Menu

First RNAi Therapy Approved by FDA

Alnylam’s patisiran interferes with the production of a mutated protein present in people with hereditary transthyretin amyloidosis.

Aug 10, 2018
Kerry Grens

ABOVE: © ISTOCK, KOTO_FEJA

For the first time, the US Food and Drug Administration has approved a drug based on RNA interference. Patisiran (Onpattro) contains RNA strands that disrupt the production of a protein that clumps up and causes nerve damage in patients with a rare genetic disorder called hereditary transthyretin amyloidosis.

“After 16 years, tireless perseverance through near-death moments, billions of dollars in investment, we’ve finally succeeded in advancing RNAi therapeutics as a whole new class of innovative medicines,” Alnylam CEO John Maraganore says in an email to Xconomy. “What matters most? The difference we can now make in the lives of patients with hATTR amyloidosis.”

Hereditary transthyretin amyloidosis affects about 50,000 people worldwide. The disease is caused by mutations in the gene that codes for the protein transthyretin; the abnormal protein forms deposits in the nervous system. By infusing lipid nanoparticles filled with short interfering RNAs into the liver, transcripts for the aberrant protein are blocked from translation.

See “RNA Interference Drug Excels in Clinical Trials to Treat Rare Disorder

“This approval is part of a broader wave of advances that allow us to treat disease by actually targeting the root cause, enabling us to arrest or reverse a condition, rather than only being able to slow its progression or treat its symptoms,” FDA Commissioner Scott Gottlieb says in a press release.

November 2018

Intelligent Science

Wrapping our heads around human smarts

Marketplace

Sponsored Product Updates

Slice® Safety Cutters for Lab Work

Slice® Safety Cutters for Lab Work

Slice cutting tools—which feature our patent-pending safety blades—meet many lab-specific requirements. Our scalpels and craft knives are well suited for delicate work, and our utility knives are good for general use.

The Lab of the Future: Alinity Poised to Reinvent Clinical Diagnostic Testing and Help Improve Healthcare

The Lab of the Future: Alinity Poised to Reinvent Clinical Diagnostic Testing and Help Improve Healthcare

Every minute counts when waiting for accurate diagnostic test results to guide critical care decisions, making today's clinical lab more important than ever. In fact, nearly 70 percent of critical care decisions are driven by a diagnostic test.

LGC announces new, integrated, global portfolio brand, Biosearch Technologies, representing genomic tools for mission critical customer applications

LGC announces new, integrated, global portfolio brand, Biosearch Technologies, representing genomic tools for mission critical customer applications

LGC’s Genomics division announced it is transforming its branding under LGC, Biosearch Technologies, a unified portfolio brand integrating optimised genomic analysis technologies and tools to accelerate scientific outcomes.