Search results
The privileged status of orphan medicines in Germany
Pharmaceutical Technology via Yahoo Finance· 1 day agoThe German market has traditionally been appealing to orphan drug developers, taking advantage of the initial free...
FDA grants Medivir´s MIV-711 Rare Pediatric Disease Designation and Orphan Drug Designation for the...
FOX 23 News Albany· 2 days agoThe FDA defines a rare pediatric disease as a serious or life-threatening disease in which the...
Edgewise gets EU orphan drug status for muscular dystrophy drug (NASDAQ:EWTX)
Seeking Alpha· 4 days agoEdgewise Therapeutics (EWTX) has received orphan drug designation from EU regulators for sevasemten...
Edgewise's sevasemten earns EU orphan drug status By Investing.com
Investing.com· 4 days agoEdgewise Therapeutics, Inc. (NASDAQ:EWTX), a biopharmaceutical company focused on muscle diseases,...
Deverra Therapeutics Granted FDA Fast Track Designation for DVX101
Digital Journal· 3 days agoDesignation Validates Preliminary Data and Supports Accelerated Drug Development SEATTLE, WA / ACCESSWIRE / April 24, 2024 / Deverra Therapeutics, Inc., a clinical-stage biotechnology ...
Kura Oncology Gets Breakthrough Designation For Leukemia Therapy
Market Watch· 5 days agoThe San Diego-based biopharmaceutical company said the U.S. Food and Drug Administration had granted ziftomenib the status as a potential treatment for relapsed/refractory NPM1-mutant acute ...
Sobi® receives positive CHMP opinion recommending approval of efanesoctocog alfa for once-weekly...
FOX 5 San Diego· 1 day agoSobi® today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion recommending approval of efanesoctocog alfa, for ...
Medicure Receives US FDA Fast Track Designation for MC-1 for PNPO Deficiency
Morningstar· 4 days agoFood and Drug Administration (FDA) has granted Fast Track designation for MC-1, an investigational product for the prevention or treatment of seizures associated with pyridox ...
Novartis bags paediatric FDA label expansion for Lutathera
Pharmaceutical Technology via Yahoo Finance· 3 days agoTelix Pharmaceutical also expanded its radiopharmaceutical pipeline with the acquisition of QSAM...
Caliway Announced CBL-514 Phase 2 Study for Cellulite Treatment Met All Primary and Secondary...
WJTV Jackson· 2 days agoCBL-514 is under development for multiple indications, including Cellulite, Local Fat Reduction (currently in Phase 2b), and Durum's disease (currently in Phase 2), which brought Caliway the ...