More and more, specialty drugs carrying eye-popping price tags are winning FDA approvals. While more are certainly on the way—look at Novartis' recent presentation that a gene therapy could be worth millions per patient—analysts at drug pricing website GoodRx have tallied up the most expensive pharmacy-dispensed drugs in the U.S. per month as of November. Readers may notice many of pharma's costliest drugs are absent from the rankings, as many of those are administered in a doctor's office or hospital. For its ranking, GoodRx instead focused on retail pharmaceuticals.

Some are marketed by small pharma companies such as Vyera Pharmaceuticals, while top biotechs and pharmaceutical companies market others. Some have seen controversy, while others are lesser known. Rare disease meds make up much of the list, while some treatments such as those for hepatitis C treat diseases that affect millions of people. Gilead, Shire and AbbVie each have multiple offerings. The drugs range from about $300,000 per year to more than $600,000 per year. For reference, several doctor- or hospital-administered meds such as BioMarin's rare disease med Brineura rank above pharmacy meds; that med costs $702,000 per year before discounts.

Many drugmakers offer patient assistance programs to help patients afford the meds, and the prices are listed before confidential rebates and discounts.

Nos. 1 through 5: From a price-hike poster child to a blockbuster-to-be

Topping the drug pricing rankings is Horizon’s Actimmune for malignant osteoporosis and chronic granulomatosis. The drug makes routine appearances on most-expensive lists, and has changed hands several times since its 2000 approval. Horizon hiked its price by 9.9% at the end of last year, according to Jefferies analysts. The drug now costs $52,322 per month, according to GoodRx. 

Behind Actimmune comes the controversial Daraprim, a toxoplasmosis treatment formerly marketed by Martin Skhreli’s Turing Pharmaceuticals and now sold by Vyera Pharmaceuticals. Shkreli increased the drug’s price by 5,000% overnight and lit a drug pricing firestorm that's led to a now-constant drumbeat of pricing criticism. Now, lawmakers and others are threatening more action against pharma, and the increased scrutiny has hurt the industry's pricing power.

Since the notorious price increase, Shkreli has been sentenced to a prison term on fraud charges relating to his time at another biotech, Retrophin. Daraprim costs $45,000 per month, GoodRx reports.

Shire’s hereditary angioedema drugs Cinryze and Takhzyro take the No. 3 and No. 4 spots, respectively, carrying list prices of $44,140 per month. Cinryze is the older of the two meds; patients typically require 16 vials in a month.

Takhzyro, for its part, won approval in August and requires two vials per month. Each vial costs $22,070 before discounts, according to GoodRx. Jefferies analysts have predicted $1.8 billion in peak sales for the newer drug, making it among the blockbusters on the most-expensive rankings. The two brands are part of a rare disease lineup that attracted a $62 billion buyout offer from Takeda—a deal set to close in January.

Behind Shire’s two meds falls Chenodal, a drug to dissolve gallstones marketed by rare disease drugmaker Retrophin. On Shkreli’s watch, in 2014, the drug's price grew fivefold, GoodRx reports. And even though Chenodal is off patent, generic drugmakers can’t duplicate it, thanks to a "closed distribution system" that prevents them from obtaining samples needed for testing, according to the website.

The drug costs $42,570 per month, because patients typically take 90 tablets at $473 per tablet, GoodRx said. Some patients need up to 210 tablets per month, though, pushing their monthly price to nearly $100,000.

Nos. 6 through 10: Featuring a new Ionis drug and an in-the-news Mallinckrodt med

Outside of the top 5, Aegerion's Myalept costs $42,138 per month and treats leptin deficiency in patients with generalized lipodystrophy. Aegerion picked up the drug—which patients self-administer once daily—from AstraZeneca in a 2014 deal worth $325 million up front.

Mallinckrodt’s controversial H.P. Acthar makes an appearance at the No. 7 position, costing nearly $39,000 per month. Mallinckrodt picked up the drug in its 2014 deal for Questcor, which had dramatically raised its price—including a huge hike to $23,000 per vial from $1,650 overnight in 2007, according to reports. The drug won its initial approval in 1952 and cost $40 per vial in 2001.

Rare disease drugmaker Aegerion's Juxtapid holds the No. 8 spot. The drug treats homozygous familial hypercholesterolemia, a hereditary form of extremely high cholesterol, and costs nearly $37,000 per month, GoodRx said. Aegerion ran into trouble for promoting the drug off label, and in September 2017 it inked a $36 million settlement with the Department of Justice to settle those allegations. A federal judge rejected the deal, but later sentenced the drugmaker to pay the same amount, stipulating that a portion of the funds should go to patients.

