ZURICH, Dec 19 (Reuters) - Novartis aims to give away 100 doses of its $2.1 million-per-patient Zolgensma for spinal muscular atrophy (SMA) in 2020 in a free-drug programme that one patient group worried was a "health lottery" that could neglect some babies.
Starting Jan. 2, Novartis's AveXis unit which developed Zolgensma will set aside 50 doses of the world's costliest single-dose treatment through June for babies under 2 years old, Novartis said on Thursday, with up to 100 total doses to be distributed through 2020.
The programme would apply to countries where the medicine is not yet approved for the rare genetic disorder that affects only about 1 in 10,000 live births, but which can lead to death and profound physical disabilities.
Zolgensma, with sales of $175 million through September, won U.S. approval in May and has been touted as potentially curative for babies treated before symptoms begin.
But regulatory decisions in Europe and Japan have been delayed until 2020, curbing access that Novartis hopes to partially address with free medicine where such giveaways are allowed.
Families in Belgium, Hungary, Israel and elsewhere have launched crowd-funding programmes for Zolgensma treatment.
"AveXis' intention is for this to be a long-term commitment," a Novartis spokesman 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."
Zolgensma, hit by months of turmoil including data manipulation allegations and the suspension of a trial over safety concerns, is the second approved SMA treatment, after Biogen's Spinraza.
Rival Swiss drugmaker Roche is expecting approval for its investigational oral medicine risdiplam by May.
TreatSMA, a British SMA advocacy group, applauded Novartis's free Zolgensma initiative but had reservations about the programme in which an independent commission starting on Feb. 3 would conduct bi-weekly draws of eligible babies.
"Unlucky" patients not chosen would be entered into subsequent draws until their eligibility expired, the group 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," TreatSMA said, adding it is gathering feedback from other SMA groups before formulating a formal position.
TreatSMA added it was unlikely that British rules would let patients participate in Novartis's programme. (Reporting by John Miller; editing by Jason Neely)