Amid Zolgensma crisis, Novartis gets a gene therapy boost as NICE embraces Luxturna

In December 2017, Luxturna became the first directly administered gene therapy approved in the U.S. Now, it’s recommended for use on the NHS in England.

England’s drug cost watchdog, the National Institute for Health and Care Excellence (NICE), has recommended coverage for the one-time rare eye disease drug, which was developed by Spark Therapeutics and is sold by Novartis outside the U.S.

At its list price of £613,410 ($745,650) per patient, the drug would have failed the Budget Impact Test, meaning it would have cost the public health system too much, NICE said. However, like almost all other drugmakers do to win NICE backing, Novartis has offered NHS England a confidential discount.

"This latest deal reinforces the benefits for companies willing to engage with us and be pragmatic with their pricing—which is good news for patients, tax payers and industry," NHS chief executive Simon Stevens said in a statement.

In the U.S., the drug’s list price reaches $850,000. The Institute for Clinical and Economic Review (ICER), a U.S. cost watchdog, previously found it should cost 50% to 57% less to be considered cost-effective, accounting for direct healthcare system costs and other societal benefits.

On average, even within NICE’s Highly Specialized Technologies pathway, it would take the agency 38 weeks to wrap up an evaluation. But, eager to reach a deal, Novartis won over NICE in just 20 weeks.

"The company’s willingness to work with us early and constructively has allowed us to publish this guidance on a much faster timeline than normal which is good news for patients," Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE, said.

Novartis accomplished the feat without clinical data showing Luxturna could work in the long term, even though the lack of such evidence is a reason NICE frequently cites in rejecting coverage for a drug. Instead, the assessment committee "considered it is biologically plausible that the treatment effect is likely to continue for decades," the agency said.

Luxturna scored its EU approval last November, about 10 months after Novartis shelled out $105 million upfront and committed $65 million in milestones for its ex-U.S. rights. The one-time gene therapy is designed to treat people with certain inherited retinal dystrophy by delivering a functional copy of the RPE65 gene directly into the retina by a surgeon.

The company estimates that 86 people living with the condition will be eligible for the treatment through NHS. If no one appeals NICE’s draft guidance, the final version will come out next month, and coverage will start from January 2020.

Though Luxturna calls itself the first gene therapy available in the U.S., it’s not the first one covered by NICE. That title belongs to "bubble boy" drug Strimvelis, which GlaxoSmithKline offloaded to Orchard Therapeutics in 2018. The drug, which involves editing a patient’s own hematopoietic stem cell outside of the body, is not FDA-approved.

NICE’s backing is a timely win for the Swiss drugmaker and CEO Vas Narasimhan’s blueprint based on novel platforms, as the company is deeply embroiled in a data manipulation scandal around the spinal muscular atrophy gene therapy Zolgensma.  

The FDA in August unveiled that Novartis’ AveXis unit used falsified animal testing data in Zolgensma’s new drug application package. In a widely criticized move, the company confirmed the allegations internally in early May but didn’t alert the FDA until more than a month after it greenlighted the drug.

During an August conference call with analysts, AveXis President David Lennon said it had shared the information with the European Medicines Agency and doesn’t expect any delays to the discussions around approving Zolgensma in Europe. Novartis has said it expects an EU decision on the drug in the fourth quarter.

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