Fabry Disease Treatment Market - Growth, Trends, COVID-19 Impact, and Forecasts (2022 - 2027)

Fabry Disease Treatment Market - Growth, Trends, COVID-19 Impact, and Forecasts (2022 - 2027)

The Fabry Disease Treatment Market is expected to register a CAGR of 6.8% over the forecast period (2022-2027).

The COVID-19 pandemic led to seriously deleterious effects on public health systems in many countries. For instance, the healthcare industry was burdened with pandemic crisis management. Furthermore, worldwide lockdowns were imposed to curb the viral outbreak. This in turn led to a shortage of healthcare staff across the globe. Similarly, the pharmaceuticals' manufacturing activities and supply chain worldwide were also obstructed. In addition, the Fabry disease treatment market also suffered substantial losses during the COVID-19 pandemic. During the pandemic, there was a sharp decline in the outpatient services offered in hospitals and clinics. Thus, Fabry disease patients receiving treatment in the hospitals experienced delayed drug infusion or disruptions in drug infusion services provided by the healthcare staff. Hence, this leads to a decrease in demand for drugs used in Fabry disease treatment. For instance, according to a survey finding released by National Organization for Rare Disorders, Inc. (NORD) in May 2020 in the United States, 74% of patients suffering from rare diseases had a medical appointment canceled. Similarly, according to a survey conducted by the Rare Diseases Clinical Research Network (RDCRN) in the year 2020, only a few patients suffering from rare diseases were able to get an appointment with a medical provider. Thus, the decline in hospital visits during the pandemic impacted the growth of the studied market.

The factor contributing to the market's growth includes a surge in awareness associated with Fabry disease treatment. Furthermore, the rise in R&D related to Fabry disease coupled with promising pipeline products also drives the growth of the market studied.

Awareness related to Fabry disease is crucial for patients and healthcare staff. In recent years, there has been a rise in awareness related to the treatment of Fabry disease in different countries such as the United States, United Kingdom, and others. For instance, according to National Fabry Disease Foundation (NFDF), every year “April” is observed as Fabry Disease Awareness Month. Furthermore, other organizations that formally observe April as Fabry Disease Awareness Month include Fabry International Network (FIN), Fabry Australia, and MPS Society in the United Kingdom. In addition, during April, these organizations raise awareness of Fabry disease. This includes educating the public, patients, caregivers, and healthcare professionals. Furthermore, the awareness activities help to enhance the perception, diagnosis, and management of Fabry disease.

Furthermore, pharmaceutical companies also play a major role in increasing awareness related to Fabry disease. For instance, Takeda Pharmaceutical Company Limited runs a charitable access program for people with Fabry disease. In addition, this program aims to provide enzyme replacement therapy (ERT) along with other healthcare-oriented support to patients suffering from lysosomal storage disorders residing in underserved communities across the globe. Moreover, Takeda donates ERTs to nonprofit organizations. These organizations in turn distribute the ERTs to patients. Hence, the rise in awareness has resulted in early diagnosis and an increase in research related to diseases. Thus, this has resulted in a rise in demand for drugs used in the treatment of Fabry disease thereby driving the growth of the market.

In addition, the rise in research and development related to Fabry disease treatment also drives the growth of the market. Organizations across the globe are constantly funding research and development activities for Fabry disease. For instance, Horizon 2020 Framework Programme (H2020) funded a project named Smart functional GLA-nano formulation for Fabry disease (Smart-4-Fabry). The project was started in 2017 and the final workshop of the project was held recently in February 2021. The project aimed to obtain a new nanoformulation for the treatment of the disease.

Furthermore, various Fabry disease pipeline products which have the potential to receive regulatory approvals also drive the growth of the market. For instance, in March 2022 Protalix BioTherapeutics declared the results from the BRIGHT phase 3 clinical trial. This trial evaluated pegunigalsidase alfa for the potential treatment of Fabry disease. Furthermore, the results reflected that the key objectives for safety, efficacy, and pharmacokinetics were satisfied and achieved during the trial. Therefore, owing to the aforementioned factors the studied market is anticipated to witness growth over the forecast period.

