Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market

Growing Prevalence of Lysosomal Storage Disorders (LSD)

The global burden of lysosomal storage disorders (LSD) has aggrandized in recent years. Based on an exhaustive study undertaken in Australia between January 1980 and December 1986, over 27 LSDs were diagnosed in over five hundred individuals.

At least 1 in 57,000 infants was diagnosed with Gaucher disease while 1 per 4.2 million live births detected evidence of sialidosis. Since then, at least 41 distinct genetic diseases became part and parcel of LSDs.

Attributed to this increasing incidence, government bodies across the world are encouraging efforts to accelerate development of effective treatment solutions based on enzyme replacement therapy (ERT), leading to the spawning of numerous manufacturers across key regions.

Proliferating Orphan Drug Designations to Broaden Scope

The National Center for Biotechnology Information (NCBI) estimates that between 1983 and 2019, 124 orphan drug designations were granted by the Food & Drug Administration (FDA) for compounds intended to treat 28 lysosomal storage diseases.

These designations were mostly for Gaucher disease (16), Fabry disease (10), Pompe disease (16), Mucopolysaccharidoses (MPS) II (10), MPS I (9) and a host of other genetic disorders. Presently, the drug development pipeline for LSDs is ever evolving, with high focus on gene therapy and small-molecule targets.

In 2019, the FDA granted an orphan drug designation to Avrobio’s AVR-RD-01 investigational gene therapy for treating Fabry disease. The therapy is based on autologous stem cell transplant, using hematopoietic stem cells to produce functional alpha-galactosidase-A enzyme. 

The Nudge from Key Collaborations

The Gaucher and Pompe Disease ERT market is characterized by numerous collaborations and alliances amongst major players to fast-track development of replacement enzymes, an important growth catalyst for the market in forthcoming years.

For instance, in 2018, Takeda Pharmaceuticals, formerly Shire PLC, entered into a collaboration with NanoMedSyn to further research for potential lysosomal storage disorder treatment based on the latter’s proprietary AMFA technology.

Likewise, in 2017, Protalix Biotherapeutics entered into a license collaboration agreement with Chiesi Farmaceutici S.p.A with the objective of commercializing its pegunigalsidase alfa (PRX-102) for the treatment of Fabry disease in markets outside the United States.

Alternative Treatment Approaches to Impede Growth

While ERT has significantly improved the prognosis in infantile and later onset of Pompe and Gaucher Diseases, inefficiencies regarding distribution and uptake in the target muscular system has prompted healthcare providers to find alternative approaches for more effective treatments.

Moreover, the presence of the blood-brain barrier and pathological preconditions further prove to be obstacles for effective uptake of ERT. Efforts are thus underway to develop more precise and accurate treatment methods.

For instance, a research team from the Cincinnati Children’s Hospital have touted the C5aRA molecule as a more effective approach to counter a molecular pathway which drives pro-inflammatory processes in Gaucher Disease. 

Gaucher and Pompe Disease ERT Market: Regional Insights

Europe Gaucher and Pompe Disease ERT Market

Europe is likely to dominate the market, generating 30% of the global revenue by 2020-end. Growth is attributed to numerous regulatory approvals granted to multiple treatment approaches in recent years.

Recently, in October 2020, the European Medicines Agency (EMA) accepted a review of the Marketing Authorization Application (MAA) for avalglucosidase alfa ERT for long-term treatment of patients with Pompe Disease.

Besides this, numerous approvals for other disorders requiring enzymatic therapy have also opened up the market for massive investments, For instance, in 2017, Allergan received approval for its Enzepi pancreatic ERT by the Committee for Medicinal Products for Human Use (CHMP) for use by infants, children and adults.

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North America Gaucher and Pompe Disease ERT Market

Until 2019, North America generated 1/4^th of the global revenue share in the Gaucher & Pompe Disease ERT market. Growth is attributed to the high incidence of genetic disorders amongst the local population.

As per the National Organization for Rare Disorders, nearly 6,000 individuals across the United States are afflicted by Gaucher disease, thus broadening the scope for treatment in recent years.

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Presently, the FDA has approved numerous ERT and SRT (Substrate Reduction Therapy) drugs such as Cerdelga^®, Zavesca^® and VPRIV^® across multiple healthcare settings.

