Isis' Gene Silencing Drug Approved by FDA

By Admin
The promise of antisense technology, a gene silencing technique aimed at treating genetic disorders, may finally be coming to fruition. After two decad...

The promise of antisense technology, a gene silencing technique aimed at treating genetic disorders, may finally be coming to fruition. After two decades of research and a number of false hopes for commercially viable drugs, the U.S. Food and Drug administration announced Tuesday the approval of Kynamro, marketed to treat an inherited cholesterol disorder.

The Isis Pharmaceuticals’ drug has been shown to effectively treat homozygous familial hypercholesterolemia (HoFH), affecting roughly one in a million people in the U.S. The inherited disease renders sufferers unable to remove LDL-C, or “bad” cholesterol, from the blood, ultimately resulting in increased risk of heart attack and death before patients see their 30th birthday.

“Kynamro, an injection given once a week, works with other lipid-lowering medications and diet to impair the creation of the lipid particles that ultimately give rise to LDL-C,” Eric Colman, M.D., deputy director of the Division of Metabolism and Endocrinology Products at the FDA’s Center for Drug Evaluation and Research, said in a released statement.

Antisense Therapy and the Creation of the SOD1 Protein

The Carlsbad, California-based pharmaceuticals company has poured nearly $2 billion into researching antisense technology since its founding in 1989. Founder and chief executive Stanley T. Crooke said in an interview Tuesday, “I told people it would be at least 20 years and $2 billion before we knew if the technology would work. We think it’s a seminal day for the technology and the company.”

Researchers active in the pursuit of antisense technology are hesitant to believe the drug will find widespread use, saying it’s still too early to tell. Though antisense drugs should theoretically be able to easily shut down any number of targeted genes, in reality, difficulties have arisen regarding sufficient potency and lack of toxicity making it to cells.

Currently, Isis is working on developing similar drugs to treat spinal muscular atrophy, lower triglycerides and reduce scarring from operations.

Sanofi’s Genzyme division will market the drug, with experts estimating treatment at $235,000-$295,000 a year.

Share

Featured Articles

UPS Healthcare Addressing Connected Logistics Needs

UPS Healthcare is meeting increased need for integrated, globally connected healthcare logistics services in Republic of Ireland, where pharma is booming

HPV Vaccine 'Protects Males from Cancers' - Global Report

As a new report shows the human papillomavirus vaccine can also protect men and boys, we take a look at HPV vaccines, and their impact on world health

Tata Consultancy Report Shows AI in Healthcare on Rise

Tata Consultancy Services AI for Business Global Study shows that three-quarters of healthcare leaders are deploying AI to drive innovation and efficiency

McKinsey: Consumers Demand Data-driven Wellness Products

Medical Devices & Pharma

SpaceX Starlink Launch Boost for Indonesia Healthcare

Technology & AI

J&J Targets Supplier Sustainability to Cut Healthcare GHGs

Sustainability