On a rocky road toward hoped-for approvals, Sanofi's ($SNY) mipomersen has hit a serious rut with European regulators. A European Medicines Agency panel handed down a negative opinion late this week on the antisense drug from Isis Pharmaceuticals ($ISIS) for treating a genetic disease that causes blood cholesterol to jump, citing safety concerns such as signs of liver toxicity and cardiovascular risks in patients in clinical trials.
Mipomersen, to which Sanofi gained rights through its 2011 buyout of Genzyme, narrowly passed muster with an FDA panel in October with a REMS plan to track the safety of the therapy in patients taking it for the genetic disease, homozygous familial hypercholesterolaemia (HoFH). The EMA's Committee for Medicinal Products for Human Use (CHMP), however, "was not convinced that the company had proposed sufficient measures to prevent the risk of irreversible liver damage," the EMA stated in a document.
Sanofi's Genzyme unit, leading the regulatory charge for mipomersen, which the company hopes to market under the name Kynamro, plans to request the EMA give its application another look through a re-examination process. Yet the fortunes of the program appear to be waning with every safety red flag reviewers have raised about the drug. And Aegerion Pharmaceuticals ($AEGR) has a rival oral therapy for the same disease that has already gained wider support from the FDA panel that narrowly recommended approval of mipomersen months ago.
"We are disappointed by the Committee's recommendation. Patients with HoFH carry extreme, ongoing cardiovascular risk with significantly elevated LDL-C levels despite use of currently available therapies," said David Meeker, President and CEO, Genzyme. "This is a rare disease patient population, with a life-threatening condition, in need of new therapies. We will work closely with the CHMP during the re-examination process to address the Committee's concerns, with the goal of making this important medication available to HoFH patients in Europe."
Isis certainly has a lot riding on mipomersen too, as the company has been prolific in securing partnerships, notably with with Biogen Idec ($BIIB) and AstraZeneca ($AZN), and slow to advance its antisense therapies to the market.
The European Commission gets the final say on drug approvals and often follows the advice of the CHMP.
In related CHMP news, the committee also advised against approval of Vanda Pharmaceuticals' ($VNDA) Fanaptum for treating schizophrenia, with the CHMP raising concerns about evidence of marginal efficacy of the drug compared with placebo in studies and heart risks associated with the treatment.
- here's a link to info on the CHMP opinion on mipomersen
- see Genzyme's release
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