Novartis touts record year for manufacturing changes, but Kymriah still an overhang
- Novartis on Wednesday noted 2018 was one of the most transformative years ever for the Swiss pharma's manufacturing footprint. Executives foresee more work ahead as the company attempts to strengthen production capabilities, particularly in the areas of cell and gene therapy.
- A barometer of whether Novartis is succeeding in those efforts will be Kymriah, a CAR-T cell therapy that Novartis expects to achieve blockbuster status. So far, however, the therapy has underwhelmed Wall Street as manufacturing challenges translated into weak sales. Kymriah sales during the fourth quarter totaled $28 million, which fell below some analysts' expectations.
- CEO Vas Narasimhan noted during the earnings call that Novartis announced 16 plant transformations over the last year, including eight plant exits. Collectively, the actions are "the biggest set of moves we've made on our manufacturing footprint," Narasimhan added.
Novartis sees a big market opportunity for Kymriah (tisagenlecleucel), but manufacturing hiccups have stood in the way.
"All the product we deliver to patients, be it commercial or investigational, we have the dose," Pascal Touchon, Novartis' head of cell and gene therapy, told BioPharma Dive during a November interview at the annual meeting of the American Society of Hematology. "The challenge is more around the percentage of viable cells."
The company has already received approval to expand cell viability specifications for Kymriah in Europe, and anticipates a similar approval in the U.S. soon. Securing regulator agreement should help to address the issues the pharma's had to date.
At the same time, Novartis has been building out its cell therapy production capacity. Susanne Schaffert, the new CEO of Novartis Oncology, noted on the recent fourth quarter earnings call that the company already has two Kymriah manufacturing sites in the European Union as well as partnerships in Asia. The goal for 2019 is to increase overall capacity fourfold.
Novartis’ manufacturing plans go beyond just cell therapy, however.
The company is also investing heavily in gene therapy, as evidenced by its $8.7 billion acquisition of AveXis last year. U.S. regulators are expected this year to make an approval decision on the lead candidate from that deal, the spinal muscular atrophy therapy AVXS-101.
Analysts see a high probability for approval, as does Novartis. The Swiss pharma is already in the process of ramping up its gene therapy production, with buildouts ongoing at plants in Chicago and North Carolina. "We're not losing any time," Narasimhan said on the earnings call.
Novartis conceded that there are still unknowns, and not just with the technology. Namely, the amount of product the company ends up making will directly correlate with how broad of a label regulators potentially give to AVXS-101.
"We work all scenarios as you'd expect. It will be a little bit label-dependent, but we're positive that we can be prepared for whatever need that presents itself," Narasimhan said.
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