Morquio Syndrome Treatment Market Size, Share and Global Trend By Treatment (Enzyme Replacement Therapy), By Disease Type (Morquio A, Morquio B), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, Online Pharmacies), Regional Forecast, 2024-2032

Morquio Syndrome, also known as MPS IV and mucopolysaccharidosis IV, refers to a rare inherited metabolic disorder or birth defect that mostly affects the skeleton. The FDA approval of VIMIZIM (elosulfase alpha), the first and the only specific treatment for the patients with this ultra-rare genetic condition, firmly established BioMarin’s position as a leading market player for the treatment of MPS diseases.

Apart from the recent regulatory approvals, there has been increasing activity in the R&D for the Morquio syndrome treatment as evidenced by the greater inclusion of the disorder in the clinical pipelines of the major market players. For instance, Ultragenyx Pharmaceutical Inc. has a Phase 3 Study undergoing for the Morquio syndrome, Shire has an ongoing clinical trial for the pediatric patients suffering from the syndrome and ArmaGen, Inc. also has a drug (AGT-181) in the clinical trials. This combined with the greater awareness of the disease is also driving the growth in Morquio syndrome treatment market.

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The growth in the number of products in the preliminary and advanced stages of R&D along with the focus on treatment of rare diseases is anticipated to drive the growth in the Morquio syndrome treatment market at a considerably fast rate. With the recent approval of the elosulfase alpha (VIMIZIM), the market is expected to be boosted a number of products currently in the pipelines of various companies can be anticipated to undergo regulatory approvals for the marketing, if they have favourable clinical trials results. 

The factor that is expected to limit the growth of the market is the huge price tag associated with Morquio syndrome treatment products. For example, VIMIZIM is expected to cost patients around US$ 380,000, which is estimated to be the product’s annual cost. Other factors which may limit the growth of the market is the lack of awareness in the emerging markets and the regulatory and the clinical hurdles. 

Key Players Covered 

Some of the major companies that are present in the global Morquio Syndrome Treatment Market are BioMarin, Genzyme Corporation, Shire, JCR Pharmaceuticals Co., Ltd., Sanofi, Sangamo Therapeutics, ArmaGen and GREEN CROSS CORP, Concert Pharmaceuticals Inc., Alexion Pharmaceuticals Inc., and other players. 

 SEGMENTATION 

At present, enzyme replacement therapy accounts for the largest proportion of the market and the trend is expected to remain same during the forecast period due to the approval of elosulfase alpha, the only FDA approved treatment option, is an enzyme replacement therapy. 

Key Insights 

• Prevalence of Morquio syndrome by key countries


• Advancements in research & development of treatment options


• Overview of advancements in novel therapeutics


• Pricing Analysis by key countries


• Pipeline Analysis


• Regulatory scenario by key countries


• New product launches 

Regional Analysis 

The global Morquio Syndrome Treatment market has been segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America followed by Europe are the two largest markets for Morquio Syndrome Treatment at present and they are expected to account for a large proportion of the market in the forecast period as well. The world’s first and only Morquio Syndrome Treatment, VIMIZIM, received marketing approval in the U.S. and Europe in 2014 and as of date, this is the only specific treatment for Morquio syndrome that is approved by the regulatory authorities. In the Asia Pacific region, Japan is expected to provide the largest market opportunity. This was primarily due to the early approval and adoption of VIMIZIM as a treatment option for the Morquio syndrome. 

Key Industry Developments

• In 2014, Bio Marin’s product offering of VIMIZIM (elosulfase alpha) was approved as the only treatment option for Morquio syndrome across a number of regulatory agencies in the U.S., Europe and Japan


• In 2017, Sangamo Therapeutics Inc., who have the gene therapy candidate (SB-913) in their clinical pipeline, was granted the rare pediatric disease designation from the FDA


• Another drug from Sangamo, SB-318 was also granted the orphan drug status from the U.S. FDA