Novartis approval for gene therapy signals new cancer treatment era

Georgia Reed
August 31, 2017

The United States approved the first gene therapy in the nation on Wednesday - a treatment that uses a patient's own immune cells to fight childhood leukaemia.

The approval of Novartis' CAR-T therapy comes just two days after Gilead Sciences Inc. announced an agreement to spend $11.9 billion and buy Kite Pharma Inc., which is developing its own CAR-T therapy scheduled for an FDA ruling by November 29.

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", said FDA Commissioner Scott Gottlieb, who was appointed to the position by President Donald Trump earlier this year.

In another novel development Wednesday, Novartis plans to price the therapy, Kymriah, "based on the clinical outcomes achieved", and specifically only allow for payment when ALL patients "respond to Kymriah by the end of the first month". Within 3 months of treatment, the overall remission rate was 83%.

"We are so proud to be part of this historic moment in cancer treatment and are deeply grateful to our researchers, collaborators, and the patients and families who participated in the Kymriah clinical program", added Bruno Strigini, CEO of Novartis Oncology. The FDA also expanded approval of tocilizumab (Actemra) for the treatment of CAR-T cell-induced severe or life-threatening CRS in patients 2 years or older, according to the release.

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The disease is the most common type of childhood cancer in the US, making up 25 percent of cancer diagnoses for children under 15 years old, according the National Cancer Institute. The FDA said hospitals and clinics must become certified to distribute the treatment, meaning they are prepared to recognize and treat CRS and other potentially fatal neurological events. "Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease", Peter Marks, MD, PhD, direct of the Center for Biologics Evaluation and Research, FDA, said in the release.

Kymriah is a genetically-modified autologous T-cell immunotherapy.

The company announced it would be collaborating with the U.S. Centers for Medicare and Medicaid Services on CART-T therapy. Kymriah is approved for use in the most common form of the disease and is only for patients whose cancer hasn't responded to initial treatments.

In the main study that informed the advisory committee's decision in July, roughly half of 68 patients experienced high-grade CRS, though none died from it.

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