FDA Approves Leber’s Congenital Amaurosis Gene Therapy to Potentially Cure Blindness

Orange County, CA - December 21st 2017 -  Inherited blindness is a rare mutation that gradually extirpates normal vision.  According to Spark Therapeutics’ website, more than 220 genes are responsible for the inheritance of retinal diseases. On Thursday, the FDA approved a gene therapy which could improve or save the vision of those who have inherited the rare genetic disorder called Leber’s Congenital Amaurosis (LCA).

"This is the first time that a genetic therapy has been used to treat an inherited disease in the United States," CBS News medical contributor Dr. Tara Narula told "CBS Evening News" anchor Anthony Mason. "This disorder we are talking about, LCA, is a disorder that has no treatment. It has no cure."

"People can really only see in very bright lights," she continued. "They have blurry vision and eventually lose their vision and become extremely visually impaired over time." The flawed gene resulting in the disease can be passed down for generations undetected. LCA only occurs when a child inherits a copy of the altered gene from both parents. Only a few thousand people in the U.S. are thought to have the condition.

The new therapy, an injection called Luxturna, is the first therapy approved by the FDA in which a corrective gene is given directly to the patient through an injection in each eye. The damaged gene blocking the retina’s conversion of light into electric signals is replaced with a working gene to allow the signals to reach the brain without hindrance.

"Today's approval marks another first in the field of gene therapy- both in the[therapy’s process and its expansion] beyond the treatment of cancer to the treatment of vision loss- and this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases," FDA Commissioner Scott Gottlieb, M.D., said in a statement. "I believe gene therapy will become a mainstay in treating, and [possibly] curing, many devastating and intractable illnesses."

With decades of research being translated into marketable treatments, the FDA has approved three gene therapies. The alternative treatment options cost an average of$400,000. Spark Therapeutics has yet to announce how much the Luxturna injections will cost, however, internal analysts at Spark valued the drug at $1,000,000. This evaluation was made under the assumption that Luxturna will only be given once and last a lifetime. In patient trials, individuals who that received the injections 4 years ago have yet to see any deterioration in their vision.

"What this really does is open the door for treatment of other inherited retinal disorders in terms of progressive scientific development and research," Dr. Narula said.  "Bottom line, it gives independence to people- it allows them to go to school, to work- and to return to normal." 

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FDA Approves Leber’s Congenital Amaurosis Gene Therapy to Potentially Cure Blindness Orange County, CA – December 21st 2017 –  Inherited blindness is a rare mutation that gradually extirpates normal vision.  According to Spark Therapeutics’ website, more than 220 genes are responsible for the inheritance of retinal diseases. On Thursday, the FDA approved a gene therapy which could […]