Cell and gene therapies are offering patients potentially curative treatments for a growing scope of diseases. Insurance companies are trying to figure out how to pay for them. Industry consultants speaking at the HLTH conference offered some strategies they see payers taking to these new therapies.
The two deaths reported following treatment with Novartis’s Zolgensma mark the first fatal cases of acute liver failure associated with the gene therapy. Liver complications are a known side effect risk of Zolgensma and other genetic medicines that are delivered by engineered viruses.
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The company plans to enroll 60 patients who have received its drug, Spinraza, following suboptimal response to Novartis' Zolgensma, which was approved last year. The trial, RESPOND, is anticipated to start enrolling in the first quarter of 2021, pending regulatory approval.
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The hold places a pause on enrollment in the high-dose cohort of the company's Phase I study of intrathecal AVXS-101, a different administration for the marketed therapy Zolgensma. It does not affect the marketed product and is unrelated to an early data manipulation scandal.
The company posted strong sales for Zolgensma, which the FDA approved five months ago for treating infants with spinal muscular atrophy. Strong and growing sales were also reported for the breast cancer drug Piqray and the CAR-T cell therapy Kymriah.
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Interim Phase I/II data for Zolgensma showed improvements in children aged 2 to less than 5 with spinal muscular atrophy. The therapy received FDA approval in May for infants younger than 2.
The Aug. 23 document from AveXis, posted on the FDA website Tuesday, fingered "two AveXis senior executives" who had been terminated. Brothers Brian and Allan Kaspar were ousted from AveXis last month, though the former denied any wrongdoing.
Novartis subsidiary AveXis is presenting Phase III data at a European pediatric neurology conference on the therapy, Zolgensma, which won approval in May for spinal muscular atrophy in infants.
The payer unveiled Embarc Benefit Protection, which it said would enable plans to pay a monthly, per-member fee to enroll, while patients would not have to pay anything out of pocket.
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The Wall Street Journal reported that the agency was focusing on the two-month delay between the March 14 discovery of data manipulation by Novartis' AveXis unit regarding the therapy Zolgensma and its May 15 launch of a formal inquiry into the matter.
Subsidiary AveXis said the two executives being replaced had not been involved in operations since May and were no longer with the company. Another publication reported that they had been ousted in connection the scandal.
Five Democratic senators - two running for president - wrote in a letter to the FDA's acting commissioner that the scandal highlighted industry "privilege and greed."
Novartis CEO Vas Narasimhan said the data manipulation issue was confined to a disused potency assay for use in mice and predated the company's acquisition of AveXis, while trying to reassure analysts that the disclosure delay was for the sake of completing an internal investigation.
In a statement Tuesday, an FDA official wrote that subsidiary AveXis disclosed manipulation of mouse data for the gene therapy Zolgensma, which the agency approved in May for spinal muscular atrophy. The official wrote that civil or criminal penalties were possible.