Novartis approval for gene therapy signals new cancer treatment era

Novartis won FDA approval for its CAR-T drug Kymriah on Wednesday

Novartis said it expects between 30 and 35 centers to be certified to offer the treatment by the end of the year. Of the 52 patients who had CR+ CRi, 29 were still in remission at the last assessment before the data cutoff. "There are a lot of patients who are getting long-term disease control, and we hope there is actually some permanence to their disease control from this one-time therapy". Median duration of response was not reached at a median follow-up of 4.8 months.

Novartis plans additional filings for Kymriah in the US and EU later this year, including applications with the FDA and European Medicines Agency, for the treatment of adult patients with r/r diffuse large B-cell lymphoma (DLBCL).

Carraway says the doctors giving the treatment should be experienced in managing cytokine release syndrome. In fact, after Novartis opened its doors to him, he got a second invitation from the organizers of next week's CAR-TCR Summit in Boston, a who's who of scientists and executives working in the field of cell therapies for cancer.

The FDA is including a boxed warning for CRS and, in an unusual move, also approved a new indication for the rheumatologic drug tocilizumab (Actemra) as a treatment for CRS.

The Kymriah approval came just over three months after the first patient to receive it, 12-year-old Emily Whitehead of Philipsburg, Pa., celebrated five years cancer-free in May. Also, CRS was among the most common adverse reactions in the trial, along with hypogammaglobulinemia, infections, pyrexia, decreased appetite, headache, encephalopathy, hypotension, bleeding episodes, tachycardia, nausea, diarrhea, vomiting, viral infectious disorders, hypoxia, fatigue, acute kidney injury, and delirium.

The approval of tisagenlecleucel "marks an important shift" in the treatment paradigm for hematologic malignancies, according to ASH President Kenneth C. Anderson, MD, director of Lebow Institute for Myeloma Therapeutics at Dana-Farber Cancer Institute.

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"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", FDA Commissioner Scott Gottlieb said in a press release. Children's Hospital of Philadelphia (CHOP) was the first institution to investigate Kymriah in the treatment of pediatric patients and led the single-site trial.

"While the importance of CAR-T can not be overstated, this approval only pertains to a small population of children", Anderson added.

Eleven patients who had CR+ CRi relapsed after tisagenlecleucel prior to data cut-off and before any new cancer therapy.

The FDA approved Novartis' Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia.

The therapy was produced "via pioneering technology and a sophisticated manufacturing process", she says - however, "We recognize our responsibility in bringing this innovative treatment to patients".

Disclosure: Grupp reports research support from Novartis.

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