Kynamro mipomersen regulatory update
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 Sanofi unit 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. Genzyme has exclusive 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. Additionally, 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. ...