The Committee for Medicinal Products for Human Use (CHMP) announced a Positive Opinion recommending marketing authorization of Alnylam’s (ALNY) RNAi product Onpattro™ (patisiran). The product is designed to treat hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy.
The CHMP opinion is based on conclusive positive results from APOLLO Phase 3 study on patients with hATTR amyloidosis. The results were published in The New England Journal of Medicine (NEJM) in early July.
The European Medicines Agency reviewed patisiran under the accelerated assessment procedure usually granted for treatments that the CHMP believes represent therapeutic innovations of major impact on public health.
It is for the European Commission now to deliver its final decision after reviewing the CHMP recommendation which, will become applicable to all the 28 European member states, in addition to Iceland, Liechtenstein and Norway.
Patisiran is an RNAi compound that targets transthyretin by silencing specific messenger RNA, probably blocking the production of TTR protein before it is generated in the cells. This interference reduces the accumulation of the TTR amyloid and helps its clearance from peripheral tissues. The product is currently under priority review as a Breakthrough Therapy with the U.S. Food and Drug Administration (FDA), with an action date of August 11, 2018. Regulatory filings in other markets, including Japan, are also planned for mid-2018.
hATTR amyloidosis is an inherited, progressively debilitating and often fatal disease. The cause of the disease is mutations in the TTR gene. The TTR protein is mostly generated in the liver and normally carries vitamin A. Mutations in the TTR gene allow abnormal amyloid proteins to accumulate, which causes organs’ and other tissues’ damages, including peripheral nerves and the heart. The symptoms include severe pain and the complications include potential cardiomyopathy. haTTR affects about 50,000 people worldwide.
Median survival is 4.7 years after diagnosis, and even shorter, 3.4 years, in patients with cardiomyopathy.
The great news is APPOLO’s positive trial results and the most exciting news is the EU CHMP’s recommendation for approving Alnylam’s RNAi product Onpattro™ (patisiran) for a devastating life-threatening disease hATTR amyloidosis. The approval is expected to first come from Europe, to be followed with an approval by the United States and by other countries’ regulatory agencies around the world. The greatest news definitely for the patients suffering the hATTR amyloidosis’ excruciating pain and debilitation symptoms who are spending every minute of their short lives in expectation for heart deterioration and imminent death at a young age.
The credit for this success surely belongs to Alnylam, which, for decades has struggled to make the RNAi and other antisense products work safely and effectively – a task that almost nobody believed it would be a success. As a matter of fact, The long Road Alnylam walked was full of problems that obstructed most of the solutions aimed at making the RNAi a therapeutics, i.e., safe and effective as treatment. Thanks to its consistency and strong scientific fundamentals Alnylam seems to have won this battle.
We hope that this news will be the first in a chain of more approvals that many honest investors were hoping for, but were made skeptical about.
With the indispensable use of novel criteria for the evaluation of the biotech firms, read in the Prohost upcoming Letter about firms that we believe have the greatest potential of future growth and why.