FDA approves patisiran

First RNAi Medication is based on Max Planck Technology

August 23, 2018

The United States Food and Drug Administration (FDA) approved the first-of-its-kind RNA interference (RNAi) therapeutic patisiran (Onpattro) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. Onpattro is the first and only FDA-approved treatment for this indication. The therapy is, among others, based on patented research findings from the Max Planck Society, which have exclusively been licensed to Alnylam by the technology transfer organization Max Planck Innovation. Patisiran was shown to improve polyneuropathy with significant benefit on the neurological components of the disease in a majority of patients and to improve a composite quality of life measure, reduce autonomic symptoms, and improve activities of daily living.

Hereditary transthyretin (TTR)-mediated amyloidosis (hATTR) is an inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene. TTR protein is primarily produced in the liver and is normally a carrier of vitamin A. Mutations in the TTR gene cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as the peripheral nerves and heart, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations. The median survival is 4.7 years following diagnosis. Until now, people living with hATTR amyloidosis in the U.S. had no FDA-approved treatment options.

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. Thomas Tuschl and his colleagues at the Max Planck Institute for Biophysical Chemistry in Göttingen succeeded in demonstrating that this mechanism also functions in mammals and, therefore, in humans.

Alnylam is the exclusive licensee of the therapeutic and diagnostic application of Max Planck Society´s groundbreaking patents, which are a central component of Alnylam’s technology portfolio for the development, manufacture, and marketing of RNAi therapeutics. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach in developing medicines to improve the care of patients with genetic and other diseases.

Halting the disease

The FDA approval of Onpattro was based on positive results from the randomized, double-blind, placebo-controlled, global Phase 3 APOLLO study, the largest-ever study in hATTR amyloidosis patients with polyneuropathy. In APOLLO, the safety and efficacy of the drug were evaluated in a diverse, global population of hATTR amyloidosis patients in 19 countries, with a total of 39 TTR mutations. “FDA approval represents an entirely new approach to treating patients with polyneuropathy in hATTR amyloidosis and shows promise as a new era in patient care,” said John Berk, M.D. from Boston University School of Medicine and assistant director of the Amyloidosis Center at Boston University School of Medicine. “Given the strength of the APOLLO data, including data showing the possibility of halting or improving disease progression in many patients, the therapeutic holds tremendous promise for people living with this disease.”

“It is very gratifying to see that Max Planck spin-off company Alnylam succeded in bringing the first-ever RNAi-based drug to the market. Being the first FDA approved drug against the severe disease hATTR amyloidosis Alnylam´s new product ONPATTRO has the potential to bring relief to many patients in the US and, as the company files for approval of patisiran by other Regulatory agencies, hopefully across the world. Having a strong therapeutic pipeline with several RNAi  candidates in advanced stages we are looking forward to see Alnylam bring more RNAi-based drugs on the market soon,” says Jörn Erselius, CEO of Max Planck Innovation, the technology transfer organization of the Max Planck Society.

Other Interesting Articles

Go to Editor View