Biogen/Ionis’s Spinraza Approved; Second Antisense Drug For Neurodegeneration In 2016
Analysts expect strong uptake for the antisense oligonucleotide (ASO) given the high unmet need in SMA and positive data in multiple studies, while the first ASO approved this year – Sarepta’s Exondys 51 – has seen some pushback from payers despite the lack of DMD therapies.
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Therapeutic areas and unmet needs drive big company decision-making about when and what technologies to bring in from external sources, executives explained during a panel at the 2020 Cell and Gene Therapy Meeting on the Mesa.
Some analysts see a 50-50 chance of US FDA approval despite the controversial dataset for the anti-amyloid antibody, but at least one expects an eventual CRL requesting a third Phase III trial.
The total cost of Zolgensma is more than $2m – in line with estimates – or $425,000 annually for five years if payers choose an annuity-like payment plan. Novartis is also negotiating value-based reimbursement agreements for infants treated with the one-time infusion.