FDA approves gene therapy for a type of blindness

The US Food and Drug Administration has approved a gene therapy treatment for patients with a rare inherited eye dise...

Posted: Dec 21, 2017 11:47 AM
Updated: Dec 21, 2017 11:47 AM

The US Food and Drug Administration has approved a gene therapy treatment for patients with a rare inherited eye disease.

Voretigene neparvovec, which will be sold as Luxturna, is made by Philadelphia-based Spark Therapeutics Inc.

Luxturna is the first gene therapy treatment for an inherited genetic mutation

The treatment is set to be available in the first part of 2018

The one-time treatment is approved for children and adults with retinal dystrophy due to a mutation of the RPE65 gene, which causes severe visual impairment beginning in infancy. As it progresses, patients experience gradual loss of peripheral and central vision, which can eventually lead to blindness.

There are more than 200 genes that can cause retinal dystrophy. Patients can be tested to determine whether the cause is indeed a mutation of the RPE65 gene and they are thus a potential candidate for this new treatment.

Luxturna is only the third gene therapy approved for use in the United States, preceded by two others this year. However, this is the first to correct an inherited genetic mutation. Some scientists believe that the approval could open the door to other treatments that correct flaws in the inherited genome.

FDA Commissioner Dr. Scott Gottlieb called Tuesday's approval a milestone for gene therapy and noted the potential for treatment of many diseases.

He said the approval "marks another first in the field of gene therapy -- both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss."

Adding a third gene

Luxturna supplies a normal RPE65 gene to retinal cells. It is a liquid that is injected into the eye with a microscopic needle during a surgical procedure. Within the liquid is a gene therapy virus that contains a healthy version of the gene.

Essentially, the drug adds a third version of the gene, which, unlike the mutated genes, is able to code for a protein crucial to vision.

Risks of the treatment include cataracts, elevated eye pressure, retinal tears and holes, and inflammation.

Researchers treated 41 patients between 2007 and 2015 to establish safety and efficacy. Those patients are still being followed. In a separate trial, researchers treated 31 patients and measured changes in their vision based on their ability to complete an obstacle course in low-level light.

The treatment is not permanent, according to William Hauswirth, an ophthalmology professor at the University of Florida College of Medicine who worked on developing it. He said most patients' vision regresses to what it was before therapy after about six years later, but he still considers it successful.

'This is incredible!'

Perhaps the most well-known patient from clinical trials of Luxturna is singer Christian Guardino, who appeared this year on season 12 of "America's Got Talent." The 17-year-old from Patchogue, New York, was eliminated from the show in the semifinals but told his mother, Elizabeth, that the ability to walk unaided onto the stage and see faces in the audience gave him confidence.

In October, he told an FDA advisory committee that the first 12 years of his life were spent in darkness, but he is now able to see things he hadn't seen before, thanks to the treatment.

On Facebook, he called the new approval "the best Christmas gift ever" and said, "this is just incredible!"

When the drug becomes available in early spring, it will become the first approved treatment option for the condition. An estimated 1,000 to 2,000 people in the United States stand to benefit from this option, according to Spark.

The company said cost information and details on timing will be released next month.

Indiana Coronavirus Cases

Data is updated nightly.

Cases: 663511

Reported Deaths: 12633
CountyCasesDeaths
Marion908511647
Lake48510882
Allen35951638
Hamilton32138398
St. Joseph30096513
Elkhart25423417
Vanderburgh21283379
Tippecanoe20096203
Johnson16380360
Porter16009272
Hendricks15855300
Clark11999181
Madison11764319
Vigo11636230
Monroe10363163
Delaware9854179
LaPorte9790197
Howard9075198
Kosciusko8574111
Bartholomew7479147
Warrick7430151
Hancock7418132
Floyd7226170
Wayne6645192
Grant6437157
Boone610389
Morgan6103125
Dubois5923111
Dearborn549068
Cass545899
Marshall5432105
Henry542593
Noble510178
Jackson465167
Shelby461490
Lawrence4189113
Gibson401781
Harrison401064
Clinton396553
Montgomery388483
DeKalb386478
Miami357663
Knox357585
Whitley349837
Huntington346277
Steuben339255
Wabash332376
Putnam331159
Ripley327762
Adams323949
Jasper317043
White297452
Jefferson295474
Daviess285596
Fayette271956
Decatur271088
Greene261680
Posey261431
Wells258575
Scott251250
Clay241544
LaGrange241270
Randolph225576
Spencer218030
Jennings215744
Washington212127
Sullivan203639
Fountain201842
Starke188451
Owen182853
Fulton179337
Jay178328
Carroll176519
Perry173435
Orange171150
Rush165322
Vermillion160842
Franklin159535
Tipton146841
Parke139416
Pike127833
Blackford120727
Pulaski106744
Newton96632
Brown95139
Benton92213
Crawford91013
Martin80214
Warren75814
Switzerland7558
Union67310
Ohio53911
Unassigned0433

Ohio Coronavirus Cases

Data is updated nightly.

Cases: 972605

Reported Deaths: 16750
CountyCasesDeaths
Franklin1123671214
Cuyahoga961861832
Hamilton735721034
Montgomery47359903
Summit40384802
Butler35577505
Lucas35489685
Stark29456803
Warren22464271
Lorain22068414
Mahoning19509537
Lake18445318
Clermont18425201
Delaware16499116
Licking15022189
Fairfield14563180
Trumbull14369413
Greene13595218
Medina13423226
Clark12288248
Wood11575163
Portage11050164
Allen10793216
Richland10316183
Miami10035189
Muskingum8195112
Columbiana8151200
Pickaway8067107
Tuscarawas8054227
Marion7996125
Wayne7886193
Erie6914140
Ross6152128
Geauga6083136
Hancock6006116
Ashtabula5978152
Scioto596280
Lawrence527118
Union513537
Darke5036114
Belmont496659
Huron4825107
Jefferson4806107
Sandusky4780108
Seneca4702107
Washington465791
Athens465047
Mercer458780
Auglaize456180
Shelby441679
Knox4037101
Putnam399390
Madison393553
Fulton380658
Ashland380581
Defiance372583
Brown369844
Crawford358396
Logan355772
Preble353678
Clinton340852
Highland328349
Ottawa324369
Williams302662
Jackson290545
Champaign288149
Guernsey287844
Perry269847
Fayette269141
Morrow260334
Henry246658
Hardin245858
Holmes243293
Coshocton237454
Van Wert229854
Gallia222631
Adams217036
Pike215626
Wyandot210447
Hocking194554
Carroll181341
Paulding160530
Meigs135730
Noble129233
Monroe117027
Morgan101119
Harrison100624
Vinton76613
Unassigned00
Fort Wayne
Partly Cloudy
37° wxIcon
Hi: 41° Lo: 29°
Feels Like: 30°
Angola
Mostly Cloudy
34° wxIcon
Hi: 38° Lo: 27°
Feels Like: 25°
Huntington
Partly Cloudy
37° wxIcon
Hi: 41° Lo: 28°
Feels Like: 28°
Fort Wayne
Mostly Cloudy
37° wxIcon
Hi: 41° Lo: 29°
Feels Like: 30°
Lima
Cloudy
32° wxIcon
Hi: 40° Lo: 28°
Feels Like: 22°
Sunny, Cooler Thursday
WFFT Radar
WFFT Temperatures
WFFT National

Community Events