Lundbeck has gained orphan drug designation (ODD) from Japan’s Ministry of Health, Labor and Welfare (MHLW) for amlenetug to ...

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 impact less ...

With orphan drugs typically being high value, the potential risk of stock losses should be a top concern for biotechs looking ...

The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Plus Therapeutics’ injectable ...

Treatments for autoimmune diseases and those in orphan markets are on the up and up, as are drugmakers’ first launches, ...

Plus Therapeutics (PSTV) announces that the U.S. Food and Drug Administration has granted Orphan Drug Designation to Rhenium Obisbemeda for the ...

The FDA granted fast track designation to ATSN-201 for the treatment of X-linked retinoschisis, according to a press release from Atsena Therapeutics.

PTC Therapeutics could boost revenue with vatiquinone for Friedreich's ataxia. Find out why PTCT stock is poised for growth ...

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