Zolgensma pricing at fore as Novartis touts more than 25 potential blockbusters in pipeline

Novartis CEO Vas Narasimhan said Thursday that the company's pipeline contains "more than 25 potential blockbusters," including the spinal muscular atrophy (SMA) Type 1 gene therapy Zolgensma (onasemnogene abeparvovec). The executive noted at an analyst and investor event that the drugmaker "is on track to become a leading medicines company powered by advanced therapy platforms and data science, and is on track to deliver sustained accretive growth."

The FDA is expected to issue a decision on approval of Zolgensma later this month, with the price of the gene therapy eagerly anticipated. Novartis previously suggested that the therapy would be cost effective in a range of $4.6 million to $5.4 million, but Narasimhan indicated that "we want to be far lower than that."

Initial approval of Zolgensma is expected to be for use in infants, although the executive noted that Novartis is in discussions with the FDA about whether the label will include other forms of SMA beyond Type 1. The company is also in talks with commercial insurers in the US over the price of the treatment.

Along with Zolgensma, Novartis is preparing launches for possible blockbuster drugs over the next two years including Mayzent (siponimod), brolucizumab, ofatumumab and fevipiprant. Meanwhile, the company indicated that more than 10 potential blockbuster medicines could be launched by 2021. The drugmaker is also "capitalising on faster and broader access" in China, where it expects double-digit growth for its pharmaceuticals unit, with the aim of making the country its second largest market behind the US.

However, Narasimhan suggested that attractive takeover targets in China are scarce, citing a lack of novel drugs, concerns over data quality and lagging regulatory standards. The executive noted that many pharmaceutical companies in the country are focused on so-called "patented fast-follower" drugs, which mimic those discovered elsewhere. "We've yet to identify significant fundamental scientific innovation," Narasimhan said, adding "the CAR-T space is one where we're watching very closely. But I think there, the regulatory standards need to go up, and the data quality needs to go up, to actually have comparable data."

The CEO pointed out "it's not always clear how many lines of prior therapy a patient had, [or] is it really even a patient with the given disease." However, he indicated that "as those things get resolved in the five- to 10-year period, you will see a [mergers and acquisitions] uptick, but those are the barriers we see."

Meanwhile, Narasimhan also suggested that the company's financial target for its main prescription drugs business could be hit "a little sooner" than its current goal of 2022, boosted by new product launches and cost reductions. "Our goal right now is to get to the 35-percent [mark], and make sure that's a sustainable 35 percent," he said, having reported in the first quarter that core operating income of $2.9 billion rose to 33.3 percent of sales. "We'll see based on how the launches go. If they did (launch well), I think we would get there sooner, and our goal would be then to continue to push beyond that," the executive added.

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