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East Asia Gaucher and Pompe Disease ERT Market

The East Asian market is yet another lucrative revenue hotspot for the Gaucher and Pompe Disease ERT market. Growth is primarily attributed to a proliferating healthcare infrastructure in order to deliver state-of-the-art medical treatment.

Since the medicines are quite expensive, governments are subsidizing rates to alleviate financial strain on customers. For instance, in China, one Cerezyme treatment session costs around 23,000 yuan (US$ 3,250), totaling to over 2 million yuan annually.

Provincial governments, therefore, are providing public health insurance which covers up to 80% of Cerezyme treatments such as in Qingdao. Such measures are helping patients avail treatment on a much larger scale, thus broadening growth prospects.

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Category-wise Insights

Type-1 Gaucher Disease Remains Lucrative

Type-1 Gaucher Disease is the most prevalent condition, also called neuronopathic Gaucher Disease, as it leaves the brain and spinal cord intact. The disease occurs in 1 out of 50,000 to 100,000 people, mostly affecting the Ashkenazi Jews of Eastern and Central Europe.

According to FMI’s analysis, the type-1 Gaucher disease segment is expected to register a CAGR of nearly 8% by 2020-end. Several ERTs are currently available to treat this condition, including Sanofi’s Cerezyme injection, Shire PLC’s VPRIV proprietary gene activation method and Pfizer’s Elelyso plant-cell recombinant ERT.

Parenteral Administration Remains Preferred

Maximum patients prefer a parenteral administration of Gaucher and Pompe ERT drugs due to high demand for precision-based treatment and delivery of faster results. Parenteral administration also ensures timely compliance of medicine schedules, which may not be the case while consuming oral medication.

According to the National Center for Biotechnology Information (NCBI), around 4% of American adults suffer from dysphagia, while a significant proportion cannot swallow a drug due to nausea. Based on these factors, the parenteral administration segment is expected to generate over 80% of the revenue by 2020.

Online Pharmacies Acquiring Popularity

Hospital and specialty treatment pharmacies are expected to constitute 3 out of 4 sales of ERT drugs and injections throughout 2020, attributed to high degree of reliability regarding availability of the prescribed medications.

However, sales across online pharmacies are experiencing notable upsurge, expanding at over 7% CAGR by the end of the current year. Growth is largely attributed to increasing frequency of home-based ERT treatments in the wake of the pandemic crisis.  

COVID-19’s Impact on the Gaucher & Pompe Disease ERT Market

COVID-19 has deeply impacted the global healthcare industry. According to IMF’s World Economic Output report published in April 2020, the burden of pharmaceutical industry contraction has been heavy for the developed world, which have experienced an economic deceleration worth 6%, a significant drop in comparison to the 2007-2008 financial crisis.

With respect to surgeries and treatments, top priority is being given to COVID-19 positive patients, leading to delays and postponement of several procedures. This has also impacted the Gaucher & Pompe Disease ERT segment, with countries such as Spain recording a shortfall of nearly a quarter for hospital-based treatment.

Fortunately, the blip has not lasted for long, with government authorities directing healthcare providers to resume treatment for Type-1 and Type-3 Gaucher Disease patients. Studies have concluded that people suffering from this disorder also have co-morbidities which could jeopardize their survival rate in case of a COVID-19 infection.

Gaucher & Pompe Disease ERT Market: Competitive Landscape

The Gaucher & Pompe Disease ERT landscape is characterized by the presence of several prominent healthcare and pharmaceutical corporations, concentrating on path breaking technological developments to introduce more effective therapeutic solutions by virtue of strategic collaborations with various entities.

Prominent players profiled in FMI’s study on the market include*:

• Pfizer Inc.
• Alexion Pharmaceuticals Inc.
• BioMarin Pharmaceutical Inc.
• Ultragenyx Pharmaceutical Inc.
• Janssen Pharmaceuticals Inc.
• Sigma-Tau Pharmaceuticals Inc.
• AbbVie Inc.
• Sanofi SA
• Shire PLC
• Merck KGaA
• Tadeka Pharmaceuticals

*This list is indicative- full details about Gaucher & Pompe Disease market players are available upon request.

In November 2020, Ultragenyx Pharmaceutical Inc. announced plans to establish a new large-scale manufacturing facility for gene therapy products in Bedford, Massachusetts, enabling the pharmaceutical giant to manufacture in-house its pipeline of clinical stage adeno-associated virus-based (AAV) gene therapies for ornithine transcarbamylase (OTC) deficiency.

