FDA halts Novartis’ Zolgensma gene therapy study due to safety concerns

The U.S. Food and Drug Administration (FDA) on Wednesday halted a clinical trial of Novartis’s Zolgensma gene therapy because of a safety issue identified during animal research, foreign media reported. The STRONG trial is believed to be aimed at testing larger doses of Zolgensma drugs injected into the spinal cord of older children with spinal muscular dystrophy (SMA). For approved infant and child treatment, this is not affected.

因安全隐患 FDA叫停了诺华的Zolgensma基因治疗研究

Screenshot of PDF document (from: Novartis, via Statnews)

Novartis Pharmaceuticals said its subsidiary, AveXis, informed regulators of the results of an animal study that it had “inflammation of the drG mononucleosis and occasionally accompanied neuronal cell degeneration or deactivation.”

The clinical significance of this poor safety signal is unclear, but the company added that it may be related to the ‘sensory effects’.

Novartis’s efforts to expand Zolgensma’s use have suffered another setback as the FDA halts ITS STRONG clinical trials, which the company had hoped to extend to treatment for elderly SMA patients.

Meanwhile, rival Biogen’s Spinraza treatment has been approved for older SMA patients, and Roche’s (RHHBY) clinical trial application for a new drug is expected to be approved next year.

But Novartis said it had not seen any reports of such adverse reactions and was actively working with the FDA to make a rigorous assessment of Zolgensma’s safety issues and restore the dose in clinical trials.

Infact, before the FDA’s action on Wednesday, there had been controversy over the manipulation of data used to support Zolgensma.

In an unusual case of accountability, the agency noted in August that AveXis knew that clinical data had been tampered with before the drug was approved in May, but delayed notifying the agency late.

The scandal sparked outrage among lawmakers, despite the FDA’s belief that the drug could remain on the market and Novartis CEO Vas Narasimhan’s pledge to act as soon as possible on the integrity of data disclosure.

Zolgensma, one of the world’s most expensive drugs, sells for up to $2.1 million.

Earlier this month, Novartis said it had been used to treat 100 patients since the gene therapy was introduced, bringing in $160 million in third-quarter revenue, beating analysts’ expectations.

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