Makers of Kynamro and Fanaptum to appeal negative CHMP opinions

French drug major Sanofi’s (Euronext: SAN) subsidiary Genzyme and US partner Isis Pharmaceuticals (Nasdaq: ISIS) have said they plan to request a re-examination of the negative opinion issued last Friday by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) regarding their drug Kynamro (mipomersen) for the treatment of patients with homozygous familial hypercholesterolemia (HoFH). USA-based Vanda Pharmaceuticals (Nasdaq: VNDA) also received notification that the CHMP does not recommend the firm’s Fanaptum (oral iloperidone tablets), which it too plans to appeal.

In the case of Kynamro, the CHMP was concerned about the medicine’s safety. The Committee noted that a high proportion of patients stopped taking the medicine within two years, even in the restricted group of patients with homozygous familial hypercholesterolemia, mainly due to side effects such as flu-like symptoms, injections site reactions and liver toxicity. This was considered important because Kynamro is intended for long-term treatment in order to maintain the cholesterol-lowering effect. The CHMP was also concerned by liver test results in patients taking Kynamro showing a build-up of fat in the liver and increased enzyme levels, and was not convinced that the company had proposed sufficient measures to prevent the risk of irreversible liver damage. Moreover, the Committee said it was concerned that a greater proportion of patients taking Kynamro experienced serious cardiovascular events (problems with the heart and blood vessels) than patients taking placebo.

"We are disappointed by the Committee's recommendation. Patients with HoFH carry extreme, ongoing cardiovascular risk with significantly elevated LDL-C levels despite use of currently available therapies,” said David Meeker, president and chief executive of Genzyme, adding: “This is a rare disease patient population, with a life-threatening condition, in need of new therapies. 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 to HoFH patients in Europe."