Global Fabry Disease Treatment Market - 2021-2028
発行: DataM Intelligence
ページ情報: 英文 180 Pages
The global Fabry Disease Treatment Market size was valued US$ XX billion in 2020 and is estimated to reach US$ XX billion by 2028, growing at a CAGR of XX % during the forecast period (2021-2028).
Fabry disease also called as Anderson-Fabry disease and alpha-galactosidase-A deficiency is an X-linked, hereditary, lysosomal storage disease caused due to paucity of an enzyme α galactosidase A (an enzyme required to metabolize lipids, fat-like substances that include oils, waxes, and fatty acids).
The global fabry disease treatment market growth is driven by the expanding base of patients suffering from fabry disease coupled with increased adoption of novel therapies such as chaperone treatment has helped the market gain momentum. Besides this, extensive R&D activities and potential approval of promising pipeline products including substrate reduction therapies and enzyme replacement therapies are projected to drive the growth of the over the forecast period.
The presence of robust medication pipeline indicated for treatment of fabry disease is expected to drive the growth in the forecast period
The presence of robust medication pipeline indicated for treatment of fabry disease is also expected to be a major factor fueling growth of global fabry disease treatment market over the forecast period.
In recent past, various regulatory bodies of key regions have approved novel medications for treatment of fabry disease. Approval and launch of such novel medications is expected to significantly support global fabry disease treatment market growth over the forecast period.
For instance, in August 2018, Amicus Therapeutics, Inc., received the U.S. Food and Drug Administration (FDA) approval for its drug Galafold (migalastat). It is the first oral medication indicated for the treatment of adults with Fabry disease.
Furthermore, key players in the market are majorly investing in development of novel therapies for Fabry disease treatment, such strategic funding's by various players is expected to drastically fuel global fabry disease treatment market growth over the forecast period.
For instance, in February 2018, AvroBio Inc., closed US$ 60 million financing to support the phase 2 study of its novel gene therapy, AVR-RD-01 underdevelopment for the treatment of Fabry disease. Considering these factors will drive the growth of the market in the forecast period.
Lack of a curative solution for Fabry disease treatment are projected, expected to drive the market growth
Patients suffering from Fabry disease commonly experience stroke, extreme pain, early heart disorder, compromised kidney function often leading to full kidney failure, and disabling gastrointestinal symptoms. Due to this lack of a curative solution for Fabry disease treatment options aims to manage complications associated with disease progression and giving symptomatic relief. Many of times, various patients remain undiagnosed due to moderate symptoms. Considering these factors, the global fabry disease market is projected to grow at a significate rate in the forecast period.
Low diagnostic rate due to unavailability of such diagnostic procedures and trained professionals is likely to hinder the market growth
However, fabry disease is a very rare disease affecting around 1 individual in 50,000 (according to the National Fabry Disease Foundation), the diagnosis of Fabry disease is confirmed by demonstrating an enzyme deficiency in males and by identifying the specific GLA gene mutation in males and females. Hence, low diagnostic rate due to unavailability of such diagnostic procedures and trained professionals in emerging economies are expected to be major factor restraining global fabry disease treatment market growth.
COVID-19 Impact Analysis
Coronavirus Disease-19 (COVID-19), caused by the SARS-CoV-2 virus was labeled as a global pandemic and patients with fabry disease can be considered at high risk of developing severe complications. The impact of COVID-19 on patients with fabry disease receiving enzyme replacement therapy is still unknown. Many patients who receive treatment in the hospital experienced infusion disruptions due to fear of infection. Effects of temporary treatment interruption was described in more detail in other lysosomal storage diseases, but the recommencement of therapy does not fully reverse clinical decline due to the temporary discontinuation. When possible, home-therapy seems to be the most efficient way to maintain enzyme replacement therapy access during pandemic. These factors will drive the growth of the market in the forecast period.
Enzyme replacement therapy, segment is expected to grow at the fastest CAGR during the forecast period (2021-2028)
The standard of care for disease management is enzyme replacement therapy. Although, both the enzyme replacement therapy, Sanofi's Fabrazyme and Shire's Replagal, have been approved in Europe, only Fabrazyme has been approved in the U.S. The focus of current clinical trials is to improve safety and efficacy profile of enzyme replacement therapy and introduction of novel oral therapies that can eliminate the need for intravenous infusions. Recently Amicus Therapeutics' Galafold has been approved as the first oral chaperone therapy for the treatment of adult patients in the U.S., Canada, EU, Japan, Australia, Israel, and South Korea.
