Fwd: [Wcb-l] FW: [leadership] Article: FDA approves first gene therapy targeting rare form of inherited blindness

---------- Forwarded message ----------
From: Steve Fiksdal <steve@fiksdal.com>
Date: Tue, 19 Dec 2017 22:52:30 +0000
Subject: [Wcb-l] FW: [leadership] Article: FDA approves first gene
therapy targeting rare form of inherited blindness
To: "wcb-l@wcbinfo.org" <wcb-l@wcbinfo.org>



From: Kelly Gasque via leadership [mailto:leadership@acblists.org]
Sent: Tuesday, December 19, 2017 12:42 PM
To: leadership@acblists.org; announce@acblists.org
Subject: [leadership] Article: FDA approves first gene therapy
targeting rare form of inherited blindness

FDA approves first gene therapy targeting rare form of inherited blindness
Article link: https://www.statnews.com/2017/12/19/gene-therapy-blindness-fda-approval/<https://mandrillapp.com/track/click/30489975/www.statnews.com?p=eyJzIjoicnJwemFUYkYwOUJZZWZDeEFnbmNGa3lERUVZIiwidiI6MSwicCI6IntcInVcIjozMDQ4OTk3NSxcInZcIjoxLFwidXJsXCI6XCJodHRwczpcXFwvXFxcL3d3dy5zdGF0bmV3cy5jb21cXFwvMjAxN1xcXC8xMlxcXC8xOVxcXC9nZW5lLXRoZXJhcHktYmxpbmRuZXNzLWZkYS1hcHByb3ZhbFxcXC9cIixcImlkXCI6XCI1YzYyMjhkMjVhYWI0ZTViOTM2ZmFhMWI2OGI0NTAxN1wiLFwidXJsX2lkc1wiOltcIjU4OTE2MDcwNmU0ZGQ5MTM5MTFlMzVlMzRhODRkMmM1OTc5NmJlYTVcIl19In0>
A first-of-its-kind gene therapy received approval from the Food and
Drug Administration on Tuesday to treat a rare, inherited form of
childhood blindness.
The FDA marketing clearance of Spark Therapeutics's
Luxturna<https://mandrillapp.com/track/click/30489975/www.statnews.com?p=eyJzIjoidnpVNjFiTGxUZ2dZakdxWTBOS0x3NUV5bHdZIiwidiI6MSwicCI6IntcInVcIjozMDQ4OTk3NSxcInZcIjoxLFwidXJsXCI6XCJodHRwczpcXFwvXFxcL3d3dy5zdGF0bmV3cy5jb21cXFwvMjAxN1xcXC8xMFxcXC8wOVxcXC9zcGFyay1nZW5lLXRoZXJhcHlcXFwvXCIsXCJpZFwiOlwiNWM2MjI4ZDI1YWFiNGU1YjkzNmZhYTFiNjhiNDUwMTdcIixcInVybF9pZHNcIjpbXCIwNWQ4ZGZhNmVjYzQzZjY5ZTliY2ZkMDFhOTQ0NWNlZTk3NjlhZjRhXCJdfSJ9>
is historic for scientific and financial reasons. Luxturna is the
first gene therapy approved in the U.S. that targets a disease caused
by mutations in a specific gene.
A Spark spokesman said the company will not disclose the Luxturna
price tag until early January. Wall Street analysts expect the gene
therapy to command a $1 million price tag — another first, but not
necessarily a welcome one. At a time when drug prices are coming under
intense scrutiny, Spark will need to convince insurers, politicians,
and pharma critics that the benefit to patients offered by Luxturna
justifies its high cost.
The Institute for Clinical and Econonic Review (ICER), an influential
non-profit that studies the cost effectiveness of new drugs, is
holding a meeting to review
Luxturna<https://mandrillapp.com/track/click/30489975/icer-review.org?p=eyJzIjoic1dqYTVLYnVwamtVTU1tOEhuTkRrTzlwUzMwIiwidiI6MSwicCI6IntcInVcIjozMDQ4OTk3NSxcInZcIjoxLFwidXJsXCI6XCJodHRwczpcXFwvXFxcL2ljZXItcmV2aWV3Lm9yZ1xcXC90b3BpY1xcXC92b3JldGlnZW5lLW5lcGFydm92ZWNcXFwvXCIsXCJpZFwiOlwiNWM2MjI4ZDI1YWFiNGU1YjkzNmZhYTFiNjhiNDUwMTdcIixcInVybF9pZHNcIjpbXCI0YjZkZGEyMjRiYTcwOTBkMGMzYTI1OWI0NmZmNzMxNzA4MmMzODdkXCJdfSJ9>
on Jan. 25.
Spark CEO Jeffrey Marrazzo called the FDA announcement a landmark
moment "decades in the making for the field of gene therapy."
"This one-time gene therapy for an inherited disease represents a
first-of-its-kind breakthrough that may lay the groundwork for the
development of gene therapies for other conditions that are not
adequately addressed today," Marrazzo said in a statement.
Like other gene therapies, Luxturna inserts a functional piece of DNA
into cells in order to replace or override a defective,
disease-causing gene. For Spark, the injection site is the eyes of
people, mostly children and young adults, who have a type of inherited
retinal disease caused by a mutation in a gene called RPE65. People
born with mutated RPE65 genes suffer from severe vision problems,
including night blindness. As the disease progresses, people lose all
functional vision and can eventually become totally blind.
Luxturna is not technically a cure. The surgical injection — one in
each eye — aims to halt disease progression and restore some
functional vision.
Wall Street analysts, on average, are forecasting 2018 Luxturna sales
of $78 million, growing to $238 million in 2019 and peaking at $445
million, according to Bloomberg. These estimates carry a
higher-than-normal degree of uncertainty, however, due to the
uncertainties about Luxturna's price and reimbursement.
Based in part on the Spark approval process, the FDA plans on
unveiling a "modern, comprehensive framework of how we'll advance the
field of gene therapy" in the coming year, FDA Commissioner Scott
Gottlieb told reporters.
The agency will release a series of guidance documents, he said, that
will give companies a better idea of how to create safe development
pathways, optimize clinical trial design, and develop meaningful
clinical endpoints. The documents will focus in on areas where there's
already a lot of interest in gene therapy — particularly single-gene
disorders — and help companies better use expedited regulatory
pathways, such as the FDA's breakthrough designation.
Beyond inherited disease, gene therapy is showing a lot of promise in
autoimmune disorders, cancer, diabetes, heart disease, and HIV/AIDS,
said Dr. Peter Marks, director of the FDA's Center for Biologics
Evaluation and Research.
"Still, we also have a good deal to learn about how these products
work, how to administer them safely, and whether they will continue to
work in the body without adverse side effects," Marks said.
Spark shares were up 4 percent to $50.82 following the FDA's announcement.