Speaking of gene therapy, is it a good business?
While the public discussion of gene therapy tends to focus on how much they cost, the anxiety in biotech circles is a bit different. The products are expensive to make, complicated to administer, and usually treat tiny patient populations. Is anyone going to make money on these things?
We’ll get a useful hint this morning from Novartis, which is slated to report its earnings and by extension the sales for Zolgensma, a gene therapy for spinal muscular atrophy. Novartis’s product quite famously costs $2.1 million for a one-time dose, and it has also shown an unprecedented benefit for children with the most severe form of SMA.
On paper, Zolgensma’s combination of efficacy and convenience would make it the treatment of choice for some SMA patients, preferable to Biogen’s Spinraza, which is taken every few months for life and costs about $375,000 a year. But in practice, getting the health care system on board with a large one-time cost might prove difficult.
And thus the commercial fate of Zolgensma will be keenly watched by the likes of Bluebird Bio, BioMarin, Audentes and anyone else hoping to make a profit on one-time treatments.
We’ll get a useful hint this morning from Novartis, which is slated to report its earnings and by extension the sales for Zolgensma, a gene therapy for spinal muscular atrophy. Novartis’s product quite famously costs $2.1 million for a one-time dose, and it has also shown an unprecedented benefit for children with the most severe form of SMA.
On paper, Zolgensma’s combination of efficacy and convenience would make it the treatment of choice for some SMA patients, preferable to Biogen’s Spinraza, which is taken every few months for life and costs about $375,000 a year. But in practice, getting the health care system on board with a large one-time cost might prove difficult.
And thus the commercial fate of Zolgensma will be keenly watched by the likes of Bluebird Bio, BioMarin, Audentes and anyone else hoping to make a profit on one-time treatments.
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