FDA green lights two targeted cancer therapies, leading to optimism in genetic research and oncology

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HUNTSVILLE, Ala. -- Cancer cells are constantly evolving and mutating, meaning doctors and researchers have to continue to stay one step ahead of the genetic mutation.

What could be a 'breakthrough' in treating some forms of cancers just got accelerated approval by the Food and Drug Administration.

It's an exciting time in the world of oncology. "Most cancer therapies have been approved for a single cancer like breast cancer, or liver cancer. In this case, it's a treatment that's approved based on the genetic biomarker instead of the type of cancer," says Dr. Sara Cooper, a faculty investigator at Hudson Alpha.

The FDA's approval of two new cancer therapies -- Vitrakvi and Xospata has doctors and researchers like Cooper at Hudsonalpha optimistic.

Vitrakvi is "a treatment for adult and pediatric patients whose cancers have a specific genetic feature (biomarker)." Its accelerated green light came after major success including a two-year-old girl born with a tumor in her arm -- who is now cancer free.

"We were like where does this happen, it's a miracle," said her mother after receiving the drug twice a day showed immediate results, causing the tumor to shrink enough for doctors to operate without compromising function in her arm.

The drug targets solid tumors with an NTRK (neurotrophic receptor tyrosine kinase) gene fusion that do not have a known resistance mutation, that are not metastatic or where surgical removal is likely to lead to severe morbidity, and that have no alternative treatments or have progressed after treatments.

Xospata is a treatment for relapse adult patients of acute myeloid leukemia. Both drugs target a specific gene change that occurs in a tumor instead of where it is in the body. Alongside the Xospata tablets, the agency also approved a diagnostic to detect the mutation.

Cooper says the approval of these drugs helps set the tone for future genetic biomarker research. Because there is still much to learn and develop. "We're working really hard including here at Hudson Alpha to try to identify what are some of those biomarkers that we can use and develop more drugs that can help more and more people."

For now, doctors still rely on methods like chemotherapy or combining the therapies to help those with the disease -- something Cooper says is still successful.

Vitrakvi has been effective in 11 other cancers including lung and colon, but the price tags are steep at $11,000 a month for children and $32,000 for adults.
Bayer, who set the price for the drug, says the monthly out-of-pocket cost will be $20 a month. "Bayer will ensure that no eligible patient with TRK fusion cancer will go without this highly effective therapy," the company said in a statement. They say they plan to help patients pay the co-pays.

Both treatments were granted Priority Review designation.

Priority Review, established in 1992, means the FDA aims to review the drug or treatment within six months, opposed to 10 months for a standard review.

"A Priority Review designation will direct overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications," the FDA says.

Both treatments also received orphan drug designation, a status granted to drugs for rare diseases or conditions.

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