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Tags: first | million-per-treatment | drug

First $1 Million-per-Treatment Drug on Way to Patients

First $1 Million-per-Treatment Drug on Way to Patients
(Sergei Primakov/Dreamstime)

By    |   Tuesday, 08 May 2018 01:37 PM

The first $1 million-per-treatment drug is coming – one targeting hemophilia – with some believing its cost will set a new normal in gene therapy drug pricing. It follows a DNA-related blindness cure priced at $850,000 per treatment.

Two analysts at Leerink said on Monday that new gene therapies targeting hemophilia, a disease affecting blood's ability to clot, may carry prices of $1.5 million or more, CNBC reported. Drugmakers BioMarin, Spark Therapeutics and UniQure have led in the development of the hemophilia drugs.

Gene therapies deliver a healthy copy of a gene to make up for a defective one that causes disease, aiming to cure, or greatly improve, the ailment in one treatment.

"It appears the seemingly impervious million-dollar threshold may be breached with hemophilia gene therapy, which could do so while still creating value for society by reducing the cost of factor replacement therapy," Leerink analysts Joseph Schwartz and Dae Gon Ha said in their research note, according to CNBC.

Bloomberg reported last month that drug companies were trying to figure out how to charge for such DNA treatments that are designed as permanent fixes to be given just once. Spark Therapeutics Inc. had set a price for its Lexturna treatment to repair a rare, inherited form of blindness at $850,000, or $425,000 per eye.

"It's wildly expensive but, to be very frank, I think they've priced it what I'll call responsibly," said Dr. Steve Miller, chief medical officer of pharmacy benefits manager Express Scripts, which is partnering with Spark on distribution and specialty pharmacy services for Luxturna, per CNBC.

"The product is just phenomenally innovative, and we've been talking about gene therapy for over 20 years. We're now at the threshold of having gene therapy reaching patients," Miller said.

UniQure chief scientific officer Sander van Deventer told Bloomberg in April that he didn’t see the cost of the hemophilia drug as a deterrent to people who would need it.

"In the developed world, there is no resistance to relatively high-priced drugs, as long as they work very well," said van Deventer, whose company is based out of Amsterdam.

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TheWire
The first $1 million-per-treatment drug is coming – one targeting hemophilia – with some believing its cost will set a new normal in gene therapy drug pricing. It follows a DNA-related blindness cure priced at $850,000 per treatment.
first, million-per-treatment, drug
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2018-37-08
Tuesday, 08 May 2018 01:37 PM
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