The top 10 most-expensive meds in the U.S.—and they're not the usual suspects

Two new gene therapies—from Novartis and Spark Therapeutics—carry price tags higher than any other drugs in the U.S., elbowing aside the brands we've seen at the top for years, a GoodRx analysis shows. And with drugmakers doubling down in rare diseases, the trend will likely continue.

According to the GoodRx top 10 ranking, Novartis’ new spinal muscular atrophy gene therapy Zolgensma far and away leads the list with its one-time price of $2.125 million.

Despite its high cost, some experts have said Zolgensma is cost-effective because it can cure a disease that costs hundreds of thousands of dollars to treat annually. Even so—and despite installment plans that spread the payments over time—other experts have criticized Novartis for choosing that price tag.

Another one-time gene therapy, Spark’s Luxtura, stands in second place behind Zolgensma at less than half the sticker: $850,000. That treatment can cure an inherited retinal disease that leads to blindness. Roche is in the process of buying Spark, but the deal has run into numerous regulatory delays.


GoodRx regularly compiles lists of the most expensive pharmacy-administered meds. But this time, the team expanded its research to drugs patients receive directly from healthcare practitioners, which often come with high price tags. 

The prices GoodRx quotes don't account for the behind-the-scenes discounts and rebates drugmakers offer to payers.

The No. 3 drug in the rankings is the first pharmacy-dispensed drug in the list: Aegerion’s Myalept to treat leptin deficiency in patients with generalized lipodystrophy. The med typically costs $778,314 per year, according to GoodRx.  

Spectrum Pharmaceuticals’ peripheral T-cell lymphoma med Folotyn ranks No. 4 with an annual price of $745,785. Spectrum recently inked a deal to sell its marketed therapies to Acrotech Biopharma, a subsidiary of India’s Aurobindo, for $300 million. With the deal, the company plans to focus on novel oncology drugs. 

RELATED: The 20 most expensive pharmacy drugs in 2018, featuring names big and small

Alexion’s Soliris is a drug that regularly appears on most-expensive lists—and has spent time at the top—this time captures the No. 5 spot with an annual price tag of $678,392. The med treats paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome, and it has been a cornerstone of Alexion’s business for years. The company also markets follow-up Ultomiris, but that drug doesn’t appear on GoodRx’s ranking. 

While many drugs on the list are from smaller, rare disease-focused companies, Amgen makes an appearance with the No. 6 spot. The company’s Blincyto to treat Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia typically costs $641,533 per year, GoodRx found.  

An Amgen spokeswoman said ALL patients usually “have a very poor prognosis, with a median overall survival of three to five months.” Patients on Blincyto typically live twice as long as those on standard-of-care chemo, she said. A course of treatment is $106,900 per cycle before discounts, and the majority of patients receive two cycles or less.  

RELATED: FDA gifts Alexion quick OK for Soliris follow-up Ultomiris 

Horizon Pharma markets two of the next three drugs in the rankings—No. 7 Ravicti to treat urea cycle disorders and No. 9 Actimmune to treat osteopetrosis and chronic granulomatous disease. The meds typically cost $633,072 and $575,540 per year, respectively. Sanofi’s Lumizyme for Pompe disease ranks No. 8 at $630,630 per year. 

Takeda’s Takhzyro rounds out the top 10 with an annual cost of $573,820. Picked up in Takeda’s Shire buyout, the med helps prevent attacks of hereditary angiodema.