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Gaucher Disease And Japan: Approval Of Genzyme’s Cerdelga

April 8, 2015


Genzyme announces March 30th that Japan’s Ministry of Health, Labor and Welfare (MHLW) grants marketing approval for Cerdelga (Eliglustat), the only oral therapy for the treatment of adults with Gaucher Disease (GD) Type 1 in Japan. Cerdelga has been approved by the following international regulatory agencies:

•   FDA in August 2014 (also has orphan drug designation)

•   European Commission (EC) in January 2015 (also has orphan drug designation)

•   Australian Therapeutics Goods Administration (TGA) in February 2015 (also has orphan drug designation).

GD is a LSD caused by a defect in a particular enzyme which breaks down fatty substance, or lipid, called glucocerebroside. As a result, glucocerebroside accumulates in cells and organs in the body, which prevents them from working correctly. There are three subtypes of GD:

•   Type 1 is the most common form of GD with swelling, enlargement, and dysfunction of the spleen and liver; also, lung and bone problems

•   Type 2 causes brain damage and death before two years of age

•   Type 3 is progressive and affects the brain, with patients living often into their early teens and adulthood.

Genzyme has been researching an oral therapy for GF for 15 years.  “The Cerdelga clinical development program is the largest ever conducted in Gaucher disease, with approximately 400 patients treated in 29 countries.” Ceredelga will help to expand and protect Genzyme’s GD franchise and will also increase the company’s market share further. Pharmaceutical companies often develop oral therapies to complement or replace their injectable therapies, as a way to protect their franchise.

The current Standard of Care (SOC) for GD, is Genzyme’s orphan drug Cerezyme, an Enzyme Replacement Therapy (ERT) requiring regular intravenous infusions for life. Genzyme pioneered ERT for GD with Cerezyme, which has enjoyed a period of market monopoly. Competition in the form of two new ERTs, Shire’s Vpriv and Pfizer/Protalix BioTherapeutics’ Elelyso, start to appear in 2010 as the new players in the GD marketplace. The GD ERT market is crowded with only a patient population worldwide of less than 10,000.

It is important to note the difference between Cerdelga and the ERTs. Cerdelga is a specific ceramide analogue inhibitor of glucosylceramide synthase, which inhibits the accumulation of fatty deposits in cells. An ERT like Cerezyme, breaks down the fatty deposits that build up in cells.

Please Note: “First Pigment Analysis (5893349712)” by NASA ICE (First Pigment Analysis Uploaded by russavia) [CC-BY-2.0] | Wikimedia Commons.

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