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The CTL019 Adcom Mists Begin To Clear

Jul. 11, 2017 11:40 AM ETNovartis AG (NVS)OXBDF2 Comments
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Briefing documents to guide the review of Novartis' (NYSE:NVS) US filing for CTL019 seem to have made at least one thing clear: the focus of the panel tomorrow will be on safety and manufacturing, and not on efficacy. The basic level of information provided suggests that Novartis might expect an easy ride.

Safety questions centre on some long-standing problems of gene therapies, as well as toxicities that have plagued CAR-T in particular, but the advice accepts that efficacy is beyond doubt. The good news for Novartis is that this is also in line with a physicians' survey carried out by EvaluatePharma.

This found that the US doctors - mainly Pediatric oncologists - thought the main concern for the panel would be CTL019's lengthy, complex and relatively unreliable manufacturing process, with toxicity in second place.

Meanwhile, the view was that efficacy would drive uptake, though reimbursement and availability of safer treatments would be the biggest constraints. The adcom will discuss the CTL019 construct, production and safety in the morning, before considering risk-mitigation and follow-up trials, and finally voting on CTL019's benefit-risk profile.

Readers are invited to register to follow a live blog, a collaboration between Stat and EP Vantage, taking place all day tomorrow.


Manufacturing issues are spelled out in the briefing documents, which state that the starting composition of T cells from apheresis is highly variable and not predictable. Given this lack of control "inter-patient variability in the manufacturing process" might have to be addressed, the documents suggest.

On a press call on Friday Novartis said it was continuing to optimize manufacturing, especially to minimize production failures and to ensure scalability. 30-35 hospitals will initially be used for cell apheresis and reinfusion, and production will take place solely at Morris Plains.

The Eliana trial that serves as the basis

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EP Vantage is a forward-looking comment and analysis service tailored to the needs of pharma and finance professionals, focusing on the events that will define the future of companies, products and therapy areas. Written by experienced journalists, EP Vantage provides timely financial analysis of regulatory and patent decisions, marketing approvals, licensing deals, and M&A, giving fresh angles and insight to both current and future industry triggers. EP Vantage is powered by EvaluatePharma, the industry leader in consensus forecasts.

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