Lysosomal storage diseases (LSDs) are a group of rare inherited metabolic disorders that result from defects in lysosomal function. The absence of lysosomal enzymes such as hydrolase responsible for breakdown of specific substrate, its activator or a transporter, result in accumulation of substrates, leading to LSDs. Examples of LSDs include the mucopolysaccharidoses, mucolipidoses, oligosaccharidoses, Pompe disease, gaucher disease, Fabry disease, the Niemann-Pick disorders, and neuronal ceroid lipofuscinoses.
The global lysosomal storage diseases therapeutics market is estimated to account for US$ 7,217.9 Mn in terms of value in 2019 and is expected to reach US$ 15,734.5 Mn by the end of 2027.
Global Lysosomal Storage Diseases Therapeutics Market: Drivers
Significant prevalence of LSDs is expected to boost growth of the global lysosomal storage diseases therapeutics market over the forecast period. For instance, according to a report updated by National Center for Biotechnology Information, in January 2020, the prevalence of Fabry disease in white, male populations was 1:17,000 to 1:117,000. Moreover, classic Fabry disease mutations were evident in around 1:22,000 to 1:40,000 males and atypical presentations were associated with about 1:1000 to 1:3000 males and 1:6000 to 1:40,000 females.
Moreover, increasing awareness regarding rare diseases is also expected to aid in growth of the market. For instance, the European Organization for Rare Diseases celebrates Rare Disease Day annually on the last day of the month of February.
North America region held dominant position in the global lysosomal storage diseases therapeutics market in 2018, accounting for 32.4% share in terms of value, followed by Europe.
Global Lysosomal Storage Diseases Therapeutics Market: Restraints
Geographic dispersal of LSDs hampers efforts to understand the disease, given that certain rare diseases are even rarer in some regions. For instance, birth prevalence of LDSs differ considerably between various countries and population groups—Ashkenazi Jewish Ancestry have high prevalence of several genetic diseases, including some LSDs. Also, there is unusually high prevalence of Mucopolysaccharidosis (MPS) VI, GM1 gangliosidosis and fucosidosis in UAE, owing to ethnic isolation.
Moreover, high cost of enzyme replacement therapy for the treatment of LDSs is also expected to hamper growth of the market.
Global Lysosomal Storage Diseases Therapeutics Market: Opportunities
R&D in LSDs is expected to offer lucrative growth opportunities for players in the global lysosomal storage diseases therapeutics market. For instance, in February 2020, researchers from BioStrategies LC, State University (U.S.), reported that biodistribution studies in LSD models provide evidence for the theory that the RTB-lectin transports corrective doses of enzymes across the blood–brain barrier for the treatment of central nervous system pathologies.
Moreover, use of advanced technologies in the treatment of LSDs is also expected to aid in growth of the market. For instance, in November 2019, researchers from Nacional Health Institute Doctor Ricardo Jorge, Portugal, reported that CRISPR-Cas9 has great potential for successful disease modelling of sphingolipidoses, an important group of rare diseases among LSDs.
Enzyme replacement therapy segment in the global lysosomal storage diseases therapeutics market was valued at US$ 6,856.2 Mn in 2019 and is expected to reach US$ 14,938.7 Mn by 2027 at a CAGR of 10.0% during the forecast period.
Market Trends/Key Takeaways
The cost of therapies for the treatment of LSDs is high. For instance, Elaprase was regarded as one of the most expensive drugs by Forbes Index, which costs US$ 375,000 per year whereas Naglazyme costs US$ 365,000/patient/year.
In the U.S., Medicare and Medicaid programs provide good extent of reimbursement for LSDs. Around 84% of prescription drug plans covered under Medicare part D and part B provide reimbursement for orphan drugs for FDA approved indication. For instance, cost of Fabrazyme therapy reimbursed under Medicare part B is 80% of the allowable amount for Fabrazyme, for beneficiaries who administer Fabrazyme at a physician’s office or as hospital outpatient.
Global Lysosomal Storage Diseases Therapeutics Market: Competitive Landscape
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., and Amicus Therapeutics, Inc.
Global Lysosomal Storage Diseases Therapeutics Market: Key Developments
Major players in the market are focused on adopting M&A strategies to expand their product portfolio. For instance, in January 2020, Quest Diagnostics acquired Blueprint Genetics to expand its product portfolio in genetic and rare diseases.
Major players in the market are also focused on adopting collaboration strategies to expand their portfolio. For instance, in 2019, Amicus Therapeutics, Inc. collaborated with the University of Pennsylvania, under which the company received disease-specific worldwide rights to the university’s Next Generation Gene Therapy Technologies from the Wilson Lab for LSDs and other twelve rare diseases.
The deficiency of enzymes that causes a specific disease condition called lysosomal storage disease includes diseases such as Fabry Disease, Gaucher disease, lysosomal acid lipase deficiency, mucopolysaccharidosis, Hunter syndrome, and Pompe disease. Enzyme replacement therapy involves intravenous administration of enzymes in order to correct the deficiency of enzymes. Enzyme replacement is associated with fewer side effects than other treatment methods. Enzymes are obtained from sources such as human cells, animal cells, and recombinant DNA technology. Enzyme replacement, although does not provide permanent cure, helps prevents permanent damage to the body caused due to deficiency of a specific enzyme. Treatment consists of weekly or monthly doses depending upon the disease.
Restraints of the Global Lysosomal Storage Diseases Therapeutics Market
Factors such as high cost associated with the enzyme replacement therapy and complex reimbursement scenario causing therapy to be excluded from coverage by insurers due to this being rare diseases, hamper growth of the market over the forecast period.
Key features of the study:
“*” marked represents similar segmentation in other categories in the respective section.