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- FDA proposes new framework prioritizing consistency over flexibility for rare disease drug reviewsThe FDA announced a shift in its approach to evaluating rare disease therapies, emphasizing a more uniform assessment process. Agency officials explained that the flexibility shown over the summer…
- Ultragenyx sells second priority review voucher as Biomea shares tumble after oral GLP-1 dataUltragenyx Pharmaceutical, a commercial-stage rare disease company, completed its second sale of a priority review voucher this month. The voucher sale reflects the company's strategy of monetizin…