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FDA approves Kynamro

January 30, 2013 2:27 AM UTC

FDA approved an NDA for Kynamro mipomersen from Genzyme Corp. as an addition to lipid-lowering medications and diet to treat homozygous familial hypercholesterolemia (hoFH). The unit of Sanofi (Euronext:SAN; NYSE:SNY) said it will disclose pricing when it launches the drug, but declined to provide a time frame on a launch for the second-generation antisense inhibitor of apolipoprotein B-100 (APOB-100) mRNA. The approval triggers a $25 million milestone payment from Genzyme to Isis Pharmaceuticals Inc. (NASDAQ:ISIS). Genzyme has rights to Kynamro from Isis.

Kynamro's label includes a boxed warning on the risk of hepatic toxicity. The drug also was approved with a REMS that includes prescriber and pharmacy certification and documentation of safe-use conditions, which requires a prescription authorization form for each new prescription. Genzyme also is required to develop a sensitive assay that binds double-stranded DNA and to conduct a study to assess the presence of antibodies to dsDNA in patients receiving Kynamro. The company is required to run a long-term safety study of Kynamro and a pharmacovigilance program to monitor reports of malignancy, immune-mediated reactions and hepatotoxicity. ...