Sanofi and Isis Pharmaceuticals Inc. failed to win European Union regulatory backing for a drug to treat patients with a rare disorder that causes run-away levels of the fat-like substance cholesterol.
The medicine, Kynamro, shouldn’t be allowed on the market because it leads to flu-like symptoms and liver damage that deter patients from sticking with treatment, the European Medicines Agency said in a statement today. The European Commission, the 27-nation EU’s executive arm, usually adopts the advice from the agency’s Committee for Medicinal Products for Human Use. The companies will request a re-examination of the opinion, Paris-based Sanofi said in a statement.
“We are disappointed,” David Meeker, chief executive officer of Sanofi’s Genzyme unit, said in the statement. “We will work closely with the CHMP during the re-examination process to address the committee’s concerns, with the goal of making this important medication available.”
Sanofi, France’s biggest drugmaker, gained access to the product in April 2011 when it completed the $20.1 billion purchase of U.S. biotechnology company Genzyme Corp. The French company needs new treatments to replenish its drug pipeline and make up for sales declines caused by generic competition to some of its biggest products, such as the Plavix blood thinner. The treatment, also known as mipomersen, in October won the backing of U.S. regulatory advisers.
Sanofi rose 0.1 percent to 71.80 euros at the 5:30 p.m. close in Paris. Carlsbad, California-based Isis shares advanced 2.4 percent to $9.84 in U.S. trading after falling as much as 6.9 percent earlier in the session.
“It’s only a half surprise,” Philippe Lanone, an analyst at Natixis in Paris, said by telephone. Among the drugs in Sanofi’s pipeline, Kynamro “was the one for which approval was most at risk, given its side-effect profile.”
Sanofi and Isis have been seeking clearance for mipomersen to treat patients with an inherited condition known as homozygous familial hypercholesterolemia, or HoFH, who don’t respond to standard drugs such as Pfizer Inc.’s Lipitor.
“A high proportion of patients stopped taking the medicine within two years,” mainly because of side effects including flu-like symptoms and liver toxicity, the CHMP said today. The committee “was not convinced that the company had proposed sufficient measures to prevent the risk of irreversible liver damage.”
The agency also said patients taking Kynamro experienced “serious” problems with the heart and blood vessels.
“This prevented the CHMP from concluding that Kynamro’s intended cardiovascular benefit, in terms of reducing cholesterol levels, outweighed its cardiovascular risk,” the EMA said in the statement.
HoFH is caused by a gene mutation that keeps the body from clearing a form of cholesterol known as LDL from the bloodstream. People with the disorder have high cholesterol levels from birth, and suffer from heart attacks at an early age. The disease affects about 300 people in the U.S., said Eric Le Berrigaud, an analyst at Bryan Garnier & Co. in Paris.
The U.S. Food and Drug Administration is scheduled to make a decision on Kynamro by the end of January. Advisers to the FDA voted 9-6 in favor of the treatment on Oct. 18 at a meeting in Silver Spring, Maryland, saying Sanofi and Isis provided sufficient data on the medicine’s safety and efficacy. The FDA isn’t obliged to follow the panel’s recommendation.
High levels of LDL or “bad” cholesterol are a source of blockages in the arteries, according to the National Institutes of Health.
Genzyme bought a stake in Isis as part of a deal to license Kynamro in 2008.