A brand-new drug captured ninth place: Ionis' Tegsedi made its debut among the 10 most expensive retail drugs in the U.S. with its $34,600 monthly price tag. The drug treats hereditary transthyretin-mediated amyloidosis and will face Alnylam's Onpattro on the market. Pfizer's tafamidis is looming in ATTR as well.

Shire's third drug in the rankings, Firazyr, costs $32,468 per month and rounds out the top 10 list of most expensive retail drugs in the U.S., as ranked by GoodRx. Firazyr won approval in 2011 and treats patients patients with hereditary angioedema after an attack. One carton contains three syringes, according to GoodRx.

Nos. 11 through 15: Hepatitis C and an aging $261-per-tablet remedy

Aside from No.1 ActImmune, Horizon Pharma also markets the eleventh most expensive drug in the U.S., GoodRx reports. Horizon's Ravicti treats urea cycle disorders and came to the drugmaker through the company's $1.1 billion buyout of Hyperion in 2015. Ravicti was Horizon's top-performing med by sales last year, generating $193 million.

No. 12 in the rankings may be the most commercially successful drug among the most expensive retail meds in the U.S. Gilead's Harvoni, a megablockbuster hep C drug, costs $31,500 per month. It's important to note that the drug is taken in a three-month course and cures hep C, potentially allowing for a higher price than meds that require ongoing use. After launch in 2014, the drug took off, generating billions in sales before competition eventually caught up and hurt Gilead's pricing power. As its hep C business has faltered more recently, Gilead is launching authorized generics to its own meds to provide list price relief.

Price-hike poster child Valeant—now known as Bausch Health Companies—markets the No. 13 drug on GoodRx's list. Cuprimine, a decades-old drug that treats Wilson's disease and rheumatoid arthritis, runs at more than $261 per tablet, according to the pricing website. Over the years, Valeant dramatically increased the price of the med and others, sparking investigations and controversy. The high-flying company's fortunes eventually fell on increased scrutiny, and this year it changed its name in an effort to move forward.

Three hep C drugs fall next in the rankings. Rounding out the top 15 are Gilead's Sovaldi, which costs $28,000 per month, and AbbVie's Viekira Pak at $27,773 per month. The drugs were the first in a new class of highly effective hep C therapies. Gilead's Sovaldi hit the market first, giving the big biotech pricing power that it used to generate billions in sales. It wasn't until AbbVie launched Viekira that net prices came down in the class; they've fallen significantly since then due to even more competition.

Nos. 16 through 20: Rare disease meds and another hep C drug

AbbVie's extended-release version of Viekira holds the No. 16 position at $27,773 per month. Since AbbVie released its Viekira drugs, the company has also rolled out the successful Mavyret, which cures hep C in 8 weeks. In the first three quarters of 2018, AbbVie's hep C sales rang in at $2.754 billion, of which $2.61 billion came from Mavyret. Viekira generated $135 million in the first 9 months.

Orfadin, ringing in at No. 17, treats hereditary tyrosinemia type 1 and costs $27,247 per month, according to GoodRx. According to the drug's FDA label, Apotek manufactures Orfadin and Sobi markets it in the U.S. Orfadin won initial approval in 2002.

Johnson & Johnson's Actelion unit markets Zavesca, a type 1 Gaucher disease med that costs $26,820 per month and ranks 18th among the most-expensive retail drugs. The med isn't a huge seller, and it has head-to-head competition from Sanofi's Cerdelga, according to the National Gaucher Foundation. In 2016, before its buyout by J&J, Actelion reported about $105 million in Zavesca sales. J&J doesn't break out Zavesca's sales numbers.

Agios' Tibsovo, approved in July, is among the newer meds on the GoodRx ranking at $26,115 per month. The drug treats certain patients with relapsed/refractory acute myeloid leukemia, a group that amounts to 700 to 1,100 patients in the U.S., SunTrust analyst Yatin Suneja said, predicting $300 million in peak sales. After its July approval, Agios CEO David Schenkein said the FDA nod is an "incredibly exciting milestone" for the biotech and for patients "who have been waiting for new treatment options that work radically different than conventional chemotherapy."

Rounding out GoodRx's top 20 rankings is United Therapeutics' Remodulin, which treats pulmonary arterial hypertension. The drug costs $25,466 per month, but could face generics soon under patent agreements the company has inked with Sandoz, Teva, Par Pharmaceuticals and other generics companies. Remodulin won initial approval in 2002.

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