FDA Approves new gene therapy for treatment of rare form of blindness

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@GentleSoul and @twinklestars do u have any thought…!!!

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““I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses,” said FDA Commissioner Scott Gottlieb, M.D. “We’re at a turning point when it comes to this novel form of therapy and at the FDA, we’re focused on establishing the right policy framework to capitalize on this scientific opening.””

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Also:

“The FDA explains the RPE65 mutation affects around 1,000 to 2,000 patients in the U.S, with Spark Therapeutics Inc — developers of Luxturna — previously stating nearly 6,000 people around the world could benefit from the treatment.”

This is good news. Goes to show that gene therapy is/will nit inly be applued to “mainstream” diseases. Now if i remember correctly the price of luxturna is about 1’000’000.

All in all good news for gene therapy.

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Why so expensive will they be able to afford it…!!!

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Spark isn’t disclosing price until January, but analysts have suggested a potential $1 million price tag for the one-time treatment. Last month, when reporting its third-quarter results, the company said the med offers a “a value in excess” of $1 million, Evercore ISI analyst Steven Breazzano wrote in a note.

It remains to be seen how payers will react. In recent years, some superpricey meds have struggled due to lack of demand, payer restrictions or both. UniQure, for instance, had to discontinue its $1 million gene therapy Glybera in Europe, while GlaxoSmithKline recently said it’s exploring a sale for its rare disease portfolio. The company’s gene therapy Strimvelis has only made its way to a few patients since launch.

With those factors considered, it’ll be up to Spark to convince payers and the medical community its med is worth the price. Consensus estimates for sales are $75 million for 2018, Breazzano noted, which implies treatment of 75 patients at $1 million or 50 patients at $1.5 million. Evercore ISI predicted $381 million in sales for the med in 2019.

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How are you @far_cry0!!!

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@far_cry0, gussa gaya kya be!!!

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@anon68148378 bade kuch nahi mein aab roj khana banata hun… aaj dinner me allo pyaj banaya… aap ki dinner mein kya kya hein…

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Initially it will be expensive but over time the price will decline.

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