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 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.
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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".
(Reporting by John Miller; editing by Jason Neely)
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