Alnylam Makes History With Approval Of RNA Therapy

Biopharmaceutical company Alnylam Pharmaceuticals ALNY made history this week by kickstarting the future of gene therapy and disease research.

What Happened

On Friday, the U.S. Food and Drug Administration approved Onpattro, the first RNA-based therapy used to treat a rare disease, polyneuropathy. The treatment is designed to silence messenger RNA, blocking the production of certain harmful proteins.

Why It’s Important

The landmark approval is for the treatment of a rare form of polyneuropathy that is caused by hATTR that affects 50,000 people worldwide.

The disease is debilitating and often fatal, as it affects peripheral nerves, the heart and other organs, according to the FDA. 

“This approval is part of a broader wave of advances that allow us to treat disease by actually targeting the root cause, enabling us to arrest or reverse a condition, rather than only being able to slow its progression or treat its symptoms,” FDA Commissioner Scott Gottlieb said in the press release. 

“In this case, the effects of the disease cause a degeneration of the nerves, which can manifest in pain, weakness and loss of mobility.”

What’s Next

New technologies such as those developed by Alnylam have the ability to transform medicine, giving researchers more exposure to treat and cure diseases, Gottlieb said. 

“We’re committed to advancing scientific principles that enable the efficient development and review of safe, effective and groundbreaking treatments that have the potential to change patients’ lives."

The new drug should hit the market in 48 hours, according to Alnylam CEO John Maraganore.

Bloomberg reported that Alnylam has listed the annual average list price for Onpattro at $450,000, but the price could be reduced to $350,000 with discounts. 

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