FDA Approves Lifesaving Drug That Costs $2.15 Million, The Most Expensive Drug Ever

The drug treats a fatal condition in infants.

an iv bag filled with medicine
NIAID / Flickr (CC BY 2.0 Cropped, resized.)

The drug treats a fatal condition in infants.

The Food and Drug Administration (FDA) has approved a drug that comes with a $2.15 million price tag, the most expensive drug ever, Medical Express reports.

Swiss drugmaker Novartis has gained approval to sell Zolgensma (Onasemnogene abeparvovec, or AVXS-101) through its U.S. subsidiary AveXis. The drug treats spinal muscular atrophy, an extremely rare genetic condition that manifests at birth. Infants with the disease have no muscle control, and eventually are rendered unable to move, breathe, or swallow. Almost all die within their first two years of life. The disease affects about 400 children born each year in the United States.

The drug will be administered to children under two years of age who have been confirmed via a genetic test to have the disease. The medicine will be introduced via an infusion that should take about an hour.

With a price tag of $2.15 million per treatment, it is the most expensive drug in the world. Another drug that treats the same disease, Spinraza, costs $750,000 for the first year of treatment, and $350,000 per year after that. Spinraza is given every four months.

Why Is It So Expensive?

Back in 2018, maker Novartis, on its website, discussed the research and development of the drug, the results of clinical trials, and its chemical makeup. Putting aside the detailed scientific and clinical minutiae, the bottom line is that the drug is a biologic — that is, it was developed via the genetic manipulation of living cells, in this case from a harmless virus that infects some primates.

Such research and development does not come without cost. In fact, the Institute for Clinical Review, which evaluates and offers commentary on the cost of drugs, said that the staggering price tag for Zolgensma is justified.

“To reach the alternative thresholds of $100,000 to $150,000 per life year gained (LYG), a value-based price benchmark for Zolgensma would be between $1.2 million to $2.1 million.”

Not everyone sees it that way, however.

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Writing in Bloomberg, Dr. Peter B. Bach writes that no drug should cost seven figures, even life-saving drugs like Zolgensma.

“The drug industry will argue, as it always does, that unlimited and rapidly escalating prices are needed to recoup the costs of [research and development]… [In this particular case] Novartis is trying to maximize the return on its financial investment.”

The Confusing Mishmash of Drug Prices

That lifesaving or life-improving drugs can cost thousands, tens of thousands, or even millions of dollars is a problem not lost on the federal government. Just this week, a House of Representatives committee brought in pharmaceutical executives to explain why some drugs cost thousands in the U.S. but only a few dollars in foreign countries, as Business Insider reports.