ROCKVILLE, MD — (Marketwire) — 04/04/12 — MarketResearch.com has announced the addition of the new report “Neurometabolic Disorders Market to 2017 – Novel Therapies for Rare Diseases such as Gaucher-s and Niemann-Pick Poised to Supersede Existing Therapies,” to their collection of Diseases & Conditions market reports. For more information, visit
The neurometabolic disorders market value was estimated at $1.6 billion in 2010, and is expected to reach $3.3 billion by 2017, growing at a Compound Annual Growth Rate (CAGR) of 11%.
Gaucher-s disease, Fabry disease and Pompe disease account for 91.6% of the total market. The launch of new drugs in the Gaucher-s disease and Fabry disease markets, coupled with increased awareness amongst patients and physicians, is expected to increase revenues over the forecast period. As these are orphan indications (affecting fewer than 200,000 patients), government agencies and companies are taking extra measures to increase awareness among patients and physicians about the diseases and their available treatment options.
Enzyme Replacement Therapy (ERT) is currently the gold standard available therapeutic option for most neurometabolic diseases. Late stage molecules (Phase III and NDA filing) in the pipeline include Uplyso, Genz-112638 and Amigal. A New Drug Application (NDA) for Uplyso has been submitted. Amigal and Genz-112638 are in Phase III for the treatment of Fabry disease and Gaucher-s disease respectively. Four companies — Genzyme, Shire, BioMarin and Actelion — are the major players in the neurometabolic disorders market. Genzyme leads the market with a share of 64%, followed by Shire, BioMarin and Actelion.
A viral contamination at Genzyme-s Allston facility in 2009 led to the discontinuation of the manufacture of Cerezyme and Fabrazyme, encouraging rival companies to launch products and enter the Gaucher-s and Fabry disease markets. Manufacturing processes are resuming and patients are steadily switching back to ERT. Genzyme was recently acquired by Sanofi.
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