First-Of-Its-Kind Gene Therapy Approved To Treat Blindness, But Sticker Price Predicted To Be Astronomical
The price of the treatment could be more than $1 million. But the Food and Drug Administration's approval of the treatment signals a new era in gene therapy because it is the first to target a disease caused by mutations in a specific gene.
The Wall Street Journal:
FDA Blesses Blindness Treatment That Could Cost $1 Million
The Food and Drug Administration approved for sale the first therapy in the U.S. that delivers a functional gene to replace a faulty, disease-causing one—a treatment that could carry a price in excess of $1 million, its maker has said. The injected gene therapy from Spark Therapeutics Inc. is designed to improve sight in people with a rare form of vision loss caused by an inherited genetic mutation. The condition, retinal dystrophy, often manifests itself in young children and affects up to 3,000 Americans, Spark said. (Loftus, 12/19)
The Washington Post:
FDA Approves First Gene Therapy For An Inherited Disease
In a historic move, the Food and Drug Administration on Tuesday approved a pioneering gene therapy for a rare form of childhood blindness, the first such treatment cleared in the United States for an inherited disease. The approval signals a new era for gene therapy, a field that struggled for decades to overcome devastating setbacks but now is pushing forward in an effort to develop treatments for hemophilia, sickle-cell anemia and an array of other genetic diseases. Yet the products, should they reach patients, are likely to carry stratospheric prices — a prospect already worrying consumer advocates and economists. (McGinley, 12/19)
Los Angeles Times:
FDA Approves Gene Therapy To Fix Mutations That Can Lead To Blindness
Tuesday's announcement marks the third time in five months that the drug safety agency has allowed a gene therapy — a form of treatment with a long and fitful safety history — on the U.S. market. The first approval went to Kymriah, which treats a form of leukemia, in August. In October, the drug agency cleared a second gene-based treatment called Yescarta to treat a form of lymphoma. "Gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses," Dr. Scott Gottlieb, the FDA's commissioner, said Tuesday. "We're at a turning point when it comes to this novel form of therapy." (Healy, 12/19)
Stat:
FDA Approves First Gene Therapy Targeting Rare Form Of Inherited Blindness
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. (Feuerstein, 12/19)
NPR:
Luxturna Approved By FDA To Treat Inherited Form Of Blindness
In tests on patients, the treatment often produced dramatic results, restoring the ability of patients to see things they could never see before, such as the stars, the moon, fireworks and their parents' faces. The treatment also enabled patients to do many things that had been impossible, such as read, play sports, ride bicycles and go outside at night by themselves. "Today's approval marks another first in the field of gene therapy," said FDA Commission Scott Gottlieb in a statement announcing the decision. "This milestone reinforces the potential of this breakthrough approach in treating a wide range of challenging diseases." (Stein, 12/19)
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