Swiss pharma firm could give away therapy for babies, but criticized over selection

Swiss pharmaceutical company Novartis is preparing to give away 100 doses of the world's most expensive drug, which treats a rare childhood disorder, but its recipient selection process has drawn criticism.

Swiss pharma firm could give away therapy for babies, but criticized over selection
Novartis headquarters in Basel. Photo: AFP

The company announced this week that starting next month, its AveXis unit will begin distributing doses of Zolgensma, a one-time gene treatment for spinal muscular atrophy, also known as SMA.

The disease affects about 1 in 10,000 births, and which results in death or the need for permanent ventilation by the age of two in 90 percent of cases.

Novartis said a total of 100 doses — which cost around $2 million a pop — would go to eligible patients who are “under the age of two and are a citizen or legal resident of a country where the therapy is not yet approved by regulatory authorities.”

The therapy was approved by US regulators in May, but approval in Europe and Japan for instance has been delayed until next year.

Novartis stressed in a statement that AveXis so far had only one facility licensed to produce the therapy, and that the company's first obligation was to provide it where it had been approved or was pending approval, as well as to clinical trials.

“We work diligently to get two more facilities licensed in 2020,” it said.

As for the giveaways, Novartis said the intention was for a long-term commitment, with additional doses added to the program on a rolling six-month basis based on patient need and the expansion of capacity.

Novartis said AveXis had worked with an independent bioethics advisory committee to develop the programme, which it insisted was “anchored in principles of fairness, clinical need and global accessibility to best determine the equitable global distribution of a finite number of doses.”

It would not favour any child or country over another, it said.

A third party would administer “a blinded selection” every two weeks from a pool of patients proposed by their treating physicians who had been proven to be medically eligible, it said.

Novartis stressed that patients not picked in one selection round would automatically be submitted to the pool of candidates for the next selection as long as they remained medically eligible.

But the process drew criticism.

TreatSMA, a British advocacy group for the disorder, praised Novartis for trying to increase access, but it said it had “yet to be convinced that a health lottery is an appropriate way of meeting the unmet medical needs in this severe disease.”

France's AFM-Telethon, which works to raise donations to find treatments for SMA and genetic disorders, went further, criticising the programme as a cruel “lottery” offered to “parents whose children afflicted with spinal muscular atrophy are condemned to death in the short term.”

“How can one envisage even for a second that the life of a child can be the big prize in a lottery,” the organisation asked on its website.

READ ALSO: Swiss pharma giant Novartis to splash nine billion euros on US firm

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Switzerland’s Novartis to help make Pfizer-BioNTech Covid vaccine

Swiss pharmaceuticals giant Novartis said Friday it had signed an initial agreement to help produce the Pfizer-BioNTech vaccine against Covid-19, as countries scramble to boost supplies.

Switzerland's Novartis to help make Pfizer-BioNTech Covid vaccine
Novartis will help manufacture Pfizer vaccine. Photo by AFP.

The rare act of cooperation — in an industry usually marked by cut-throat competition — comes after French pharma group Sanofi announced earlier this week that it would also team up with rivals Pfizer and BioNTech to help produce 125 million doses of their jab.

The two-dose vaccine, which is based on mRNA technology, has been shown to be around 95 percent effective and has been approved for use by the World Health Organization and in some 50 countries.

But it is in limited supply as nations around the world race to immunise their populations against the coronavirus, which has killed nearly 2.2 million people in just over a year.

Novartis said in a statement that it would use its sterilised manufacturing facilities at its site in Stein, Switzerland to help produce the Pfizer-BioNTech jabs.

Under the agreement, the company said it would “take bulk mRNA active ingredient from BioNTech and fill this into vials under aseptic conditions for shipment back to BioNTech for their distribution to healthcare system customers around the world”.

Once a final agreement is reached, Novartis said it expected to begin production in the second quarter of the year, with initial shipment of finished product expected in the third quarter.

Steffen Lang, Head of Novartis Technical Operations, stressed that the company was “committed to leverage our manufacturing capabilities to help support the supply of COVID-19 vaccines and therapeutics around the world”.

“We expect this to be the first of a number of such agreements,” he said in the statement.

Novartis said it was already in “advanced discussions” with a number of other companies about with other production tasks, including of mRNA, therapeutic protein and raw material production for Covid vaccines and therapeutics.