In 2019, Alexion Pharmaceutical Inc. acquired Achillon Pharmaceuticals Inc. with the objective of furthering development of oral small molecule Factor D inhibitors for treating diseases such as paroxysmal nocturnal hemoglobinuria (PNH) and C3 glomerulopathy (C3G).

Sanofi SA is an important pioneer in the field of ERT for Gaucher Disease. Its Cerezyme® injection, manufactured by Genzyme, a subsidiary of Sanofi, is the only ERT which has shown long term efficacy and safety across multiple studies for pediatric and adult patients diagnosed with Type-1 Gaucher Disease. 

Gaucher and Pompe Disease ERT Market: Report Scope

Key questions answered in the report

• How will the Gaucher and Pompe Disease ERT Market expand in the future?

  According to research conducted by Future Market Insights (FMI), the Gaucher and Pompe Disease ERT Market is expected to remain on a positive trajectory, despite encountering a blip in the initial months of COVID-19, attributed to growing incidence of lysosomal storage disorders (LSD)

• What drivers underpin the Gaucher and Pompe Disease ERT Market growth?

  The Gaucher and Pompe Disease ERT Market is growing on the back of increasing orphan drug status designation to numerous drug candidates by the FDA and growing number of collaborations and partnerships among prominent market players

• Which is the most prominent therapeutic condition?

  Type-1 neuronopathic Gaucher disease is expected to generate maximum growth opportunities, owing to its widespread prevalence amongst patients suffering from Gaucher disease

• Which is the largest Gaucher and Pompe Disease ERT Market?

  Europe is expected to be the fastest growing and largest Gaucher and Pompe Disease ERT Market, owing to enhanced approvals by regulatory authorities to different drug candidates

• Which are the prominent Gaucher and Pompe Disease ERT Market players?

  Pfizer, Alexion Pharmaceuticals, Inc., BioMarin Pharmaceutical Inc., Ultragenyx Pharmaceutical Inc., Janssen Pharmaceuticals, Inc. (J&J), Sigma-Tau Pharmaceuticals Inc., AbbVie Inc., Sanofi S.A., Shire Plc., Merck KGaA and Takeda Pharmaceuticals are some leading players within the landscape

Enquiry before Buying

• Is the Gaucher and Pompe Disease ERT market research conducted by Future Market Insights?

  Yes, the Gaucher and Pompe Disease ERT market report has been compiled by expert analysts of Future Market Insights through a combination of primary and secondary research. To know more about how the research was conducted, you can speak to the research analyst

• What research methodology is followed by FMI?

  FMI follows a methodology that encompasses the demand side assessment of the market, and triangulates the same through a supply side analysis. This methodology is based on use of standard market structure, methods and definitions.

• Who are the respondents for primary research?

  FMI speaks to stakeholders across the spectrum, including C-level executives, distributors, product manufacturers, industry experts. For a full list of primary respondents, please reach out to us.

• What are the sources of secondary research?

  FMI conducts extensive secondary research through proprietary databases, paid databases, and information available in the public domain. We refer to industry associations, company press releases, annual reports, investor presentations, and research papers. More information about desk research is available upon request. 

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Key Segments

Therapeutic condition

• Gaucher Disease
  • Type 1 (non-neuropathic)
  • Type 2 (acute infantile neuronopathic)
  • Type 3 Gaucher disease (chronic neuronopathic)
  • Perinatal lethal Gaucher disease
  • Cardiovascular Gaucher disease
• Pompe Disease
  • Classic infantile-onset
  • Non-classic infantile-onset
  • Late-onset

Route of Administration

• Oral
• Parenteral

Distribution Channel

• Hospital Pharmacies
• Specialty Treatment Pharmacies
• Retail Pharmacies
• Online Pharmacies

Region

• North America (U.S & Canada)
• Latin America (Brazil, Mexico, Argentina & Rest of LATAM)
• Europe (U.K, Germany, France, Spain, Italy, Russia & Rest of Europe)
• East Asia (China, Japan & South Korea)
• South Asia (India, Thailand, Indonesia, Malaysia & Rest of South Asia)
• Oceania (Australia & New Zealand)
• Middle East & Africa (GCC, South Africa, Turkey & Rest of MEA)