AveXis rules out EU compassionate use of SMA gene therapy, says Biogen option is available

Novartis’ AveXis unit has given its reasons for refusing to supply its spinal muscular atrophy gene therapy Zolgensma to a seriously ill child in Belgium, saying compassionate use rules do no apply as Biogen's rival Spinraza (nusinersen) is an alternative option.

As pharmaphorum reported earlier this week, this one-off gene therapy for the rare muscle wasting disease is not yet approved in Europe, where it is being reviewed by regulators.

But there is a national campaign to make Zolgensma available to the toddler named as Pia, whose family has raised the cash needed to pay for the drug via crowdfunding.

Already approved in the US, Zolgensma is the world's most expensive drug, with a price tag of more than $2.1 million, which Novartis argues is justified given the lifelong benefits the therapy can bring.

A spokesperson for AveXis said that thanks to the crowdfunding effort the family and hospital concerned can now pay for the Zolgensma to be imported from the US, and use the therapy before a regulatory decision from the European Medicines Agency.

“They can import the normal licensed product from the US. The Belgian government will approve that and there are no contra-indications for the treatment of this child.

“We at AveXis are doing our best to make the treatment available and we are looking at the best possible way to treat the patient.”

The company also gave its reasoning for denying a request to supply the treatment on compassionate grounds.

Under EU laws pharma companies can supply unlicensed medicines to patients for free under compassionate use arrangements, when a condition is serious and there are no other authorised alternatives.

These conditions do not apply, according to AveXis, as Biogen’s Spinraza (nusinersen) has already been given the green light by regulators in Europe, in a decision that covers Belgium.

“You can only start compassionate use when there is no adequate treatment available. For this SMA population there is an adequate treatment available and reimbursed. There are a lot of children treated with nusinersen in Belgium.”

“There is no life-threatening situation in most of the countries in Europe.”

EU decision expected new year, US data row ongoing

The AveXis spokesperson added that a decision from Europe’s CHMP is expected early next year following a decision to adopt a standard review lasting 210 days, rather than the accelerated review lasting 150 days.

“We agreed with the EMA in a collaborative agreement that fast track is now a standard procedure. That gives the EMA the right time to review our answers on the list of outstanding questions they have sent to us.

“We are expecting European approval at the beginning of next year. I can’t explain the details about the dialogue we have with the EMA but it’s a mutual agreement related to a robust review.”

In a separate development, Novartis has been firefighting in the US, where it faces a probe into manipulated data that were included in the dossier filed with the FDA.

Novartis blamed AveXis co-founders Brian and Allan Kaspar for manipulating the preclinical data, before the Swiss company acquired the biotech last year.

The two brothers were fired last month, and in a remediation plan Novartis has set up a whistleblower scheme across AveXis to allow reporting of similar incidents.

The AveXis spokesperson would not comment about the matter during the interview with pharmaphorum.