Gene Therapies Dominate Top Two Spots in 2019's Most Expensive Drug List

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On June 13, the well-known U.S. prescription drug price comparison website GoodRx released the list of the 10 most expensive drugs in 2019. Two gene therapies from Novartis and Spark Therapeutics surpassed brand-name drugs that had consistently topped such lists in previous years, securing the top two spots. As the pharmaceutical industry continues to show growing enthusiasm for developing new treatments for rare diseases, this trend is likely to persist.

Novartis’ newly launched gene therapy for spinal muscular atrophy, Zolgensma, far outpaces other drugs on the list, with a one-time treatment price of $2.125 million. Despite the high cost, some experts argue that Zolgensma is cost-effective, as it cures a condition that otherwise requires annual treatments costing hundreds of thousands of dollars. Moreover, Novartis has introduced an installment payment plan to spread the cost over time. Nevertheless, some experts have still criticized Novartis for its high pricing.

Ranked second on the list is Luxturna, a gene therapy from Spark Therapeutics. Priced at $850,000, it costs less than half of Zolgensma. Luxturna is also a one-time gene therapy that can treat inherited retinal diseases causing blindness due to biallelic mutations in the RPE65 gene.

GoodRx regularly compiles a list of the most expensive pharmacy drugs. But this time, the research team expanded its scope to include medications obtained directly from healthcare providers, which often carry high price tags. The prices cited by GoodRx do not reflect behind-the-scenes discounts and rebates provided by pharmaceutical manufacturers to payers.

Ranking third is the first pharmacy-dispensed drug on the list: Aegerion’s Myalept, which is used to treat leptin deficiency in patients with generalized lipodystrophy. According to GoodRx data, the annual treatment cost for this drug is $778,314.

Spectrum’s peripheral T-cell lymphoma drug, Folotyn, ranked fourth, with an annual price of $745,785. Spectrum recently signed an agreement to sell the aforementioned therapy to Acrotech Biopharma, a subsidiary of the Indian pharmaceutical company Aurobindo, for $300 million.

Rare disease giant Alexion’s Soliris is a drug that frequently appears on lists of the most expensive medications, having topped the rankings for many years. This year, it ranks fifth with an annual price of $678,392. The drug is used to treat paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), and has long been the cornerstone of Alexion’s business. The company also markets Ultomris, an upgraded version of Soliris, but this drug did not appear in the current ranking.

Although many drugs on the list come from smaller companies focused on rare diseases, Amgen appears in sixth place. Its bispecific antibody therapy Blincyto, used to treat Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia, costs $641,533 per year.

On this list, Horizon occupies two spots, with its drug Ravicti for treating urea cycle disorders and Actimmune for treating osteoporosis and chronic granulomatous disease ranking seventh and ninth, respectively, with annual prices of $633,072 and $575,540.

Sanofi’s Pompe disease drug, Lumizyme, ranks eighth, with an annual cost of $630,630.

Takeda’s hereditary angioedema treatment, Takhzyro, ranks tenth with an annual price of $573,820. The drug was acquired by Takeda through its $64.4 billion purchase of Shire.

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