Skip to main content

Advisors to FDA Recommend Approval of Gene Therapy for Blindness

Spark Therapeutics’s Luxturna would be the first approved therapy in the U.S. that replaces or repairs a defective gene inherited from one’s parents.









Update (December 19): The US Food and Drug Administration (FDA) today approved Luxturna for the treatment of an inherited form of vision loss. It is the first gene therapy approved in the U.S. to correct a specific disease-linked gene. “We’re at a turning point when it comes to this novel form of therapy,” FDA Commissioner Scott Gottlieb said in a statement. “I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses.”

A panel of 16 independent advisers to the US Food and Drug Administration (FDA) voted unanimously today (October 12) to recommend the approval of Luxturna, a gene therapy meant to treat Leber congenital amaurosis—a rare, inherited form of childhood blindness—and other retinal disorders, STAT News reports. If the agency agrees with the panel’s recommendation, Luxturna would be the first gene therapy aimed at correcting a congenital defect approved in the U.S.
The therapy, developed by Philadelphia-based Spark Therapeutics, is injected directly into the eye in a one-time treatment. Billions of virus particles deliver a functional copy of the RPE65 gene—which encodes a protein necessary for normal vision and is mutated in patients with certain vision disorders—to the retina. So far, more than two dozen children and adults with RPE65 mutations have been treated with Luxturna in the context of clinical trials, and some 93 percent of them have shown improved light sensitivity and functional vision. The effects are long lasting, with improvements continuing for at least four years in some patients that have been tracked that long.
“If my child or myself had this condition I would not hesitate for a moment getting treatment with [Luxturna],” Albert Maguire, a retinal disease specialist at Children’s Hospital of Philadelphia who led the Luxturna clinical trials, said at the advisory panel hearing today, according to STAT News.
If approved, Luxturna would be the first gene therapy that fixes an inherited genetic disorder in the U.S. (The official title of first approved gene therapy goes to Novartis’s CAR T-cell therapy, greenlighted this August.) The FDA must make its decision by January 12, 2018.
“This is what I believe medicine is going to be like for the next 20, 30, if not 50 years,” Spark CEO Jeff Marrazzo tells MIT Technology Review of these types of gene therapies. “I think this is the beginning of an age that is going to fundamentally change medicine.”

Comments

Post a Comment

Popular posts from this blog

The Secret Science 02:The 30 Most Disturbing Human Experiments in History

Disturbing human experiments aren’t something the average person thinks too much about. Rather, the progress achieved in the last 150 years of human history is an accomplishment we’re reminded of almost daily. Achievements made in fields like biomedicine and psychology mean that we no longer need to worry about things like deadly diseases or masturbation as a form of insanity. For better or worse, we have developed more effective ways to gather information, treat skin abnormalities, and even kill each other. But what we are not constantly reminded of are the human lives that have been damaged or lost in the name of this progress. The following is a list of the 30 most disturbing human experiments in history. 30. The Tearoom Sex Study Sociologist Laud Humphreys often wondered about the men who commit impersonal sexual acts with one another in public restrooms. He wondered why “tearoom sex” — fellatio in public restrooms — led to the majority of homosexual arrests in ...
1 comment
Read more

The Strange and Stranger Case of Wyndham Lathem

A Northwestern University plague researcher has been charged with a brutal murder. Here’s what we know about him. WIKIMEDIA,  TONY WEBSTER O n July 27,  The  Chicago Tribune   reported that there was an arrest warrant issued for  Wyndham Lathem , a microbiologist at Northwestern University. The crime Lathem would later be charged with was brutal—26-year-old Trenton James Cornell-Duranleau, whose body was found in Lathem’s apartment, had been stabbed dozens of times. But Lathem was nowhere to be found. As events unfolded over the following days, it became clear he had fled from Chicago to California with a second suspect, 56-year-old Andrew Warren, a University of Oxford employee from the United Kingdom visiting the states. Along the way, the two men apparently made an anonymous $1,000 donation in Cornell-Duranleau’s name to the Lake Geneva Public Library and another donation for $5,610 to a Chicago health center. Lathem had also sent a video to fa...
Post a Comment
Read more

Popular painkiller doesn’t have more heart risks than others, study claims

NEW ORLEANS — A long-awaited study on painkillers called nonsteroidal anti-inflammatory drugs, the most widely prescribed class of drugs in the world, has concluded that the three most commonly used carry a similar risk of cardiovascular complications. Yet critics say the study was too flawed to fairly compare them. Concerns about a type of NSAID called COX-2 inhibitors peaked in 2004 when the drug Vioxx was withdrawn from the market — a decision steeped in scandal because manufacturer Merck & Co had initially hidden data that would reveal the drug’s cardiovascular risks. A second COX-2 inhibitor, Pfizer Inc.’s Celebrex, was allowed to remain on the market with the condition that Pfizer conduct a study to prove that Celebrex was no worse than two older NSAIDs, naproxen and ibuprofen. The study lasted 10 years and enrolled more than 24,000 patients, but faced challenges. Doctors in European Union countries would not participate because they were worried a...
1 comment
Read more