Sanofi Wins U.S. Approval for Pill to Treat Rare Gaucher Disease

Aug 19, 2014 4:45 pm ET

Aug. 19 (Bloomberg) -- Sanofi won U.S. approval for a pill to treat Gaucher disease, an alternative to the company’s 20- year-old intravenous infusion Cerezyme.

The Food and Drug Administration cleared Cerdelga for adults with Gaucher, a rare, inherited metabolic disorder that can cause fatigue and fragile bones. Sanofi’s existing drug, Cerezyme, replaces an enzyme missing in Gaucher patients while Cerdelga decreases the waste produced by cells that the missing enzyme is supposed to break down.

“Today’s approval offers another important treatment option for patients with Type 1 Gaucher disease,” Amy G. Egan, deputy director of the Office of Drug Evaluation III in FDA’s Center for Drug Evaluation and Research, said in a statement. “In addition, Cerdelga received orphan drug designation from the FDA, reflecting the agency’s focus and commitment to the development of treatments for rare diseases.”

It’s unclear how many Gaucher patients on intravenous drugs will make the switch to Cerdelga, a twice-daily pill, David Meeker, president and chief executive officer of Sanofi’s Genzyme unit, said in an interview before the agency’s approval. Paris-based Sanofi plans to price the drug, known chemically as eliglustat, at about $300,000 a year, which the company calculates is the average annual cost of infusions of Cerezyme once every two weeks. Cerezyme generated $914 million in sales last year, according to data compiled by Bloomberg.

“Our goal is to price this in a way that does not bias the medical decision-making process,” Meeker said.

Expense Questioned

The $84,000 price tag on Gilead Sciences Inc.’s hepatitis C pill Sovaldi has raised the ire of health insurers and U.S. lawmakers who claim the cost for such a widely used therapy will break budgets.

“Sovaldi is a good drug,” Meeker said. “The difference here is you have to go back to rarity and how you think about this. The number of patients we are treating here, it’s a very, very small fraction of our overall health-care spend.”

Gaucher disease affects 1 in 50,000 to 1 in 100,000 people, according to the National Institutes of Health. Sanofi estimates there are fewer than 10,000 people worldwide with the condition. The FDA said the disease affects an estimated 6,000 people in the U.S.

The major signs of the disease are liver and spleen enlargement, anemia and bone problems, the FDA said.

Cerdelga’s cost also reflects the 15 years Genzyme spent developing the drug and future efforts it will make working on a treatment for a small number of Gaucher patients, usually infants and children, whose disease effects the brain and often proves deadly, Meeker said. Cerdelga may generate $749 million in sales in 2020, according to the average of five analysts’ estimates compiled by Bloomberg.

Meeting the Cost

“The ability of Genzyme to spend 15 years developing the next generation and have the willingness, courage to go for a third generation that might treat a smaller brain population depends on a business model that works,” Meeker said.

Other Gaucher treatments include Pfizer Inc.’s Elelyso and Shire Plc’s Vpriv, both intravenous infusions. Actelion Ltd. also makes a Gaucher pill Zavesca that can only be used if patients can’t use the enzyme replacement infusions.

Patients who metabolize too fast or slow can’t use Cerdelga, Meeker said.

Genzyme hypothesized early that Cerdelga wouldn’t work as well as Cerezyme and instead “the results in many ways were quite comparable to the enzyme replacement therapy,” he said.

The company plans studies that compare the drugs’ effectiveness.

“There’s some evidence this treatment may be better,” Meeker said.