American regulators granted approval to a gene-altering therapy for treating patients suffering from lymphoma, the second-ever approval for a gene therapy procedure and one that could revolutionize how we fight cancer.
The drug, named Yescarta, was developed by the Israeli- founded company Kite Pharma and is expected to cost about $373,000 for each patient, likely generating $150 million-$250m. in sales next year.
Kite was purchased by Gilead Sciences in August for $11.9 billion, and Wednesday’s breakthrough may have helped with the acquisition.
The price of Yescarta is set below that of comparable treatments, according to Reuters, as Novartis AG’s gene therapy costs $475,000.
The Novartis treatment, given the regulatory nod in August, is effective against childhood leukemia.
The high price of the drugs, called CAR-Ts, may set off a debate about the bioethical implications of pricing life-saving pharmaceuticals out of the reach of most patients.
What distinguishes the Israeli- developed drug from other pharmaceuticals is that each dose offers a personalized treatment made directly from the patient’s own immune system to fight the lymphoma – white blood cell cancer.
Called immunotherapy, the patient’s infection-fighting T-cells are extracted and genetically engineered with a new gene to pinpoint and kill the lymphoma cells.
After the cells are modified, they are infused back into the patient, where they patrol for years.
Since Kite Pharma and its parent company Gilead are based in California and publicly- traded on NASDAQ – as opposed to being headquartered in Israel and traded on the Tel Aviv Stock Exchange – the local benefit from the sale is indirect.
It is possible that the government and local universities may share some of the technologies and license royalties, according to pharmaceutical analyst Sabina Levy of Leader Capital Markets.
Funding difficulties may have initially forced Kite Pharma and its team to relocate to the United States in an attempt to attract investors.
“There is no doubt that there’s a huge potential in the Israeli biotechnology industry, and we can probably see more things coming from local scientists,” Levy said.
“If [Kite Pharma] had more significant financial support, then we could see more things happening here, remaining here and not trying to go abroad.”
The company’s drug is only the second gene-altering treatment approved by the FDA and the first for treating certain types of non-Hodgkin lymphoma, and for those suffering from large B-cell lymphoma.
The drug will help treat patients for whom two kinds of initial treatment failed. The complete remission rate after being treated with Yescarta is 51%, much higher than comparable clinical trials.
The treatment does, however, have life-threatening side effects.
“Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases.
“In just several decades, gene therapy has gone from being a promising concept to a practical solution [for] deadly and largely untreatable forms of cancer,” said FDA Commissioner Scott Gottlieb in a statement.
The treatment was first developed at the National Cancer Institute, led by Dr. Steven Rosenberg, who signed an agreement with Kite Pharma in 2012.
Kite helped fund the research process, in return for receiving patent royalties.
Arie Belldegrun, the Israeli- American founder of Kite Pharma, referred to the promising research when commenting on Gilead’s purchase of Kite in August.
“From the release of our pivotal data for Axi-Cel [Yescarta] to our potential approval by the FDA, this is a year of milestones,” he said.
“Each and every accomplishment is a reflection of the talent that is unique to Kite,” The Jerusalem Post previously reported.
Diffuse large B-cell lymphoma is the most common type of the disease in adults, with some 72,000 patients diagnosed annually in the US according to the American Cancer Society. Some 20,000 will die from it this year.
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