Mathias (August 10, 2018) reports that Alnylam Pharmaceuticals Inc’s drug for a rare hereditary disease won U.S. regulatory approval on Friday, August 10, 2018. This is newsworthy for the very reason that this will be the first approved treatment from a new class of medicines that use gene silencing technology. Mathias points out that “Alnylam’s patisiran, commercially named Onpattro, was approved to treat polyneuropathy in patients with hereditary ATTR amyloidosis, a potentially fatal condition that affects an estimated 50,000 people worldwide” (p.1). He also comments that the drug Onpattro has already received a positive opinion from European regulators and approval is on the horizon in September 2018.
Lipschultz and Cortez (August 10, 2018) report that “patients who have the condition, called hereditary transthyretin-mediated amyloidosis, or hATTR, will take the drug for the rest of their lives, racking up costs in the millions of dollars” (p.1). They add that “Alnylam said it expects the net cost for the drug to be about $345,000 a year but plans to offer money back if the drug doesn’t work” (p.1). Lipschultz and Cortez also emphasize that patients with the disease slowly lose sensation in their extremities as proteins build up and damage the nerves (p.1). The damage attacks and damages the patient’s organs within a few years which leads to death. Lipschultz and Cortez point out that about 20,000 to 30,000 people around the world have the condition that Alnylam’s drug will treat (p.1).
Unfortunately, “patients with the disease have a genetic mutation that prevents a type of protein from keeping its normal structure, instead causing it to fold into an incorrect shape and accumulate in the heart, nerves and other organs” (p.1). Polyneuropathy, a symptom of the disease, is the simultaneous malfunction of nerves resulting in tingling, numbness and kidney dysfunction. Heart issues, eye, kidney and thyroid diseases are also symptoms.
Lipschultz, B. and Cortez, M. (August 10, 2018). Rare-Disease Treatment From Alnylam to Cost $450,000 a Year. Bloomberg. https://www.bloomberg.com/news/articles/2018-08-10/alnylam-wins-first-u-s-drug-approval-in-rare-genetic-disease
Mathias, T. (August 10, 2018). Alnylam’s gene silencing drug wins FDA approval. Reuters. https://www.cnbc.com/2018/08/10/reuters-america-alnylams-gene-silencing-drug-wins-fda-approval.html
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