17 Total results for product and free and sample content found
Europe’s patchwork of reimbursement agencies make it a daunting place for a small US biotech to do business. Traditionally large pharma partners or CMSOs have provided an experienced hand to hold, but as a new wave of gene therapies and orphan drugs get the EMA green light, smaller drug developers are applying innovation to commercialization and choosing to go solo.
Phase III results support early 2020 filings for what could be the RNAi specialist’s third commercial product, but two competing drugs for primary hyperoxaluria from Dicerna and OxThera aren’t far behind.
Updated guidance from the UK regulator, the MHRA, outlines the rules on orphan drug applications, criteria and exclusivity periods that will apply in Great Britain after the end of the Brexit transition period
Accrual difficulties force changes that nullify Special Protocol Assessment; ongoing, randomized FOCUS trial in patients with ocular melanoma that has metastasized to liver will become single-arm study and target enrollment will be reduced by 66%.
The European Commission's plans to evaluate the EU pediatric and orphan drugs regulation in tandem.
Topic Rare Disease
Using lessons learned from the first wave of biosimilar approvals – and citing high prices in the US – some developers are starting to 'think small' with biosimilars for rare disease markets. Alexion’s Soliris is a case in point.
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