7. Zolgensma

Novartis
After quoting potential prices of up to $5 million, Novartis set Zolgensma's final sticker at $2.125 million, and some previously skeptical cost watchdogs blessed it. (Novartis)

Generic name: onasemnogene abeparvovec
Company: Novartis
Disease: spinal muscular atrophy
2024 sales estimate: $1.56 billion

When he took the reins at Novartis, CEO Vas Narasimhan set out on a mission to remake it as a “medicines company” powered by novel therapy platforms. One of those platforms is gene therapy, and Novartis shelled out $8.7 billion to acquire a specialist in the field, AveXis—and its then-unapproved therapy Zolgensma.

The spinal muscular atrophy gene therapy, previously called AVXS-101, came through with its FDA nod—for pediatric patients less than 2 years of age—in late May. It will challenge Biogen’s current standard of care, Spinraza, which raked in $1.72 billion in 2018 after a late-2017 approval. For Zolgensma, Evaluate predicts 2024 sales of $1.56 billion.

RELATED: Novartis slaps $2M-plus price tag on newly approved gene therapy Zolgensma

Ahead of Zolgensma's green light, executives hinted at a price in the range of $4 million to $5 million for the one-time treatment. Cost watchdogs at the Institute for Clinical and Economic Review (ICER), meanwhile, figured Zolgensma a $900,000 price to meet its standard cost-effectiveness threshold of $150,000 per quality-adjusted life year. Even using a less stringent measure, Zolgensma shouldn't cost more than $1.5 million, ICER said.

Novartis split the difference when the therapy won its approval, setting a final sticker at $2.125 million. And by then, ICER had weighed newly available numbers and changed its own verdict.

“[G]iven the new efficacy data for the presymptomatic population, the price announced today falls within the upper bound of ICER's value-based price benchmark range,” ICER President Steven Pearson, M.D., said in a statement released immediately after Zolgensma's green light.

RELATED: Awaiting FDA nod, Novartis touts latest data on gene therapy Zolgensma

Novartis also pointed out that the annualized cost of Zolgensma, $425,000 for 5 years, is 50% less than the 10-year current cost of chronic SMA therapy Spinraza.

Yet, as Biogen R&D chief Michael Ehlers, M.D., Ph.D., pointed out on the company’s fourth-quarter earnings call in January, data on the durability of Zolgensma remain limited. Researchers followed 15 Zolgensma patients for up to 2.5 years, while Spinraza has data on more than 300 patients followed for up to six years, according to Ehlers.

Novartis, for its part, is also offering payment models such as five-year outcomes-based pacts and pay-over-time options for payers.

RELATED: Novartis says it is ready to meet even 'unprecedented' demand for Zolgensma

The company has had some hiccups in producing its cell therapy Kymriah, but Narasimhan said Novartis is ready to turn out Zolgensma for as many patients as needed. 

The Swiss drugmaker now has more than 1 million square feet of manufacturing prepared to ramp up, the CEO said last month: “We’re continuing to build supply with the acquisition of a manufacturing site in Colorado,” referring to the recent deal for a former AstraZeneca plant in Longmont, Colorado, which has nearly 700,000 square feet of manufacturing space.

7. Zolgensma

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