Furthermore, Enzyme Replacement Therapy is an infusion (injection) of an enzyme designed to substitute for the missing ARSA enzyme that causes metachromatic leukodystrophy. ERT has been successfully used for fabry disease treatment. These factors are driving the growth of the market in the forecast period.
Takeda Pharmaceutical Company is developing the MLD ERT therapy, which they refer to as SHP-611. The initial development and clinical trial work were performed by Shire PLC, which was acquired by Takeda in early 2019. SHP-611 is based on several decades of Shire ERT research, expertise, and commercialization for other lysosomal disease products.
Registration - MLD ERT is still in Phase I/II clinical trial and has not been submitted for any agency review or registrations. As of Q3'2019, Takeda is forecasting regulatory approval and commercialization in 2023 based on the current clinical trial.
2012 Clinical Trial - A phase I/II dose-escalation study of 24 patients receiving HGT-1110 (Shire's internal product reference code) with infusions every other week started in February 2012 and completed in January 2017. The trial analysis provided guidance to increase the dose frequency and amount for the current trial.
Orphan Designation - SHP-611 has been granted Orphan Designation by the EMA for Europe (EU/3/10/813)
Phase II ERT Trial Recruiting Status and Sites
Takeda opened recruiting for their Phase II Clinical Trial for SHP611, an ERT therapy for MLD in April 2019. Their marketing name for the study is the EMBOLDEN Study. The trial will include pre-symptomatic, and for the first time, symptomatic late infantiles (and their younger pre-symptomatic siblings). For participation with symptomatic children.
They have announced 24 sites, including for the first time, 10 sites in the US. The sites will open one by one throughout 2019 and 2020.
infections which leads to a condition called immune reconstitution inflammatory syndrome (IRIS).
North America region holds the largest market share global Fabry Disease Treatment Market
North America region is dominating the global Fabry Disease Treatment Market accounted for the largest market share in 2020. Higher adoption of novel therapies, better healthcare facilities, and favorable reimbursement policies are playing a vital role in the growth of the market in the region. Coverage of expensive medications such as Fabrazyme by health insurance programs and favorable governmental healthcare policies are also prompting drug companies to increase R&D investments in the field of rare diseases.
Fabry Disease Treatment Market Competitive Landscape
The Fabry Disease Treatment Market is moderately competitive with presence of local as well as global companies. Some of the key players which are contributing to the growth of the market include Amgen Inc., Amicus Therapeutics, Inc., Bristol-Myers Squibb Company, GlaxoSmithKline, iBio, Inc., Neuraltus Pharmaceuticals, Inc., Novartis AG, Pfizer Inc., AVROBIO, Inc., Idorsia Pharmaceuticals Ltd, Protalix, Sanofi, Shire, Takeda Pharmaceutical Company Limited, Teva Pharmaceutical Industries Ltd among others. The major players are adopting several growth strategies such as product launches, acquisitions, and collaborations, which are contributing to the growth of the Fabry Disease Treatment Market globally.
Overview: Sanofi is a healthcare company, focused on patient needs and engaged in the research, development, manufacture and marketing of therapeutic solutions. Its segments are Pharmaceuticals, Human Vaccines (Vaccines), and Other. The Pharmaceuticals segment comprises the commercial operations of various franchises, including Specialty Care (Rare Diseases, Multiple Sclerosis, and Oncology), Diabetes and Cardiovascular, Established Prescription Products, Consumer Healthcare and Generics, and research and development, production and marketing activities for all of the Company's pharmaceuticals operations.
Product Portfolio: The Company's portfolio comprised of drugs, generic medicines, food supplement, and cosmetics.
The global fabry disease treatment market report would provide an access to an approx. 69 market data table, 66 figures and 300 pages.
Global Fabry Disease Treatment Market - By Drugs
Agalsidase Beta (Fabrazyme/Replagal)
Global Fabry Disease Treatment Market - By Treatment
Global Fabry Disease Treatment Market - By Route of Administration
Global Fabry Disease Treatment Market - By Distribution Channel
Global Fabry Disease Treatment Market - By Region
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