Kynamro mipomersen regulatory update

EMA's CHMP issued a negative opinion recommending against approval of an MAA for Kynamro mipomersen from Sanofi's Genzyme Corp. unit to treat homozygous familial hypercholesterolemia (hoFH). CHMP said that while Kynamro was effective at reducing LDL-C, it was concerned about the compound's safety. The committee noted that a greater proportion of patients receiving Kynamro had serious cardiovascular events than patients receiving placebo, and said that Kynamro's cardiovascular benefit does not outweigh its cardiovascular risk. Genzyme, which has exclusive rights to the compound from Isis, said it plans to request a reexamination by CHMP.

CHMP also noted a number of other safety concerns, including the high proportion of patients who stopped taking Kynamro within two years because of flu-like symptoms, injection site reactions and liver toxicity; the committee said this is a concern because Kynamro is intended for long-term treatment. CHMP also noted that liver tests showed a build-up of fat and increased enzyme levels in patients receiving Kynamro and said it was "not convinced" that the company had proposed sufficient measures to prevent the risk of irreversible liver damage. ...