Novartis reports increased sales for gene therapy Zolgensma but the failure in the second-line DLBCL setting will affect Kymriah sales.

Dan Stanton, Managing editor

April 27, 2022

3 Min Read
Novartis CGTs first quarter 2022: Zolgensma up, Kymriah down
Image: StockPhotoSecrets

Novartis reports increased sales for gene therapy Zolgensma due to expanded global access, but the failure in the second-line DLBCL setting will affect Kymriah sales.

For Swiss Pharma giant Novartis’ cell and gene therapy (CGT) division, first quarter 2022 results were mixed.

On the positive side, Zolgensma (onasemnogene abeparvovec) pulled in $363 million during the quarter, up 18% year-on-year due to increased access outside of the US. Sales of the one-time spinal muscular atrophy (SMA) gene therapy — approved in the US in May 2019 — are expected to continue to grow going forward, CEO Vasant Narasimhan said on his firm’s results call this week.

“The Q1 highlights were really the ex-US, where we had sales growth 32% in constant currency, while the US remains steady as we continue to drive up the newborn screening rates. So right now, we have over 2,000 patients treated worldwide, which I think demonstrates the profile of this gene therapy and the confidence providers are having using this medicine.

“In the future, our growth will be driven by continuing to penetrate in the US the under two, really getting to high market share. We expect to have over 90% of children who are diagnosed in newborn screening receiving Zolgensma, that’s our goal, and continuing to drive up that newborn screening in the EU above 25%.”

On the cell therapy side, Novartis’ chimeric antigen receptor (CAR) T-cell therapy Kymriah (tisagenlecleucel) fared less well.

Sales of the therapy were not divulged (Novartis only publishes revenue from its top 20 medicines, meaning Kymriah pulled in less than $132 million during the quarter) but Narasimhan was downbeat on Kymriah’s longevity on the call due in part to its failure in being approved as a second-line treatment for diffuse large B-cell lymphoma (DLBCL) last year.

“The failure of Kymriah in the second-line DLBCL is beginning to hit demand and I think we will see Kymriah to have less growth over the coming quarters and years and potentially even decline as our two competitors build out their second-line DLBCL program,” he told stakeholders.

Rival Kite/Gilead received US FDA approval earlier this month for its CAR-T therapy Yescarta (axicabtagene ciloleucel) to be used as a first-line DLBCL treatment, while Bristol-Myers Squibb’s Breyanzi (lisocabtagene maraleucel) has received European approval for the indication.

“So realistically for us in cell therapies, of course, Kymriah is the only medicine indicated in pediatric ALL [acute lymphoblastic leukemia],” said Narasimhan. “In the longer term, it really comes down to our next-generation T-Charge platform, which we provided data on at the end of last year at ASH, where we demonstrated pretty attractive data in both DLBCL and multiple myeloma.

“I think in the intervening years, it’s really just managing pediatric ALL to provide [Kymria] to patients,” he continued, adding the goal for Novartis’ CAR-T programs is to “really focus on the next-wave [T-Charge] technology, which we expect to have materially lower COGS, hopefully much higher throughput times, better efficacy and safety and enable the overall business to be significantly more attractive and more in line with other oncology agents in the company.”

About the Author(s)

Dan Stanton

Managing editor

Journalist covering the international biopharmaceutical manufacturing and processing industries.


Founder and editor of Bioprocess Insider, a daily news offshoot of publication Bioprocess International, with expertise in the pharmaceutical and healthcare sectors, in particular, the following niches: CROs, CDMOs, M&A, IPOs, biotech, bioprocessing methods and equipment, drug delivery, regulatory affairs and business development.


From London, UK originally but currently based in Montpellier, France through a round-a-bout adventure that has seen me live and work in Leeds (UK), London, New Zealand, and China.

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