The World’s Most Expensive Drug ($1,210,000/dose) From UniQure B.V. Will Soon Become Extinct

Written by John Carroll

UniQure B.V. (QURE) announced this morning that it has decided not to seek the re-authorization authority that would have been required to keep this drug on the market past October. After bypassing the FDA as a lost cause and seeing only one reported use of the gene therapy in Europe, which provided a conditional approval, the company would prefer to stick with its newly refocused pipeline plan…

A list of the world’s most expensive drugs recently appeared, from Reinsurance Group of America, and we thought that this would be a good time to show you how, and why, a swelling group of pricey orphan therapies has been hitting the market.

In a recent investigation, Kaiser Health News concluded that the law on orphan drugs — which offers a number of incentives, including seven years of marketing exclusivity — had inspired a long list of pricey new therapies.

Here are the most expensive ones, according to annual list prices. There are undisclosed discounts available.

1. Glybera – $1,210,000

Company: uniQure B.V. (Nasdaq: QURE)
Category: Rare disease — EU only
(alipogene tiparvovec)

The world’s first gene therapy is the priciest — and also one of the least used — approved drug on the planet. OK’d only in Europe for lipoprotein lipase deficiency, payers don’t want to cover this one. UniQure has had to restructure the company in recent months, tailoring R&D to focus on the competitive hemophilia B area, Parkinson’s and a collaboration with Bristol-Myers Squibb.

2. Ravicti — $793,632

Company: Horizon Pharma plc (Nasdaq: HZNP)
Category: Rare disease – orphan drug
(glycerol phenylbutyrate)

Approved initially for Hyperion in 2013 as a treatment for urea cycle disorders, Horizon saw the potential and acquired the company for about $1.1 billion, boosting its portfolio of rare disease drugs.