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Novartis’ Zolgensma hits regulatory hurdle after FDA asks to review another study

Published on 24/09/20 at 12:45pm
--Andrew- from Flickr

The FDA has requested an additional study in older children being treated with Novartis’ Zolgensma in the review process for its spinal muscular atrophy drug. 

Rather than accept their filing based on a Phase 1/2 trial, the FDA wants a Phase 3 study of Zolgensma infused into the spinal fluid rather than intravenously like in previous studies.

This follows the FDA halting enrollment into a Phase 1/2 trial back in October, due to preclinical tests flagging up a safety problem related to spinal-cord inflammation and neuronal cell degeneration. However, this is not related to the request for a new study. 

The drug is already approved for infants up to two years old with the rare genetic condition, and the company has said this review would have no impact on this prior approval. But the request for another study will delay the potential approval to at least late 2023. Novartis wants to expand this treatment so children from between the ages of two and five can access the drug. 

Novartis said: “The FDA has acknowledged the potential of [Zolgensma] in this patient population and recommends a pivotal confirmatory study to supplement.” 

The pharma firm had spent $8.7 billion to acquire Zolgensma from AveXis, which is the world’s most expensive one-time treatment.

Conor Kavanagh

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