ZURICH (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 program 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 allocate 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 program applies to countries where the medicine is not yet approved for the rare genetic disorder affecting 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 Novartis hopes to partially address with free Zolgensma where such giveaways are allowed.

Families in Belgium, Hungary and Israel have launched crowd-funding programs for treatment.

“AveXis’ intention is for this to be a long-term commitment,” a Novartis spokesman said. “AveXis designed a program 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 favor one child or country over another.”

Novartis said manufacturing constraints — it has one licensed U.S. facility, with two plants due to come on line in 2020 — necessitated a focus on providing treatment to countries where the medicine is approved or pending approval.

Zolgensma, hit by turmoil including data manipulation allegations and suspension of a trial over safety concerns, is the second SMA treatment, after Biogen’s Spinraza.

Swiss drugmaker Roche is expecting approval for its medicine risdiplam by May.

TreatSMA, a British SMA advocacy group, applauded Novartis’s free Zolgensma initiative but had reservations about the program in which an independent commission would conduct bi-weekly draws of eligible babies. “Unlucky” patients not chosen would be entered into subsequent draws until 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,” TreatSMA said, adding it is gathering feedback before formulating a formal position.

TreatSMA added it was unlikely British rules would let patients participate in Novartis’s program.

Roche’s risdiplam is also set for a free drug program, including in India, helping extend SMA therapies to developing countries where high prices — Spinraza runs $750,000 in the first year and $375,000 thereafter — limit access.

Roche’s program for risdiplam, the price of which has not been disclosed, would initially target patients with type 1 SMA, the most severe form.

“The program will be expanded to patients with Type 2 SMA at the moment of filing of the regulatory application for risdiplam in each country,” Roche told Reuters, adding “not every country will initiate a program”.

[Source: Reuters Health]