Novartis in political row after denying Belgian toddler compassionate access to gene therapy

The controversy over Novartis’ ultra-pricey gene therapy Zolgensma has intensified after it allegedly refused to supply the one-off therapy to a sick toddler in Belgium on compassionate grounds.

According to The Brussels Times, the family of the toddler named Pia, who suffers from the ultra-rare disease spinal muscular atrophy (SMA) have been running a crowdfunding campaign to pay for treatment with the world’s most expensive drug.

Pia’s family have raised the 1.9 million euros needed to pay for Zolgensma, which has not yet been approved in Europe.

However, according to the report Belgium’s health minister Maggie De Block said the company had refused a request for compassionate use for Zolgensma (onasemnogene abeparvovec).

Compassionate use is allowed in EU laws under strict conditions, where governments can request that patients receive unauthorised medicines if there is no satisfactory alternative and they cannot enter clinical trials.

The Brussels Times reported that De Block’s spokesperson Audrey Dorigo said Novartis had rejected a request for compassionate use, even though the government considered that all conditions were met.

Dorigo told the website that the “refusal is not justified” and cited cases where other pharma companies have agreed to provide unapproved drugs under compassionate use arrangements.

Novartis has issued a statement, saying that it did not comment on individual patient cases, and adding that the drug had been priced “cost effectively” in the US based on Novartis’ analysis of the one-time transformative therapy.

The company added in a statement that “healthcare systems need to find new ways to organise care and provide coverage for treatments that provide a lifetime of benefits.

“As an industry, we are committed to maintaining and intensifying an open dialogue with all stakeholders, to find solutions together to bring new therapies to patients who can benefit in Belgium and elsewhere,” Novartis said.

Zolgensma is under review in Europe, but in July it emerged that the European Medicines Agency’s CHMP scientific committee had removed Zolgensma from an accelerated assessment programme.

The Committee for Medicinal Products for Human Use (CHMP) did not announce the reasoning behind the decision, which means the gene therapy will be reviewed in 210 days rather than the accelerated 150 days.

This paves the way for a potential approval in the final quarter of this year, or the first quarter of 2020 depending on how the review proceeds.

The company is also at the centre of an argument over Zolgensma in the US, where the gene therapy has been approved since May.

Last month politicians urged the FDA to take tough action against Novartis for failing to disclose that manipulated non-clinical data had been included in the Zolgensma filing dossier.

The information dates back to when the drug was in very early development by the biotech AveXis, before Novartis’ $8.7 billion takeover in April last year.