Why FDA Panel Backed Novartis AG (ADR) (NYSE:NVS) Leukemia Drug

A group of cancer experts advising the FDA has asked the agency to approve a Novartis AG (ADR) (NYSE:NVS) leukemia drug currently going by the name CTL019. The drug is of the class of CAR-T therapies, which work by genetically altering a patient’s immune cells so as to make them more effective at tackling diseases.

Novartis’s CTL019 is designed to treat a severe form of leukemia that usually afflicts children and young adults. The FDA advisory committee that comprises cancer experts from across the U.S. voted unanimously, 10-to-0, to recommend approval of CTL019.

Though the drug carries some serious side effects, such as interfering with production of antibodies, the experts considered that its benefits outweigh the risks.

In clinical trials, 83% of patients who received CTL019 treatment were free of cancer after three months. The drug was tried on patients whose cancer had relapsed or didn’t respond to standard treatments.

Favorable risk-benefit balance

People afflicted by B-cell acute lymphoblastic leukemia, the type of blood cancer Novartis’s CTL019 is designed to combat, are usually treated with methods such as radiotherapy, chemotherapy and bone-marrow transplants. But these standard treatments do not work for every patient.

Patients who fail to respond to standard treatments have a low chance of survival – usually 10% to 20%. But Novartis’s CTL019 therapy raises the survival chance of these patients who have run out of standard treatment options to about 80%. In light of the drug’s side effects, the FDA advisory panel viewed this as a favorable risk-benefit balance.

Treatment with CTL019 involves removing some blood from the patient and modifying the T-cells in the blood before the cells are returned into the body. As such, the treatment is tailored for each patient.

Though the FDA is not tied to follow the advice of its panel of experts, it usually follows their recommendation. The FDA is expected to issue its final decision regarding CTL019 by the end of September.

Revenue potential

According to Jefferies analysts, CTL019 could generate $850 million in annual sales for Novartis by 2021.

Shares of Novartis rose 1.5% to $83.21 on Wednesday following the news of the positive recommendation on CTL019 by the FDA advisory panel.

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