On Aug. 10, 20 years after the initial discoveries related to RNA interference were made at UMass Medical School, the U.S. Food and Drug Administration has approved the first RNAi therapeutic for patients. Patisiran (which will be sold at Onpattro), which was developed for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adults, uses the RNAi machinery first described in a 1998 paper in Nature by Craig Mello, PhD, Howard Hughes Medical Institute Investigator, the Blais University Chair in Molecular Medicine and distinguished professor of RNA therapeutics and molecular medicine, and Andrew Fire, PhD, of Stanford University.
After the Nature paper, a series of seminal advances of the science followed rapidly, including work by Phillip Zamore, PhD, Howard Hughes Medical Institute Investigator, the Gretchen Stone Cook Chair of Biomedical Sciences and chair and professor of RNA therapeutics, who in 2000 was lead author on a landmark paper in Cell that proved double-stranded RNA is chopped up into small interfering RNAs—the specific determinant for RNAi. Two years later, Dr. Zamore, along with three collaborators, formed Alnylam Pharmaceuticals to explore RNAi-based drug development.
Related story on UMassMedNow:
FDA approves first drug to use RNA interference, based on discoveries made at UMass Medical School
See is media coverage of this breakthrough below.
FDA approves first-ever RNAi-based therapy
STAT – August 10, 2018
Cambridge biotech Alnylam wins FDA approval for first drug
Boston Business Journal – August 10, 2018
Patisiran Wins Two Firsts With FDA Approval
MedPage Today – August 10, 2018
Rare-Disease Treatment From Alnylam to Cost $450,000 a Year
Bloomberg – August 10, 2018
Alnylam Prices First Gene Silencing Drug At $450,000 Per Patient, But Offers Money-Back Guarantee
Forbes – August 10, 2018
New Kind of Drug, Silencing Genes, Gets FDA Approval
Wall Street Journal – August 10, 2018
Alnylam vows value-based pricing, financial help with $450K Onpattro launch
FiercePharma – August 13, 2018
Alnylam Gets Landmark FDA OK For First-Ever RNAi Drug
FiercePharma – August 10, 2018
Alnylam Gets Landmark FDA OK For First-Ever RNAi Drug
Rare Disease Report – August 10, 2018
In first, FDA approves RNA interference drug from Alnylam
BioPharmaDive – August 10, 2018
FDA approves patisiran infusion for hereditary transthyretin-mediated amyloidosis
Healio – August 10, 2018
FDA Approves First Targeted Treatment of Polyneuropathy in Rare Disease
Pharmacy Times – August 10, 2018
FDA OKs Patisiran (Onpattro) for Polyneuropathy in hAATR
MedScape – August 10, 2018
The first gene-silencing drug wins FDA approval
Science News – August 10, 2018
Alnylam’s Onpattro is first ever FDA-approved RNAi drug
PharmaPhorum – August 13, 2018
Alnylam claims RNAi milestone with Onpattro approval
PMLive – August 13, 2018
On-time approval, on to market for Onpattro as first RNAi therapy
BioWorld – August 12, 2018
FDA approves Onpattro, first treatment for polyneuropathy caused by rare genetic disease
Healio – August 12, 2018
First ever RNA-based gene-silencing drug approved by FDA, and it's not cheap
New Atlast – August 13, 2018
FDA Approves Alnylam's Onpattro for Polyneuropathy of hATTR Amyloidosis
Genetic Engineering & Biotechnology News – August 10, 2018
