Q&A with Ultragenyx CEO: Time to increase the use of accelerated approvals for rare disease drugs
Emil Kakkis, CEO of the rare disease drugmaker Ultragenyx Pharmaceuticals, sat down with Endpoints News today in Washington, DC to discuss industry’s more recent struggles around rare and ultra-rare disease drug development, the need for an Orphan Drug Act 2.0, and why the FDA needs to qualify more biomarkers and open up the accelerated approval pathway.
At least 30 rare disease drug programs have been canceled over the last year, Kakkis said, noting that the approval process has become more difficult in some cases, with FDA raising the bar, or the money is too much.
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