triangulation, where we validate the information, we generate from secondary sources with primary interviews and then we re-validate with our internal data bases and Subject matter experts. This comprehensive model enables us to deliver high quality, reliable data in shortest possible time.
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