Mirum Pharmaceuticals, Inc.
https://mirumpharma.com
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Latest From Mirum Pharmaceuticals, Inc.
Orphan Drug, Domestic Developers Shine In China Annual Approvals Tally
10 orphan drugs imported from overseas, and 40 new drug approvals, including many from domestic developers, capped 2023 as one of the busiest years for China's drug regulatory agency, as Beijing pivoted to emphasizing affordable innovation and expanding access to newer therapies for rare conditions.
Breakthrough Bounceback Looks Unlikely At US FDA’s CDER, But CBER Is Riding Wave Of RMATs
Only two breakthrough-designated novel agents remain on the US FDA drugs center’s 2023 user fee calendar, while CBER stacks up on vaccines and gene therapies under both BTD and RMAT programs.
Ipsen Buoyant Despite EMA’s Bylvay Orphan Status Block
The French company plans to refile Bylvay for Alagille syndrome under a new brand name after the EMA's Committee for Orphan Medicinal Products said the drug did not meet its 'significant benefit' criteria.
Deal Watch: Sangamo Inks Significant Option Deal With Prevail After Losing Tie-Ups With Novartis, Biogen
Plus deals involving Novartis/DTx, Neurogene/Neoleukin, Travere/Mirum, Sanofi/Scribe, Korro Bio/Frequency, MaxCyte/viTToria and more.
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