FDA expected to approve first cancer treatment using modern gene therapy

(FoxNewsHealth.com) FDA approval of a genetic therapy being developed by Moderna Therapeutics to treat a type of cancer could come as early as this week.

The Boston-based biotech company’s therapy, which is being developed for an inherited type of non-Hodgkin lymphoma, was originally called M923. It was approved earlier this year in Europe, but U.S. approval could come before the end of this week.

FDA is expected to issue its preliminary approval letter on Thursday, according to the Washington Post. In the letter, the agency said the treatment is promising but continues to explore ways to address potential side effects.

“It could be a groundbreaking product with both U.S. and global implications,” Les Funtleyder, a healthcare investor and former pharmaceutical industry analyst, told FoxNews.com. “It’s definitely a product for the future that we’ll be hearing about, watching and investing in. It can also potentially become a duopoly, based on the limited number of cancer therapies that exist today, and how quickly the disease can progress.”

Moderna’s M923 is an RNA interference, or RNAi, therapy designed to starve the cancer cells to death.

According to Moderna, the M923 treatment successfully shut down cancer cells that overexpress a type of gene called the RET gene. The company said the treatment led to improved survival, chemotherapy-free survival, early remission and reduction in cancer relapse over traditional chemotherapy alone.

“This drug puts us on the cusp of seeing all of these breakthroughs come true for millions of cancer patients who previously have had no hope,” Moderna Chief Executive Officer Stephane Bancel said in a statement, according to the Washington Post.

“Now is the time for cancer patients, doctors and researchers to open up their eyes to the opportunities that the future holds. Moderna is in a unique position to transform the road forward for the millions of patients with rare, serious and relapsing cancer.”

Nearly 30,000 to 50,000 cases of non-Hodgkin lymphoma were diagnosed in the U.S. in 2017, the National Cancer Institute estimated. About half of those patients were elderly, leaving about 12,000 to 15,000 young, healthy survivors, Moderna said. The median age in the U.S. for a first diagnosis of non-Hodgkin lymphoma is 69.5, according to Moderna.

The FDA approved a genetically engineered DNA treatment in November that was designed to help patients with a type of blood cancer. The therapy was approved for patients with chronic lymphocytic leukemia and defined as a relapse or treatment failure after chemotherapy, according to the company.

Moderna’s RNAi therapy would be the second treatment approved this year using modern RNA technology. Earlier this year, gene therapy from Crispr Therapeutics got U.S. approval to treat hereditary angioedema, a rare disease that causes severe itching and pain in people with the disease, according to the Washington Post.

“We’re finally seeing something come to market that uses this cutting-edge technology to deal with disease and I’m so excited to see more success in the future,” Funtleyder said.

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