Enzyme Replacement Therapy (ERT) is a medical treatment that involves the administration of enzymes to replace the missing or deficient enzyme in the body. ERT is used to treat a range of inherited metabolic disorders, such as Gaucher disease, Fabry disease, and Pompe disease. These diseases are caused by mutations in genes that result in the deficiency of specific enzymes required for the proper functioning of cells and organs.
The global Enzyme Replacement Therapy market is expected to grow significantly over the next few years. The enzyme replacement therapy market was estimated at US$ 7.93 billion in 2021 and is expected to grow at a CAGR of 8.28% during 2022-2028 to reach US$ ~13.9 billion in 2028. The increasing prevalence of rare genetic diseases and the growing awareness among healthcare professionals and patients about ERT are the major factors driving the market growth.
Gaucher disease is the most common lysosomal storage disorder, and it is estimated that one in 50,000 to 100,000 people worldwide are affected by the disease. The increasing incidence of Gaucher disease and other rare genetic diseases is driving the demand for ERT. Moreover, the development of novel enzyme replacement therapies for the treatment of rare diseases is expected to drive the market growth.
In recent years, there has been a significant increase in the number of approved ERT products for the treatment of various rare genetic diseases. For instance, in 2020, the US Food and Drug Administration (FDA) approved Evrysdi (risdiplam) for the treatment of spinal muscular atrophy (SMA), a rare genetic disease that affects the muscles used for breathing, eating, and moving. Similarly, in 2021, the FDA approved Rezurock (belumosudil) for the treatment of chronic graft-versus-host disease (GVHD), a rare and life-threatening condition that can occur after a stem cell or bone marrow transplant.
The increasing number of product approvals for rare genetic diseases is expected to boost the market growth in the coming years. However, the high cost of ERT products is a major challenge faced by the market. The cost of ERT products can range from USD 100,000 to USD 1 million per patient per year, depending on the disease and the product. The high cost of ERT products can limit their accessibility, particularly in developing countries where the healthcare infrastructure is not well-developed.
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Moreover, the availability of alternative treatment options for some rare diseases, such as gene therapy and stem cell transplantation, can also affect the market growth. Gene therapy involves the introduction of a functional gene to replace the defective gene, while stem cell transplantation involves the transplantation of healthy stem cells to replace the defective ones. These treatment options are still in the early stages of development and are not yet widely available.
In conclusion, the Enzyme Replacement Therapy market is expected to grow significantly in the coming years, driven by the increasing prevalence of rare genetic diseases and the development of novel ERT products. However, the high cost of ERT products and the availability of alternative treatment options can affect the market growth. The market players need to focus on developing cost-effective ERT products and expanding their reach in developing countries to ensure the accessibility of these life-saving treatments to patients worldwide.