The Lucrative Orphan Drugs Market Attracts Investments from Big Pharma Players

Tuesday 10 April 2012, Amsterdam

The report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by a team of industry experts. The report provides an in-depth analysis of Gaucher’s disease, Fabry disease, Pompe disease, mucopolysaccharidosis VI and Niemann-Pick type C. The report 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 also provides insights into the neurometabolic disease Research and Development (R&D) product pipeline and explores the competitive landscape including major players in neurometabolic disease market. Finally, the report also includes analysis on Mergers and Acquisitions (M&A) and licensing agreements that took place in neurometabolic disease market.


The Neurometabolic Disorders Market is Poised to Grow at a Rate of 11%; Gaucher’s, Fabry and Pompe to Account for 91.6% of the Market

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 forecastperiod. 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.

See figure: Neurometabolic Disorders Market to 2017, Global, Market Forecasts ($m), 2010-2017


Four Major Players Control the Neurometabolic Disorders Market - Genzyme is the Market Leader

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.


The Lucrative Orphan Drugs Market Attracts Investments from Big Pharma Players

Gaucher’s and Niemann-Pick are orphan diseases, with a prevalence of fewer than 200,000. The orphan disorders sector has enormous market potential.

Orphan drugs are very expensive costing, on an average, more than $250,000 annually. An orphan drug designation comes with a market exclusivity of seven years in the US and 10 years in the European Union (EU), whereby the drug is insulated from competition from similar drugs. They are also granted special benefits by regulators and insurers that make them more profitable. These benefits include exemption from filing fees (it costs nearly $1.4m for a NDA with the Food and Drug Administration (FDA)) and tax credits of approximately 50% on clinical trial costs. Thus, orphan drugs for rare diseases are an attractive market.

To capitalize on the market potential, Big Pharma players are entering the market, as shown by recent licensing agreements. GlaxoSmithKline (GSK) and Sanofi have been involved in licensing agreements and acquisition deals to either enter or strengthen their presence in the orphan neurometabolic disorders market.

While Sanofi acquired Genzyme, the market leader in the neurometabolic market, GSK and Pfizer struck licensing deals with Amicus and Protalix respectively, for drugs indicated for the treatment of Fabry and Gaucher’s disease.


Genzyme’s Competitors Stand to Gain from Fabrazyme and Cerezyme Shortage

Genzyme reported a shortage of Fabrazyme and Cerezyme in 2009 due to a viral contamination in one of its bioreactors at the Allston facility. This resulted in a significant decline in revenue in 2009 and 2010.

As a result of the viral contamination and supply shortage, physicians and patients are looking to other therapeutic options. For instance, patients with Fabry disease who were on Fabrazyme have either reduced their yearly dosage of Fabrazyme or have switched to other therapies such as Replagal (approved for Fabry disease in the EU), which also rapidly captured market share in other European countries.

Sales of VPRIV and Zavesca (other therapeutic options for the treatment of Gaucher’s disease) have also reported an increase in sales due to the shortage.

Thus, companies with promising drugs for the treatment of the aforementioned indications and competitors already marketing drugs for these indications will stand to gain if the aforementioned drugs remain in short supply. Genzyme, the market leader in this segment, will lose market share to its competitors if the shortage continues.