fda gives thumbs up to antisense !! , page-17

30 May 2012
Antisense Technology Update
? ANP technology partner, Isis Pharmaceuticals Ltd. announces that the U.S. Food and Drug
Administration (FDA) has accepted for filing the New Drug Application (NDA) for KYNAMRO™
(mipomersen sodium) for the treatment of patients with homozygous familial
hypercholesterolemia (HoFH)
Antisense Therapeutics Limited’s (ASX:ANP) technology partner and major shareholder, Isis
Pharmaceuticals, Inc. (NASDAQ:ISIS) has made an announcement to the market in the US (last night
Australian time) concerning their 2nd generation antisense drug mipomersen (KYNAMROTM) -
“Genzyme and Isis Announce Filing of U.S. NDA for KYNAMRO™ (mipomersen sodium) in
Homozygous Familial Hypercholesterolemia.” Genzyme submitted an application for U.S.
marketing approval of KYNAMRO™ for the treatment of patients with HoFH in March 2012. The
application will be subject to a standard review and will have a Prescription Drug User Fee Act
(PDUFA) date of January 29, 2013 (the time by which the FDA is to complete its review).
Progress in the regulatory approval process for KYNAMROTM is a further validation milestone for the
antisense technology platform. ANP’s three drugs in its development pipeline: ATL1101 for Prostate
Cancer, ATL1102 for Multiple Sclerosis, Stem Cell Mobilisation and Asthma and ATL1103 for Growth
(Acromegaly), Cancer and Diabetes Related Disorders were all accessed by ANP via its collaboration
with Isis. These compounds, like KYNAMRO™, are all 2nd generation antisense drugs.
In July 2011, Genzyme submitted an application for EU marketing approval of KYNAMROTM for the
treatment of patients with HoFH and severe heterozygous FH (Severe HeFH).
The Isis announcement follows.