A prolonged gap in the neurology market following the shutdown of a Genzyme production facility is tempting the pharmaceutical segment to focus on degenerative brain conditions to take advantage of the lucrative nature of orphan drug designations, according to a new report by pharmaceutical experts GBI Research.
The new report* found that the space in the market left by neurometabolic disorders pharmaceuticals giant Genzyme may spark long-term medical progress in the field, as competing companies attempt to achieve market dominance.
Market leader Genzyme held a dominating 64% share in the global neurometabolic disorders market. However, a viral contamination at Genzyme’s Allston facility in 2009 led to the discontinuation of the manufacture of Cerezyme and Fabrazyme, creating shortages of these popular drugs.
Some patients with Fabry disease switched from Fabrazyme to other therapies such as Replagal, which is approved to treat Fabry disease in the EU and has rapidly captured market shares. Producers of VPRIV and Zavesca, alternative therapeutic options for Gaucher’s disease, have also reported a recent increase in sales due to the shortage of Cerezyme.
However, according to GBI Research, there is still plenty of room for other big pharma players to continue to enter the market. GlaxoSmithKline (GSK), Sanofi and Pfizer have all been involved in licensing agreements and acquisition deals in attempts to strengthen their presence in the orphan neurometabolic disorders market. Future launches of new drugs in the Gaucher’s disease and Fabry disease markets are also expected to increase revenue, with late-stage molecules (Phase III and NDA filing) such as Uplyso, Genz-112638 and Amigal in the pipeline.
Orphan drugs are very expensive, costing on average over $250,000 per year. Orphan drug designation comes with a market exclusivity of seven years in the US and 10 years in the European Union (EU), thereby preventing market competition from similar drugs. Orphan drugs are also granted exemption from filing fees and tax credits of approximately 50% on clinical trial costs, allowing companies to make higher profits. For these reasons, the development of orphan drugs for rare diseases represents a potential goldmine for pharmaceutical companies.
Companies with promising drugs for the treatment of neurometabolic disorders will therefore stand to gain if Genzyme’s products remain in short supply. 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%.
NOTES TO EDITORS
This report provides insights into neurometabolic disease therapeutics market until 2017, and provides an in-depth analysis of Gaucher’s disease, Fabry disease, Pompe disease, Mucopolysaccharidosis VI and Niemann-Pick Type C. It also examines the global neurometabolic disease treatment usage patterns, geographical distribution of neurometabolic disease and market across the US, the top five countries of Europe, and in Japan. The report provides insights into the neurometabolic disease research and development (R&D) product pipeline, and explores the competitive landscape and major players in the neurometabolic disease market. Finally, it includes analysis on mergers and acquisitions (M&A), and licensing agreements that took place in neurometabolic disease market.
The report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis conducted by GBI Research’s team of industry experts.
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