Biogen’s Spinraza Needs Long-Term Confirmatory Data, Says EMA
The EMA may have quickly assessed the US biotech’s antisense spinal muscular atrophy therapy for Europe, but the product’s rapid clinical development means long-term data, and data on its effects in milder disease, or even cures, still need to be collected.
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Biogen’s Spinraza for the rare disease spinal muscular atrophy is to be assessed by NICE, the UK HTA body for England and Wales. In the interim it will be made available under a managed access deal. The product is also being assessed by Australia’s PBAC, which has asked for more cost-effectiveness data and may require a reduction in the company’s proposed price.
The German pharma options potassium channel modulator technology from Autifony and partners with Roche on immunological approaches to irritable bowel syndrome. Roche unveils discovery pacts with Confo and DiCE.
With BioMarin's Brineura hitting the market with a $702,000 annual price tag, recent experience for other high-priced rare disease drugs supports the theory that payers will accept high costs for ultra-rare pediatric disease therapies.