Though it’s been used off-label for more than three decades to treat cerebrotendinous xanthomatosis, Mirum Pharmaceuticals Inc.’s chenodiol gained an official U.S. FDA nod Feb. 21 as the first drug approved specifically for treating the rare autosomal recessive lipid storage disease.
Several Asia biotechs this week – including Innocare Pharma Ltd., Akeso Pharmaceuticals Inc., Sanbio Co. Ltd. and Ascletis Pharma Inc. – unveiled the start of new late-stage clinical trials or interim findings from early stage studies.
The U.S. FDA approved 12 drugs in January, falling below the 2024 monthly average of 19. Only three new molecular entities received approval, trailing the yearly average of just over four per month.
Once high-flying Bluebird Bio Inc. has found a way out of its financial squeeze, as funds managed by global investment firms Carlyle and SK Capital Partners LP, along with a team of biotech executives, will be taking over the company.
As the flurry of executive orders continues to flow from the White House, leaving in its wake chaos and confusion across the overall health care sector, Tim Opler, managing director of the global health care group at investment firm Stifel, is taking the long view and urging the biopharma sector to continue focusing on innovation.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Apellis, Arctic Vision, Biorestorative, Clearside, CSL, Genmab, Irlab, Polaritybio, Signet, Sobi.
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Aptose, Assembly, Medivir, Novadip, Sellas, Thetis.
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