Ultra-rare disease specialists top 2021's pricey drugs list, but Takeda, Merck KGaA meds rank, too

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Eiger BioPharmaceuticals' Zokinvy, recently approved by the FDA for ultrarare rapid-aging diseases that affect an estimated 600 children worldwide, is the most expensive drug in U.S. pharmacies in 2021, according to GoodRx.(Jacoblund)

Eiger BioPharmaceuticals in November snagged FDA clearance for Zokinvy for ultra-rare rapid-aging diseases that affect an estimated 600 people worldwide. Orphan drugs that treat small patient populations typically come with high prices, but Zokinvy’s sticker price now beats them all in U.S. pharmacies.

At an estimated monthly price of $86,000, or more than $1 million a year, Zokinvy’s now the most expensive pharmacy drug in the U.S. as of February, according to a list compiled by GoodRx. The ranking focuses on monthly prices and doesn’t include drugs administered by doctors.

Zokinvy, a former cancer candidate Eiger licensed from Merck & Co., bumped Amryt Pharma’s fat tissue deficiency therapy Myalept off the No.1 spot from last year’s list. It’s so expensive that it even ranks as the second-most expensive drug if medicines administered in the clinic or hospital are also counted, next only to Novartis’ $2.125 million one-time spinal muscular atrophy gene therapy Zolgensma.

The FDA handed Zokinvy its go-ahead to slow the development of—not cure—Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies, two genetic diseases marked by accelerated aging that affect 400 and 200 patients worldwide, respectively. In the U.S., only 20 patients have been identified, an Eiger spokesperson said in a statement. Dosing is adjusted based on body surface area, and GoodRx based its calculation on patients taking about 200 mg of Zokinvy a day. 

Eiger officially launched the med in January as the only available treatment for ultra-rare conditions. Because most of the eligible patients identified in the U.S. have already received Zokinvy through an Eiger expanded access program, they are now rolled into a commercial program called Eiger OneCare, which offers co-pay assistance and other patient support. Its approximate average annual net price per patient, including mandatory government discounts, is expected to be around $650,000, the Eiger spokesperson said.

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With Zokinvy’s entry, Myalept, at a monthly list price of $74,159, fell to second place, followed by Merck KGaA’s multiple sclerosis drug Mavenclad, which costs $60,371 for a cycle of treatment. It’s worth pointing out that Zokinvy and Myalept are taken every day, while Mavenclad—exact dosing of which is dependent on weight—is administered only two cycles a year spaced out by a month. GoodRx’s chosen dosing would equate to about $120,000 a year for Mavenclad.

Besides Zokinvy, BioCryst Pharmaceuticals’ freshly approved oral hereditary angioedema drug Orladeyo is also a new entry in this year’s list, ranking 13th with a monthly list price of $37,308. Still, it came in below Takeda’s rival HAE injection Takhzyro, which earned the 7th spot at a monthly cost of $46,828.

RELATED: The 20 most expensive pharmacy drugs in the U.S. in 2020, from fallen blockbusters to new orphan meds

Deciphera Pharmaceuticals’ Qinlock, approved by the FDA last May for heavily pretreated patients with gastrointestinal stromal tumors, made it to 17th place at a monthly price of $32,000. Bristol Myers Squibb’s legacy Celgene blood cancer drug Idhifa rounds out the top 20 thanks to its $28,246 price.

Gilead Sciences’ aging hepatitis C drug Solvadi—once criticized for its high price—AbbVie’s rival Viekira Pak HCV co-package and Agios Pharmaceuticals’ acute myeloid leukemia drug Tibsovo are among those knocked off the top 20 roster for 2021.

Twelve of the 20 drugs have seen price increases this year, including Myalept’s 4% and Mavenclad’s 6%, GoodRx noted.