Novartis plans giveaways of world’s most expensive drug
pharmafile | December 19, 2019 | News story | Business Services | Novartis, Zolgensma, sma
Novartis plans to giveaway 50 doses of the world’s costliest single-dose treatment, Zolgensma, for babies under 2 years old, with up to 100 doses being distributed throughout 2020.
Zolgensma is designed to treat spinal muscular atrophy (SMA) and costs $2.1 million-per-patient. It had sales of $175 million in September 2019. It is being touted as a curative treatment if used before the symptoms begin, and is from Novartis’s AveXis unit.
The giveaways apply to countries where medicine is not yet approved for SMA. The drug has been delayed in Europe and Japan until 2020, due to regulatory decisions, but Novartis are giving the medicine away for free to sidestep this.
Speaking about the giveaways, a Novartis spokesperson said: “AveXis designed a programme anchored in principles of fairness, clinical need and global accessibility to best determine the equitable global distribution of a finite number of doses that doesn’t favour one child or country over another.”
However there have been some reservations about the giveaway programme, with people labelling it a ‘lottery’ that will leave some babies exposed. TreatSMA, a British SMA advocacy group, approved of the free Zolgensma but had reservations about how the babies will be chosen.
It said: “Given the lack of access to any SMA treatment in many places, we are yet to be convinced that a health lottery is an appropriate way of meeting the unmet medical needs in this severe disease.”
SMA is a group of hereditary diseases that progressively destroys motor neurons, leading to muscle weakness and atrophy. It affects 1 per 8,000 to 10,000 people worldwide.
Conor Kavanagh
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