Orphan Drug, FDA and Korro
KRRO-110 Receives Orphan Drug Designation from U.S. FDA for Alpha-1 Antitrypsin Deficiency
KRRO-110 is the first RNA editing development candidate from Korro’s proprietary OPERA TM platform and is currently being evaluated in the Phase 1/2a REWRITE clinical study for AATD. Dosing of the first two single ascending dose cohorts in healthy adult volunteers has been completed, and an interim readout is expected in the second half of 2025.
In 1983, Congress passed the Orphan Drug Act (“ODA”) as part of an effort to provide market incentives for pharmaceutical manufacturers to produce “orphan drugs,” drugs for diseases that ...
Treatments for autoimmune diseases and those in orphan markets are on the up and up, as are drugmakers’ first launches, ...
With orphan drugs typically being high value, the potential risk of stock losses should be a top concern for biotechs looking ...
GlobalData on MSN3d
Lundbeck’s amlenetug gains orphan drug status in Japan for MSALundbeck has gained orphan drug designation (ODD) from Japan’s Ministry of Health, Labor and Welfare (MHLW) for amlenetug to ...
The FDA granted fast track designation to ATSN-201 for the treatment of X-linked retinoschisis, according to a press release ...
The European orphan drug market is growing and is already comparable in size and scope to the US market. The European Union ...
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