Mirum Pharmaceuticals, Inc.
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Latest From Mirum Pharmaceuticals, Inc.
While many of his peers elbowed each other to leap into oncology, James Xue decided to focus on rare diseases such as Hunter’s Syndrome. Now, 10 years after founding CANbridge, the CEO sat down for an audio interview with Scrip to offer his views on a flurry of key issues impacting orphan drug development in potentially the world's largest pharma market.
CDER hit a high mark with 27 first-in-class approvals in the 50 novel agents cleared in 2021.
CanBridge public listing is expected to generate much needed funding and spotlights a largely untapped but challenging segment.
Only three planned marketing authorization applications are known to have been granted accelerated assessment status by the European Medicines Agency between July and October this year.
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