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Source: Thailand Medical News  Dec 21, 2019  1 year ago
Pharma Giant Novartis Does Noble Deed Of Giving Away For Free Costly Therapy For Babies With Spinal Muscular Atrophy
Pharma Giant Novartis Does Noble Deed Of Giving Away For Free Costly Therapy For Babies With Spinal Muscular Atrophy
Source: Thailand Medical News  Dec 21, 2019  1 year ago
Novartis, the Swiss pharmaceutical company is preparing to give away 100 doses of the world's most expensive drug, which treats a rare childhood disorder, through a special recipient selection process.

The pharmaceutical giant 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 pediatric 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.

According to Novartis, a total of 100 doses which cost around US$2 million a dose 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 new therapy was approved by US regulators in May 2019, but approval in Europe and Japan for instance has been delayed until next year.

In a media statement, Novartis stressed 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.

A spokesman told Thailand Medical News, "We are working diligently to get two more facilities licensed in 2020 but sometimes a country government’s bureaucracy slows things down.”

About for the free 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.

The pharmaceutical industry leader, 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.

Novartis said that 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.

Novartis also 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.


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