Isis advised on new drug's approval path

California-based Isis Pharmaceuticals has been offered guidance by the US Food and Drug Administration on the approval path for its mipomersen, being developed and commercialized with fellow US firm Genzyme.

The FDA has recommended the reduction of low-density lipoprotein cholesterol as an acceptable surrogate endpoint for the accelerated approval of the candidate for use in patients with homozygous familial hypercholesterolemia. It will require data from two ongoing preclinical studies for carcinogenicity to proceed with the filing, which is now planned to take place in 2010. A Phase III study is currently enrolling patients.

Mipomersen is a second-generation antisense drug that reduces the production of apoB-100, a protein critical to the synthesis and transport of both LDL and very low-density lipoprotein cholesterols, both of which are directly involved in the onset of heart disease.