Spark Therapeutics offers medicine for treatment of rare inherited form of blindness


The company has opted for a slightly lower price of $425,000 per eye and has announced three programmes making the one shot per eye treatment more manageable for USA payers.

The FDA has given the green light to several gene therapies since last summer, as decades of research into the genetic building blocks of life begin translating into marketable treatments. It can improve the vision of those with a rare form of blindness that is estimated to affect just a few thousand people in the U.S.

The mutation causes the gradual disruption of the cells located in the back of the eye that form the retina. This results in patients slowly losing their vision, leading to blindness for which there is no cure.

Surprisingly, a single treatment helped most of the 21 volunteers who got it, reversing vision loss nearly immediately.

The inherited disorder it treats often leads to blindness, reports CNN's Susan Scutti. The drug meant to be taken for life. Similar to Luxturna, Kymriah is a one-time treatment, and its price tag is $475,000.

"I feel like we made the right middle ground decision", Marrazzo said in an interview, balancing the company's desire to capture the economic value of Luxturna while ensuring patients will have access to the therapy. There are only about 1,000 cases of retinal dystrophy in the US now.

Drug prices are not regulated in the US, as they are in many other countries, so drugmakers can price their goods like any other manufacturer.

And it's in negotiations with the Centers for Medicare and Medicaid Services, the federal government health insurance agency, to come up with ways to let insurers pay by installments.

Hardly anyone pays the full price of drugs out of pocket, however.

Express Scripts Chief Medical Officer Steve Miller, who is well known for his critiques of high drug prices, called Luxturna's $850,000 price tag "responsible".

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In August 2017 the FDA approved the first gene therapy for public use in the United States.

Spark will also offer patient assistance programs with the aim that all eligible patients have access to Luxturna.

"We've got to figure this out, because - let's be frank - there are going to be more of these drugs coming to the marketplace for even bigger populations", Miller said.

In clinical studies, patients' sight was examined based on their ability to navigate an obstacle course in varying light levels, and those who received the treatment showed huge signs of improvement in doing so at low light after a one-year period, notes CNBC.

But patient advocate groups aren't buying it.

"Spark Therapeutics is charging as much for Luxturna as they think they can get away with", Patients for Affordable Drugs president David Mitchell said in a statement shortly after the announcement.

Luxturna is delivered to patients just once.

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.

Today, finally, is the beginning of another important chapter in the gene therapy story.