EMA committee reiterates negative Kynamro recommendation

EMA's CHMP reiterated a negative opinion against approval of Kynamro mipomersen from the Genzyme Corp. unit of Sanofi (Euronext:SAN; NYSE:SNY) to treat homozygous familial hypercholesterolemia (hoFH). Genzyme had requested a reexamination of a December negative opinion, in which CHMP had said Kynamro's cardiovascular benefit does not outweigh its cardiovascular risk. The committee said the concerns it raised in December -- which also included liver toxicity and the rate of discontinuations for the product -- are still unresolved. FDA approved the second-generation antisense inhibitor of apolipoprotein B-100 (APOB-100) mRNA for hoFH in January. Genzyme has exclusive rights to Kynamro from Isis Pharmaceuticals Inc. (NASDAQ:ISIS), which was off $1.14 to $16.83 on Friday (see BioCentury Extra, Dec. 14, 2012).

As expected, CHMP also recommended against approval of an MAA for Defitelio defibrotide from Gentium S.p.A. (NASDAQ:GENT) to treat and prevent hepatic veno-occlusive disease (VOD) in adults and children undergoing hematopoietic stem cell transplantation therapy. CHMP said data from a prevention study did not provide "sufficiently convincing evidence" of Defitelio's benefit, including a lack of data on how Defitelio is eliminated in children and patients with reduced kidney function. The committee said there were also problems with the study's documentation and data reporting, including a low number of historical controls. Gentium said it plans to appeal the opinion and request a reexamination. ...