Historical FDA approval to treatment “silencing” genes causing specific diseases

Yesterday was a historical day for medicine:  indeed, the FDA approved Alnylam Pharmaceuticals’ Onpattro (patisiran), a revolutionary treatment made up of a lipid complex that targets RNA selecting the genes responsible for polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis–a rare, highly disabling disease affecting at least 50,000 people globally. This is the first approved therapy with a revolutionary mode of action named RNA-i, based on the research conducted by scientists Andrew Fire and Craig Melo, for which the two scholars were awarded the 2006 Nobel Prize in Medicine.

Alnylam has already announced that the new therapy will be sold at $450,000 for a one-year treatment; it has potential to generate $380m as early as 2020 and to achieve a maximum of $1bn pa, experts have said.  Onpattro promises to become the first in a new class of treatments acting as RNA-interference (RNA-i) drugs. To mention the most significant programs, the same Alnylan is also developing Givoriran for the treatment of acute liver porphyrias; Amgen is developing AMG 890 for cardiovascular diseases and biotech Arbutus Biopharma is developing ARB 1467 for hepatitis C. However, it should be noted that some large drugmakers were initially eager to invest on the RNA-i technology; in fact, they soon withdrew from the business after some negative results. The most surprising cases are Roche and Merck & Co.

Alnylam was founded in Cambridge, Massachusetts, in 2002. It has already failed two attempts at marketing the RNA-i technology: specifically, an investigational therapy failed a clinical trial in 2012 and the company was forced to discontinue a  therapy development program in 2016 as the treatment had caused the death of some patients. Alnylam ’s shares rose over 3% as the approval was announced.

(Sources: WSJ, Alnylam)