Enzyme Replacement Therapy Market

Enzyme Replacement Therapy Market Outlook from 2024 to 2034

According to recent medical studies, one in every seven thousand childbirths is affected by lysosomal storage disease. This number is on the verge of increasing year by year, especially in underdeveloped countries where awareness and medical facilities regarding these diseases are found to be very scarce. This has created a conducive environment for the enzyme replacement therapy market with a staggering valuation of USD 10,707.6 million expected as of 2024.

ERT is also used in the treatment of various rare diseases such as Gaucher disease and Fabry disease. Advancements in medicare technology and the proliferation of modern and sophisticated facilities in healthcare organizations, both public as well as private, have further led to the expansion of this market. The industry is anticipated to grow at a healthy CAGR of 6.60% from 2024 to 2034.

Government initiatives to make people more aware and lenient reimbursement policies are also responsible for the growth of the market. The market is slated to reach a valuation of USD 20,289.1 million by the end of 2034.

However, increased costs and unavailability of skilled workforce and healthcare facilities are hindering market growth in some parts of the world. Coupled with a lack of awareness and concerns regarding the efficacy of these treatments, the market has definite obstacles to face in the forecast period.

Attributes	Details
Market Value for 2024	USD 10,707.6 million
Projected Market Value for 2034	USD 20,289.1 million
Value-based CAGR of the Market for 2024 to 2034	6.60%

Category-wise Insights

Medical Professionals are Increasingly Relying on ERT for Mucopolysaccharidosis

ERT is used to treat various rare diseases such as Fabry Disease, Gaucher Disease, Mucopolysaccharidosis, etc. Among these, the Mucopolysaccharidosis segment is estimated to hold the largest market share of 24.10% in 2024.

Attributes	Details
Therapeutic Condition	Mucopolysaccharidosis
Market Share (2024)	24.10%

• The demand for ERT in treating Mucopolysaccharidosis is touching the skies. The treatment has been shown to effectively alleviate symptoms and slow disease progression in patients.
• Medical professionals rely on ERT as it helps reduce the accumulation of glycosaminoglycans (GAGs) in tissues and organs. This thereby alleviates disease symptoms such as skeletal abnormalities, organ enlargement, and neurological complications.

Injectable ERTs Gain Admiration over Oral Ones

ERTs can be administered through two major routes which are oral and injectable. Among these, the injectable ERT is predicted to hold a market share of 86.90% in 2024.

Attributes	Details
Route of Administration	Injectable
Market Share (2024)	86.90%

• Clamor for injectable ERTs is touching the skies as they offer a direct route of administration, allowing for efficient delivery of the therapeutic enzyme into the bloodstream. This not only ensures rapid but also reliable absorption of medications in the patient’s body.
• Injectable ERTs enable precise dosing, ensuring that patients receive the correct amount of enzyme with each administration. This is also contributing to its increased adoption in the medical world.

Country-wise Insights

Countries	CAGR (2024 to 2034)
Japan	8.00%
China	7.30%
Canada	6.90%
Australia and New Zealand	6.30%
Brazil	6.00%

Geriatric Population in Japan Benefits ERT Market Growth

The enzyme replacement therapy market in Japan is a promising one. It is slated to grow at a CAGR of 8.00% through 2034.

Japan is currently dealing with a growing aging population. As of 2024, one in every four people living in Japan is above the age of sixty. This has created a conducive environment for ERTs as this demographic is more susceptible to rare diseases.

Responding to these demands, Japanese healthcare companies are also investing billions of dollars in curating novel technologies and therapies to tackle these fatal diseases. For instance, in March 2022, Sanofi's Xenpozyme gained approval in Japan, marking a historic milestone as the inaugural treatment for ASMD.

Government Efforts to Make China One of the Lucrative Markets for ERT

China is a lucrative country in this market. The enzyme replacement therapy market is inclined to grow at a CAGR of 7.30% through 2034 in the country.

The Chinese government, in the last few years, has been announcing a multitude of schemes and policies to strengthen its medical infrastructure. The recent pandemic has also put a lot of pressure on the Chinese healthcare sector.

All these factors have led to the adoption of best-in-class medical facilities in the country. This improved medical infrastructure has led to the expansion of the ERT market as more people become aware of rare diseases for which this treatment is used.

Top-tier Medical Research Institutions to Profit Canadian ERT Market

The future of the Canada ERT market also looks very bright. The market is slated to grow at a CAGR of 6.90% through 2034 in Canada.

The presence of numerous medical institutes and government support in the form of grants and subsidies to the same have proved to be a boon for Canada’s healthcare sector. This has also benefitted ERTs as medicare research institutes have come up with top-tier methodologies for treating deadly diseases in the country.

Australia and New Zealand Exhibit Decent Progress in the ERT Market

The Australia and New Zealand ERT market is slated to grow at a CAGR of 6.30% from 2024 to 2034.

Although the incidence of LSDs in Australia and New Zealand is very scarce, both countries are conducting continuous research and development in these fields. The purpose is to entirely terminate these diseases from their population.

Players involved in the ERT market are pouring billions of dollars into making therapies affordable for the common public. For instance, in April 2024, Inozyme Pharma initiated the Phase Ib ENERGY-1 trial, dosing the first infant with INZ-701 enzyme therapy for ENPP1 deficiency, aiming to assess safety and efficacy.

