Genzyme, a Sanofi company, has submitted a new drug application to the FDA seeking approval for KYNAMRO for the treatment of patients with homozygous familial hypercholesterolemia (HoFH).
The submission is based on trial in which significant reductions were observed in all atherogenic lipoproteins evaluated for patients receiving KYNAMRO who are already receiving a regimen of maximally tolerated lipid-lowering therapies including statins.
Isis will receive a $25m milestone payment from Genzyme following FDA acceptance of the NDA submission.
Once approved, mipomersen would be marketed under the brand name KYNAMRO, the name that has been submitted to health authorities for the investigational agent.
Genzyme cardiovascular business vice president and general manager Paula Soteropoulos said the submission marks the second of two key milestones for the KYNAMRO program including the submission of the EU marketing authorization application last year.
"These are important steps toward our goal of bringing an innovative solution to patients in great need of a novel, targeted therapy," Soteropoulos added.