Isis and Sanofi's Drug OK'd to Treat Inherited Cholesterol Disease

by Brett Chase, Minyanville staff writer

Isis Pharmaceuticals ISIS and Sanofi SNY won US approval to sell the drug Kynamro for a rare inherited condition that raises bad cholesterol and can cause heart disease in young people.

Sanofi’s Genzyme will sell the drug under a licensing agreement. Isis will receive a $25 million milestone payment for getting market clearance from the US Food and Drug Administration.

Shares of Isis rose 8% to $14.49 in pre-market trading Wednesday morning. The shares have climbed more than 50% in the past three months. Paris-based Sanofi’s US shares rose 1% to $49.35. That stock is up 11% in the past three months and jumped by almost a third over the past 12 months.

The drug’s label will carry stern warnings about risks, including the chance of liver damage. The FDA is requiring four post-marketing studies of the drug.

Kynamro’s safety issues were highlighted at a government panel review in October where the companies tried to win over expert advisers to the FDA on the benefits of the drug. The panel of government advisers split on Kynamro, voting 9-6 that the medicine should be approved for use in the US. Those split panels don’t usually bode well for a drug’s prospects for being approved.

A rival company, Aegerion Pharmaceuticals AEGR, also just received approval last month from the FDA to sell its drug Juxtapid. Both Kynamro and Juxtapid are designed to treat the condition homozygous familial hypercholesterolemia. (See: Aegerion Pharmaceuticals Wins Approval for Cholesterol Drug.)

Aegerion CEO Marc Beer said last month that his company’s drug would initially be aimed about 3,000 patients in the US and would be priced between $200,000 and $300,000 per patient a year.

FDA staff reviewers warned of safety issues with the Isis-Sanofi drug prior to the FDA advisory meeting in October. A report by the agency’s staff said concerns include possible tumor growth. A European panel that recommends drug approvals declined to back Kynamro in December due to safety issues.

Homozygous familial hypercholesterolemia is a condition marked by very high levels of bad cholesterol. People with this genetic disorder can suffer heart attacks at a young age. The genetic defect leaves a person’s body unable to remove low density lipoprotein, or LDL, from the blood. The most common treatment for patients with this disease have been cholesterol-lowering statins such as Pfizer’s PFE Lipitor, Merck’s MRK Zocor, and generic equivalents.

Isis is holding a conference call at 9:30 ET Wednesday to talk about the approval. See the company’s press release for Kynamro here.

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