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Genzyme: Power House For Orphan Drugs

September 16, 2014

This Blog Post presents Genzyme’s (Sanofi) current portfolio of marketed orphan drugs in the United States. Sanofi bought Genzyme for $20.1 billion in 2011. Genzyme posted a 25 percent jump in sales in the first half of the year, and Sanofi is hoping that Genzyme will help alleviate some of the patent losses on older drugs (such as Plavix), by growing the Multiple Sclerosis franchise of Lemtrada and Aubagio. Genzyme is currently organized into two business units:

•   Rare Diseases

•   Multiple Sclerosis.

Genzyme is best known for its treatments for Lysosomal Storage Disorders (LSDs). In 1984, the company developed the first Enzyme Replacement Therapy (ERT) for Gaucher Disease, which established the company as a leader in LSD treatments. Genzyme recently receives FDA approval for Cerdelga, “the only first-line oral treatment for Gaucher Disease Type 1, as an alternative to regular intravenous infusions of ERT ….”.

The chart below identifies Genzyme’s marketed orphan drugs for rare diseases in the United States.

Genzyme’s Marketed Orphan Drugs (U.S.) For Rare Diseases

Drug Name Indication FDA Approval Date
Aldurazyme (Laronidase) Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) April 2003
Cerdelga (Eliglustat) Adult patients with Gaucher disease type 1 who are CYP2D6 metabolizers as detected by an FDA-cleared test August 2014
Cerezyme (Imiglucerase) Gaucher disease type 1 May 1994
Fabrazyme (Agalsidase Beta) Fabry’s disease April 2003
Kynamro (Mipomersen) Homozygous familial hypercholesterolemia (HoFH) January 2013
Lumizyme/ Myozyme (Alglucosidase Alfa) Pompe Disease 2006 – August 2014
Thyrogen (Thyrotropin Alfa) Adjunctive treatment for radioiodine ablation of thyroid tissue remnants in patients who have undergone thyroidectomy for well-differentiated thyroid cancer and who do not have evidence of metastatic thyroid cancer December 2007


Looking at Genzyme’s Pipeline, an ERT is being developed to treat Niemann-Pick Type B Disease as well as other therapies for Pompe and Fabry diseases.

Please Note: “Abstract pills” by Robson# (Flickr: Pills here) [CC-BY-2.0] | Wikimedia Commons.

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