The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) and rare paediatric disease designation (RPDD) to Arbor Biotechnologies' gene editing therapeutic, ABO-101, to treat ...

On January 7, 2025, the FDA granted priority review to the new drug application for sunvozertinib (DZD9008), an oral EGFR ...

CEO Ken d'Entremont highlighted the successful FDA approval of GRAFAPEX, the branded name for treosulfan for injection in the U.S., which holds Orphan Drug Designation, ensuring at least seven years ...

Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that safety and efficacy ...

Ariceum Therapeutics is a private, clinical stage radiopharmaceutical company focused on the diagnosis and precision treatment of certain neuroendocrine and other aggressive, hard-to-treat cancers.

RenovoRx's innovative RenovoCath and TAMP platform show promising clinical data. Click here to read why RNXT stock presents ...

Neurizon (ASX:NUZ) has officially snagged a U.S. patent for its neurodegenerative disease and cancer drug candidate NUZ-001, ...

Quince Therapeutics, Inc. (Nasdaq: QNCX) is a late-stage biotechnology company dedicated to unlocking the power of a patient's own biology for the treatment of rare diseases. For more information on ...

Cumberland Pharmaceuticals' Phase 2 trial shows ifetroban improves heart function in Duchenne muscular dystrophy patients.

SHANGHAI & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD ...

C&EN’s tabulation follows approvals from the FDA’s Center for Drug Evaluation and Research; therefore, it does not include blood products, gene therapies, vaccines, and other therapies that ...