However, the lack of diagnostic resources in emerging countries impedes the diagnosis rate of Fabry disease. This in turn affects the demand for drugs used for the treatment of Fabry disease.

Key Market TrendsEnzyme Replacement Therapy is Expected to Witness High Growth Over the Forecast Period

The two major types of enzyme replacement therapies that are employed in the treatment of Fabry disease include Agalsidase Beta and Agalsidase Alfa. Furthermore, upon administration, both of these mimic the actions of alpha-galactosidase A. The major reason that drives the growth of the market includes the higher demand for enzyme replacement therapies in the treatment of Fabry disease. This is because enzyme replacement therapies such as Fabrazyme work on the primary cause of Fabry disease. For instance, Fabrazyme addresses mutations in the GLA gene which causes the lack of alpha-galactosidase A enzyme. Furthermore, Agalsidase Beta is widely used in the treatment of Fabry disease. Some agalsidase beta drug brands present in the market include Fabrazyme offered by Sanofi (Genzyme Corporation), Fabagal offered by ISU ABXIS, and others. Furthermore, the factors that contribute to the growth of this segment include the wide availability of drug brands such as Fabrazyme across the globe. For instance, Fabrazyme is a standard choice in the treatment of Fabry disease as it is a proven therapy that has been in clinical use for the last 17 years. Furthermore, Farbrazyme is the only enzyme replacement therapy that is indicated for patients of age 2 years and above. Furthermore, other enzyme replacement therapies such as Replagal offered by Takeda Pharmaceutical Company Limited have been approved in the European Union countries for the last 10 years and it is presently marketed to 46 countries worldwide except the United States. Thus, these factors lead to higher sales of enzyme replacement therapies worldwide thereby driving the growth of the market.

In addition, pharmaceutical companies are adopting strategies to market new agalsidase beta drugs for the treatment of Fabry disease. For instance, in March 2022, JCR Pharmaceuticals Co., Ltd. entered into an agreement with Sumitomo Dainippon Pharma Co., Ltd. for marketing Agalsidase Beta BS I.V. Infusion in Japan for the treatment of Fabry disease. According to the agreement, the company will market and distribute the product in the second quarter of the year 2022. Hence, this led to a surge in the availability of agalsidase beta therapy in Japan thereby driving the growth of the segment.

North America is Expected to Dominate the Fabry Disease Treatment Market

North America is expected to dominate the market owing to the high usage of the brand Fabrazyme in the region. In addition, other factors that contribute to the growth of the market in the region include the availability of advanced therapeutics used in the treatment of Fabry disease. For instance, Galafold (migalastat) is the first oral medication for the treatment of adults with Fabry disease which is approved by the FDA (Food and Drug Administration) and is available in the United States. Furthermore, Galafold is indicated in patients with a confirmed diagnosis of Fabry disease with an amenable galactoside alpha gene (GLA) variant.

In addition, other factors that drive the growth of the market in the region include the wide spread of awareness related to early diagnosis of Fabry disease. For instance, organizations such as the United States National Fabry Disease Foundation (NFDF), and the United States Fabry Support & Information Group (FSIG) work with Fabry community members in each state of the United States to obtain official state governors’ proclamations. Thus, such activities lead to a surge in awareness related to Fabry disease thereby leading to a rise in demand for its treatment.

Therefore, the factors mentioned above drive the growth of the market in the North America region.

Competitive Landscape

The Fabry disease treatment market is consolidated in nature due to the presence of a few major players operating globally and regionally. The competitive landscape includes an analysis of a few international as well as local companies that operate in the market and are well known. These include Sanofi (Genzyme Corporation), Takeda Pharmaceutical Company Limited, Amicus Therapeutics, Inc, ISU ABXIS, JCR Pharmaceuticals Co., Ltd., Protalix BioTherapeutics, Avrobio Inc., BIOSIDUS, Chiesi Farmaceutici S.p.A., and Freeline Therapeutics Holdings PLC.

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