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Korro Bio received U.S. FDA's orphan drug designation for investigational medicine KRRO-110 to treat Alpha-1 Antitrypsin ...

The FDA granted orphan drug status to rhenium-186 obisbemeda for treating leptomeningeal metastases in lung cancer.

The global orphan drug market size was USD 154.20 Billion in 2022 and is expected to register a rapid revenue CAGR of 12.3 % during the forecast period. The global orphan drug market is witnessing ...

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 ...

Korro Bio disclosed that the Food and Drug Administration granted orphan drug designation to its potential genetic disorder drug KRRO-110. Shares rose 17.6% to $29 in premarket trading. The stock is ...

Plus Therapeutics (PSTV) soars with FDA's Orphan Drug Designation for Rhenium Obisbemeda, targeting rare leptomeningeal metastases.

EN001, a novel therapy under development by ENCell, has been granted Orphan Drug Designation by the U.S. FDA for the treatment of Charcot-Marie-Tooth (CMT) disease. Phase 1 clinical trial confirmed ...

The Ministry of Health, Labor, and Welfare (MHLW) in Japan has granted Orphan Drug Designation (ODD) to Lundbeck’s investigational drug, amlenetug, a potential new treatment option targeting Multiple ...

While in 2021, orphan drugs made up over half of all the US FDA’s approvals, with the figures in European being similarly high – 19 out of 54 approved new active substances.