Connect with us

News

US drug firm offers cure for blindness – at $425,000 an eye

Spark Therapeutics says ‘responsible price’ for Luxturna gene therapy ensures access for patients with retinal defect

Published

on

 

Powered by Guardian.co.ukThis article titled “US drug firm offers cure for blindness – at 5,000 an eye” was written by Rob Davies, for The Guardian on Wednesday 3rd January 2018 18.15 UTC

A drug whose inventors claim it can cure a rare form of blindness is to be one of the most expensive medicines ever sold at 0,000 (£630,000).

Luxturna is injected directly into the eye to address the root cause of visual impairment by replacing a defective gene in the retina. It is the first gene therapy to be approved for use in the US, and was given the go-ahead by the Federal Drug Administration last month. However, the price of the treatment has only just been revealed.

Spark Therapeutics, the company behind the treatment, had previously claimed the treatment was worth m, citing the cost of a lifetime of blindness in lost earnings and wages for caregivers. But the firm said it had settled for the lower price of 0,000, or 5,000 per eye. It will also offer ways to spread the cost to health insurers, which have expressed concern about their ability to cover the expense.

“We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients,” said Spark Therapeutics’ chief executive, Jeff Marrazzo.

Luxturna is one of an emerging breed of gene therapies that differ from more established medicines administered over a period of time. Such treatments involve a one-off procedure to alter defective DNA, allowing the body to repair the problem itself.

They include a treatment for haemophilia and another for so-called “bubble baby” syndrome, where sufferers have to live in a sterile environment, which is to be offered on the NHS despite a £500,000 price tag.

The sky-high price of some drugs – and so-called price-gouging by drug firms – became an issue in the US presidential election after Martin Shkreli, a US hedge fund entrepreneur, bought the drug Daraprim, used in the treatment of Aids and cancer, and hiked its price from .50 to 0.

But the starting price of some new drugs has soared. Glybera, a gene therapy for a rare protein disorder, was launched in 2012 with a price tag of .2m. However, it was never approved in the US and was discontinued by manufacturers uniQure because of a lack of demand.

a vial of Luxturna (voretigene neparvovec-rzyl)
A vial of Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl), which was approved for use by the FDA on Tuesday. Photograph: AP

Gene therapy is not alone in commanding staggering sums, particularly when it comes to treatments for rare diseases. Soliris, a drug that treats a condition called paroxysmal nocturnal hemoglobinuria that attacks red blood cells, can cost up to 0,000 a year, while Elaprase, used in the treatment of Hunter syndrome, costs 0,000 a year.

Luxturna could potentially become available free on the NHS in the UK after being submitted for approval by the European Medicines Agency (EMA). A green light from the EMA is a prerequisite for approval by the National Institute for Health and Care Excellence, the UK’s healthcare costs watchdog.

In clinical trials, injections of Luxturna restored eyesight to people with severe visual impairment due to retinal dystrophy. Spark estimates that up to 2,000 people in the US suffer from the condition, with the number of sufferers rising to 6,000 when Europe and other markets where it could sell the treatment are included.

The company said it had agreed bespoke deals with US insurers, which cover the cost of most US prescriptions, and they will get a refund if the drug doesn’t work as expected.

Spark is also discussing a proposalfor insurers to pay for the drug in instalments over several years. The Philadelphia-based company said it would also pay transport costs not covered by insurance to help patients get access to treatment centres.

guardian.co.uk © Guardian News & Media Limited 2010

Published via the Guardian News Feed plugin for WordPress.

Continue Reading
Advertisement

News

FACT CHECK: Did President Obama lie about his knowledge of Hillary Clinton using a private email address

Published

on

BNL News tweeted that President Obama ‘lied’ about his knowledge of Hillary Clinton using a private email address

However this claim is misleading,

News reports from that period back up the claim Obama denied knowledge of the use of a private email however The White House clarified that he was referring to his knowledge of the fact that she ran it out of her home.

New York Times Report (Click To View)

However the White House later clarified that Obama was referring to Clinton’s apparently lack of compliance with State Department rules by not submitting the communications as formal records for nearly six years.

“Yes, he was aware of her email address. He traded emails with her,” Earnest said. “But he was not aware of her personal email server or that she was using it exclusively for all her business.”

Time Report (Click to View)

A FactCheck.org article goes through this in depth, Click Here To Read

NewsThisSecond rates the claim misleading due to the tweet suggesting former President Obama lied.

 

Continue Reading

News

READ: Inspector General Report on Justice Department, FBI, Clinton emails

Published

on

Continue Reading

News

READ:House GOP interim findings on Clinton email investigation

Published

on

A Republican-authored document detailing some of the preliminary findings of a joint investigation by the House Judiciary and Oversight committees into the Hillary Clinton email investigation.

Continue Reading

Popular

Copyright © 2018 News This Second