Luxturna gene therapy approved for vision loss

Spark Therapeutics' Luxturna will cost $850,000 the company announced Wednesday

This rare genetic defect causes severe visual impairment beginning in infancy and can eventually lead to blindness. The manufacturers say the price is a bargain.

Luxturna is still significantly more expensive than almost every other medicine on the global market, including two other gene therapies approved earlier a year ago in the U.S. Approved last month, Luxturna, is the nation's first gene therapy for an inherited disease. It is unclear how long the benefit of the treatment will ultimately last.

Besides Luxturna, several other medications are near the million-dollar price mark, although they are often administered in several doses.

"But it's the right move", he continues, "and highlights a recent trend toward (relative) pricing moderation with big implications for the drug industry".

Spark Therapeutics gained approval from the Food and Drug Administration in December for Luxturna (voretigene neparvovec), ahead of its February user fee action date.

The announcement comes amid a growing debate in the United States over the high cost of medicine, particularly for supposedly innovative treatments.

"We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients", said Jeffrey Marrazzo in an interview Wednesday. But gene therapies are given only once, with potentially permanent effects.

The next year will be critical in showing that gene therapies are marketable.

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Spark Therapeutics has devised a set of payment and access programs to make Luxturna more accessible while removing additional costs and risks that may face insurers and hospitals, CNBC News said.

As questions swirl around the cost and effectiveness of gene therapy, it could not be doubted that Luxturna is a breakthrough in the development of this kind of medicine.

But even compared to other one-time gene therapies Luxturna is still an outlier. "Our system can not handle unjustified prices like this, and the new payment models announced today are merely a way to disguise a price that is simply too high", said David Mitchell, a patient with incurable blood cancer and the president of Patients For Affordable Drugs. And the company is also working on covering patients' out of pocket costs as part of the overall price.

Kymriah is a leukemia treatment made by pharmaceutical giant Novartis.

The FDA then approved Luxturna a couple of weeks ago.

Fundus albipunctatus, which makes seeing in low light hard and is sometimes called 'night blindness'.

Spark Therapeutics CEO Jeff Marrazzo co-authored a recent blog post in the journal Health Affairs that said "the current payment system - accustomed to ongoing treatment of chronic diseases - creates significant challenges" for gene therapies and other treatments.

"Although much of this may still sound like the realm of mad scientists tinkering with the human body, gene therapy is an accepted experimental technique that is now being used to help patients with certain types of cancer to "target" specific antibodies that can be used to fight the disease", according to a blog post by Science Care, which runs a whole body donation program.

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