LYSOSOMAL STORAGE DISEASES THERAPEUTICS MARKET ANALYSIS

Market Challenges And Opportunities

Global Lysosomal Storage Diseases Therapeutics Market - Key Trends

On March 7, 2023, an e-learning programme was launched to help healthcare professionals better identify lysosomal storage disorders (LSDs) and help provide faster diagnosis and access to treatment. Expert nurses and health care professionals from several lysosomal disorders centres across the U.K. designed the e-Learning programme with input from the British Inherited Metabolic Disease Group (BIMDG), who are hosting the training on their website. The training is open to LSD nurses and allied health professionals through membership of BIMDG and is accredited by the Royal College of Nursing (RCN). This means that participants can earn Continuing Professional Development (CPD) points by completing the modules.

Global Lysosomal Storage Diseases Therapeutics Market – Impact of Coronavirus (COVID-19) Pandemic

Since the COVID-19 virus outbreak in December 2019, the disease spread to over 100 countries across the globe, and the World Health Organization declared it a public health emergency on January 30, 2020.

COVID-19 affected the economy in three main ways: by directly affecting the production and demand of drugs, by creating disruptions in distribution channels, and through its financial impact on firms and financial markets. Due to nationwide lockdowns, several countries such as China, India, Saudi Arabia, the U.A.E., Egypt, and others are facing problems with the transportation of drugs from one place to another.

However, the COVID-19 pandemic had a negative impact on the global lysosomal storage diseases therapeutics market. This is because of the disruption in the treatment due to COVID related restrictions. According to an article published in molecular genetics and metabolism on April 24, 2020, impact of COVID-19 related healthcare crisis on treatments for patients with lysosomal storage disorders were studied in Italy by Regional Coordinating Center for Rare Diseases, University Hospital of Udine, Italy and Institute of Hygiene and Clinical Epidemiology, Italy. 49 % of patients receiving enzyme replacement therapy in hospitals experienced disruptions, versus 6% of those home-treated. The main reasons of missed infusions were fear of infection (62.9%) and re-organization of the infusion centers (37%).

Global Lysosomal Storage Diseases Therapeutics Market - Key Players

Major players operating in the global lysosomal storage diseases therapeutics market include Shire plc, Pfizer, Inc., Sanofi, BioMarin Pharmaceutical Inc., Actelion Ltd., Raptor Pharmaceutical Corp., Protalix Biotherapeutics Inc., Quest Diagnostics, and Amicus Therapeutics, Inc.

Global Lysosomal Storage Diseases Therapeutics Market Restraint

High prices of Lysosomal Storage Diseases Therapeutics

The cost of treatment associated with Lysosomal Storage Diseases is very high. As it is an expensive method, this can restrain the growth of the global lysosomal storage diseases therapeutics market . For instance, according to an article published by Orphanet Journal of rare diseases on March 25, 2021, the enzyme replacement therapy is most effective therapy for treatment of lysosomal storage diseases. Hence research and development activities were more focused on it after initial success of ERT for Gauchers disease. Important limitation of many of the existing or pending therapeutic approaches is their expense. ERTs can cost anywhere from US$ 250– US$ 650,000/year per patient and gene therapies cost millions of dollars that are likely to be paid out over several years. These high costs are justified by the research and development costs incurred by the drug companies, the cost saving value of the treatments when compared to the medical costs of caring for these patients in the absence of these therapies and the very small commercial market for these disorders, especially when the drugs are being developed for individual diseases.

This restrain can be overcome by the introduction of newer government reimbursement policies that can help parents financially.

Geographic dispersal of lysosomal disease

The geographic dispersal of LSD hampers efforts to understand the disease, given that certain rare diseases are even rarer in some regions. For instance, according to article published by National Library of Medicine on July 25, 2022, both ethnicity and geography play a part in the incidence of LSDs. For example, Gaucher disease(GD) occurs in 1 in 40000 to 1 in 60000 in the general population. In Eastern Europen Jews (Ashkenazi Jews), GD is as high as 1 in 800, Tay-Sachs disease as high as 1 in 3900. Niemann-Pick A and mucolipidosis IV also occur with increased frequency in this population. In the Finnish population, aspartylglucosaminuria occurs at a frequency of 1 in 18500. Salla disease is also common in the Finns. Cultural and geographical genetic contribute to this high incidence.

This can be overcome by arranging awareness programs and dedicated research and development activities to understand disease and its relation with genetics.