Genzyme Corporation's New Rare Disease Pill Will Cost Patients $310,250 A Year

September 4, 2014

By Jessica Wilson, BioSpace.com Breaking News Staff

Genzyme Corporation , the Cambridge, Massachusetts-based biopharmaceutical company, owned by French pharmaceutical firm Sanofi S.A. , has priced its recently FDA-approved drug, Cerdelga, a pill for the treatment of Gaucher disease, at $310,250 a year, according to the Boston Globe.

The FDA approved Cerdelga on August 19, 2014 after the successful conclusion of Phase III trials. Because Gaucher disease affects about 2,000 people in the U.S. (and fewer than 10,000 people worldwide), the FDA has granted it Orphan Drug status. Due to this status, Genzyme can qualify for tax credits and patent extensions for the drug.

Cerdelga, a capsule that some patients with the disease can take twice daily, could replace the more complicated Cerezyme treatment, an enzyme replacement therapy that consists of a two-hour infusion every two weeks, also developed by Genzyme. The Cerezyme treatment currently costs $300,000 annually.

“Gaucher is among the rarest of the rare diseases,” Genzyme spokeswoman Lori Gorski was quoted as saying in the Boston Globe article. “The health care system already has been taking care of people with Gaucher disease. This is not a new burden on the system. This gives patients and physicians the ability to choose the therapy that is best for their circumstances without consideration of the price.” She did acknowledge that the actual cost of producing the capsule is less than that of producing Cerezyme due to the different ways they treat the disease.

The pricing of Cerdelga, though, takes into consideration costs such as the development of the drug. Genzyme has been researching an oral therapy for the disease for the past 15 years, “from early chemistry and preclinical research through clinical development,” said the company in a press release.

The National Institute of Neurological Disorders and Stroke (NINDS), a part of the National Institutes of Health, describes Gaucher disease as, “the most common of the inherited metabolic disorder known as lipid storage diseases." NINDS explains further that, “Gaucher disease is caused by a deficiency of the enzyme glucocerebrosidase. Fatty materials can accumulate in the spleen, liver, lungs, bone marrow, and brain."

The key difference between Cerezyme, which is produced via an expensive organic process, and Cerdelga is that Cerezyme is an enzyme replacement therapy that breaks down “fatty deposits that build up in cells and cause a variety of symptoms, [while] Cerdelga inhibits the accumulation of these fatty deposits in the first place," according to Dr. Pramod Mistry, MD, PhD, Professor of Medicine and Pediatrics and the Director of National Gaucher Disease Treatment Center at Yale University School of Medicine.

Patients with Gaucher disease could end up switching to the more convenient Cerdelga from competing treatments made and marketed by the Irish company Shire PLC and the Israeli company Protalix BioTherapeutics.