Rising Incidences of LSDs to Increase the Adoption of ERTs in Brazil

The market in Brazil is very likely to flourish in the coming years. It is slated to grow at a CAGR of 6.00% through 2034.

Lysosomal storage disorders are gradually picking up pace in Brazil. To mitigate these diseases altogether Brazilian companies are collaborating with international partners. They are also investing in research and developing innovative therapies to address lysosomal storage disorders in the country.

Competitive Landscape

The enzyme replacement therapy market is a very limited one as the disease mortality rate is minuscule. This is why very few companies exist in the market, most of which have already established themselves as key players.

The competition is also not that intense as top players hold the majority share of the market. Besides this, these companies are found collaborating with research institutions for more effective and affordable therapies.

Recent Developments in the Enzyme Replacement Therapy Market:

• In February 2024, Sanofi unveiled positive outcomes for its enzyme replacement therapy, avalglucosidase alfa, for Pompe disease.
• In February 2024, the first trial for a gene therapy treating Hunter syndrome was launched in Manchester, United Kingdom, aiming to replace weekly enzyme therapy. Led by Professor Brian Bigger, the research follows successful trials in mice, offering hope for young patients.
• In January 2024, the USA FDA designated JR-441, an enzyme replacement therapy by JCR Pharmaceuticals, as an orphan drug for Sanfilippo syndrome type A. It received incentives for clinical development and review, aiming to aid its progress.
• In February 2024, BioMarin Pharma received a DOJ subpoena on sponsored testing programs for two therapies. The company disclosed it had provided documents and was cooperating but acknowledged uncertainty about compliance with laws.
• In November 2023, the FDA approved Takeda's enzyme replacement therapy for a rare blood disorder. It marked a significant milestone, offering hope for patients facing life-threatening conditions and highlighting advancements in medical treatment.
• In November 2023, AstraZeneca's rare disease unit received a NICE recommendation for its enzyme replacement therapy for Wolman disease in infants. This marked the first NHS-available treatment for the rare genetic condition, impacting around one in 350,000 births.
• In August 2023, Amicus Therapeutics launched Pombiliti for late-onset Pompe disease in the UK The therapy, a combination of cipaglucosidase alfa and miglustat, received approval from the MHRA. It aimed to address symptoms and improve patient's quality of life.

Key Companies

• Sanofi Genzyme
• Shire (now part of Takeda Pharmaceuticals)
• BioMarin Pharmaceutical Inc.
• Amicus Therapeutics
• Alexion Pharmaceuticals
• Ultragenyx Pharmaceutical Inc.
• Sarepta Therapeutics
• Biomarin
• Genzyme (now part of Sanofi)
• Takeda Pharmaceuticals
• Pfizer Inc.
• AbbVie Inc.
• Vertex Pharmaceuticals
• Sangamo Therapeutics
• Regeneron Pharmaceuticals Inc.
• Spark Therapeutics (now part of Roche)
• Orchard Therapeutics
• Novartis AG
• CSL Behring
• Audentes Therapeutics

Key Coverage in the Enzyme Replacement Therapy Market

• Industry trends in enzyme replacement therapy
• Comparative analysis of enzyme replacement therapies
• Market penetration strategies for ERT manufacturers
• Growth assessment of treatment options for lysosomal storage disorders
• Patient access and reimbursement challenges in ERT

Key Segments of the Enzyme Replacement Therapy Market

By Therapeutic Conditions:

• Enzyme Replacement Therapy for Fabry Disease
• Enzyme Replacement Therapy for Gaucher Disease
• Enzyme Replacement Therapy for Mucopolysaccharidosis (MPS):
  • MPS I (Hurler Syndrome)
  • MPS II (Hunter Syndrome)
  • MPS IVA (Morquio Syndrome, Type A)
  • MPS VI (Maroteaux-Lamy Syndrome)
  • MPS VII (Sly Syndrome)
• Enzyme Replacement Therapy for Pompe Disease
• Enzyme Replacement Therapy for Lysosomal Acid Lipase Deficiency
• Others

By Route of Administration:

• Oral Enzyme Replacement Therapy
• Injectable Enzyme Replacement Therapy

By Distribution Channel:

• Hospital Pharmacies
• Specialty Treatment Pharmacies
• Retail Pharmacies

By Region:

• North America
• Latin America
• Europe
• Asia Pacific
• Middle East and Africa

Frequently Asked Questions

How Much is the Enzyme Replacement Therapy Market Currently Worth?

The market is expected to be worth USD 10,707.6 million in 2024.

What is the Sales Forecast for Enzyme Replacement Therapy Through 2034?

The enzyme replacement therapy market is expected to reach USD 20,289.1 million by 2034.

At what Rate is the Enzyme Replacement Therapy Market Growing?

The market is in line to grow at a CAGR of 6.60% from 2024 to 2034.

Which are the key Companies in the Enzyme Replacement Therapy Market?

BioMarin Pharmaceutical Inc., Amicus Therapeutics, Sanofi Genzyme, Alexion Pharmaceuticals, and Ultragenyx Pharmaceutical Inc., are some of the major players in the market.

What was the Value of the Enzyme Replacement Therapy Market in 2023?

The market valuation in 2023 was USD 10,011.1 million.