Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Prominent Players : Shire PLC, Ultragenyx Pharmaceuticals Inc., Sigma-Tau Pharmaceuticals Inc., Pfizer Inc., Alexion Pharmaceuticals Inc., Sanofi SA

As the global burden of genetic disorders such as Gaucher disease, Fabry disease, Pompe disease and other abnormalities rise, healthcare providers are scrambling to develop potential therapeutic approaches to offset their impacts.

According to the National Gaucher Foundation, one in every 450 people belonging to the Ashkenazi Jewish community suffer from Type-1 Gaucher disease, the most prevalent form, thereby opening up credible growth prospects for the future.

Key Takeaways

• Gaucher and Pompe Disease ERT market to expand at 5.6% value CAGR through 2020
• Type-1 non-neuronopathic Gaucher disease to remain key treatment area, accounting for over 90% revenue by 2020-end
• Parenteral ERT administration to be most frequent, attributed to high demand for precision-based medicine
• Online pharmacies gaining credible impetus, expanding at over 7% CAGR through 2030
• Europe to retain lucrativeness, North America to be the 2^nd largest market by 2030

“Increasing regulatory approvals by government authorities is paving way for the entry of prominent enzyme replacement therapy providers, thus generating immense revenue prospects across promising markets,” concludes the FMI analyst.

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COVID-19 Impact AnalysisAs healthcare organization reorient their resources to fight the coronavirus pandemic, the market for Gaucher & Pompe Disease ERT experienced a substantial blip in the initial months, with treatment sessions declining significantly across several countries.

For instance, until July 2020, Spain recorded a 25% decline in enzyme replacement therapy sessions amongst people suffering from Type-1 and Type-3 Gaucher Disease respectively. This largely occurred as a consequence of rescheduling treatments at a later date.

In the United States, hospital visits to avail ERT have registered considerable decline for avoiding exposure to COVID-19 patients. As a result, patient self-administration at home have burgeoned. Furthermore, the pandemic has provided an opportunity to undertake research on lysosomal dysfunction and disease pathogenesis which would enhance clinical care.

Competitive LandscapeProminent players operating in the Gaucher & Pompe Disease ERT landscape include Shire PLC, Ultragenyx Pharmaceuticals Inc., Sigma-Tau Pharmaceuticals Inc., Pfizer Inc., Alexion Pharmaceuticals Inc., Sanofi SA, Merck KGaA, Tadeka Pharmaceuticals, AbbVie Inc. and BioMarin Pharmaceutical Inc. to name a few.

Manufacturers rely on introduction of novel technologies to advance effective ERTs for numerous genetic conditions. For instance, in October 2018, the U.S Food & Drug Administration approved Sigma-Tau Pharmaceuticals’ (Lediant Biosciences) Revcovi^TM injection for treating adenosine deaminase severe combined immune deficiency (ADA-SCID).

Likewise, Sanofi SA markets the Cerezyme^® ERT ^­injections through its Genzyme subsidiary, which offers a long term efficacy to elderly and pediatric patients suffering from Type-1 Gaucher Disease, and is the only known long-term cure for the condition.

More Insights on FMI’s Gaucher and Pompe Disease ERT Market ReportFuture Market Insights, in its new report, offers an unbiased analysis of the global Gaucher and Pompe Disease ERT market, analyzing historical demand from 2015-2019 and forecast statistics for 2020-2030. The study reveals growth projections on the Gaucher and Pompe Disease ERT Market on the basis of therapeutic condition (Gaucher disease and Pompe disease), route of administration (oral and parenteral) and distribution channel (hospital pharmacies, specialty treatment pharmacies, retail pharmacies and online pharmacies) across seven prominent regions.

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Key SegmentsTherapeutic 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)

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