FDA Approves $450,000-a-Year Treatment for Rare Disease

The Food and Drug Administration
UNITED STATES - NOVEMBER 9 - The outside of the Food and Drug Administration headquarters is seen in White Oak, Md., on Monday, November 9, 2015. The FDA is a federal agency of the United States Department of Health and Human Services and has been in commission since 1906. (Photo By Al Drago/CQ Roll Call)
Al Drago—CQ-Roll Call,Inc.

Alnylam Pharmaceuticals Inc. won approval from the U.S. Food and Drug Administration for its first-of-a-kind treatment for a rare, often deadly nerve disease and plans to charge about $450,000 a year for the therapy.

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. After expected discounts in the U.S., Alnylam said it expects the net cost for the drug to be about $345,000 a year. It plans to offer money back if the drug doesn’t work.

Alnylam spent years developing the drug, called Onpattro, based on Nobel Prize-winning science. Patients with the disease slowly lose sensation in their hands, arms, feet and legs as proteins build up and damage the nerves. Eventually it attacks their organs, often killing them within a few years of diagnosis. About 20,000 to 30,000 people around the world have the condition that Alnylam’s drug treats.

Alnylam set the price based on what it said was a fair reward for an urgent condition with no other treatments that can stop or reverse the disease. In a statement, the biotechnology company called the price a “reasonable return for a limited period of time for the investments it made and risks it took.” It said it would offer financial support to patients so they can afford the drug.

Alnylam shares fell at 7.6 percent to $90 after the market closed in New York

Narrow Group

The FDA approved the drug for a narrower group of patients than investors had hoped. The drug regulator said that Onpattro should be used in patients who already have some nerve damage from the disease.

That could limit the early uptake of the drug. Alnylam said that fewer than 3,000 people in the U.S. have been diagnosed. That number could grow as more patients are treated, said Chief Executive Officer John Maraganore.

“Once we start diagnosing patients, there will be many other family members who will recognize that perhaps they’re also afflicted,” Maraganore said. “I think we’ll begin to see a ripple effect.”

Patients who get the drug will undergo an exam to get a baseline assessment. Nine months to a year later, another exam will determine if the drug is reducing their pain and keeping them out of the hospital. Alnylam will get full payment if patients improve or hold steady. Discounts will be given for patients whose disease worsens, based on the severity of their progression.

Alnylam President Barry Greene said on a conference call Friday that for patients that do well, “we deserve payment in full because that patient is getting tremendous benefit and frankly costing the insurer less.”

“If the patient does less well — they’ve being hospitalized or suffering other medical conditions — there should be some discount on what we are paid,” Greene said.

Silence the Messenger

Onpattro affects what is known as RNA, the biological messenger that takes the coding from DNA and carries those genetic instructions around the body. Silencing or interfering that message can halt or slow the body’s production of the problematic protein found in hATTR patients.

Alnylam’s drug is the first-ever RNA-interference drug approved by the FDA. European regulators have also recommended approval, with a final decision expected in the coming weeks.

Analysts have estimated the drug, which will be marketed as Onpattro, will have sales of $24 million this year and more than $1 billion by 2023, though the restrictions in the FDA level may limit the market. Shares of partner Arbutus Biopharma Corp., which will get royalties on sales, sank as much as 10 percent intraday.

Other drugmakers studying potential medicines in the amyloidosis space include partners Ions Pharmaceuticals Inc. and Akcea Therapeutics Inc., as well as Pfizer Inc., and Eidos Therapeutics, Inc.

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