Albuquerque Journal

Price tag on gene therapy for rare blindness: $850K

Critics say cost cut from $1M a distractio­n from ongoing price problems

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WASHINGTON — A first-ofits kind genetic treatment for blindness will cost $850,000 per patient, making it one of the most expensive medicines in the world and raising questions about the affordabil­ity of a coming wave of similar gene-targeting therapies.

The injectable treatment from Spark Therapeuti­cs can improve the eyesight of patients with a rare genetic mutation that affects just a few thousand people in the U.S. Previously, there has been no treatment for the condition, which eventually causes complete blindness by adulthood.

Pricing questions have swirled around the treatment due to a number of unusual factors — it is intended to be a one-time treatment, it treats a very small number of patients and represents a medical breakthrou­gh.

Previously, Spark suggested its therapy, Luxturna, could be worth more than $1 million. But the company said Wednesday it decided on the lower price after hearing concerns from health insurers about the affordabil­ity of the treatment.

Consternat­ion over skyrocketi­ng drug prices, especially in the U.S., has led to intense scrutiny from patients, politician­s, insurers and hospitals.

“We wanted to balance the value and the affordabil­ity concerns with a responsibl­e price that would ensure access to patients,” said CEO Jeffrey Marrazzo, in an interview with The Associated Press.

Luxturna is still significan­tly more expensive than nearly every other medicine on the global market, including two other gene therapies approved earlier last year in the U.S.

Pharmaceut­ical industry critics said the slightly lower cost is a distractio­n from the ongoing problem of unsustaina­ble drug prices.

“The company very cleverly convinced everyone that they were going to charge a million dollars, so now they are being credited for being reasonable,” said Dr. Peter Bach, director of a policy center at Memorial Sloan Kettering Cancer Center in New York.

 ?? BILL WEST/ASSOCIATED PRESS ?? Dr. Albert Maguire checks the eyes of Misa Kaabali, 8, at the Children’s Hospital of Philadelph­ia after he received his gene therapy.
BILL WEST/ASSOCIATED PRESS Dr. Albert Maguire checks the eyes of Misa Kaabali, 8, at the Children’s Hospital of Philadelph­ia after he received his gene therapy.

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