Novartis intends to give away 100 doses of its $2.1 million-per-patient Zolgensma for spinal muscular atrophy (SMA) next year in a free-drug program that one patient group worried was a “health lottery” that would miss some babies.
Starting January 2, Novartis’s AveXis subsidiary, which produced Zolgensma will allocate 50 doses of the world’s costliest single-dose treatment via June for babies under 2 years old, Novartis mentioned Thursday, with as much as 100 total doses to be distributed by 2020.
The program applies to nations where the medication isn’t yet accepted for the rare genetic disorder affecting 1 in 10,000 live births, however, which might result in death and profound physical disabilities.
Zolgensma, with sales of $175 million via September, received a U.S. license in May and has been promoted as potentially curative for infants treated before symptoms start.
However, regulatory decisions in Japan and Europe have been delayed until 2020, curbing access Novartis hopes to address with free Zolgensma where such giveaways are permitted partially.
Households in Belgium, Hungary, and Israel have launched crowd-funding programs for therapy.
Novartis stated manufacturing constraints — it has one authorized U.S. plant, with two factories due to available in 2020 — necessitated a focus on providing treatment to nations where the drugs are accepted or awaiting approval.
Zolgensma, hit by turmoil along with data manipulation allegations and suspension of a trial over safety issues, is the second SMA treatment, after Biogen’s Spinraza.
TreatSMA, a British SMA advocacy organization, lauded Novartis’s free Zolgensma initiative, however, they had reservations about the program wherein an independent fee would conduct bi-weekly draws of eligible infants. “Unfortunate” sufferers not chosen would be entered into next draws until eligibility expired, the group stated.