08/04/2026 FDA Grants Orphan Drug Status to Plus Therapeutics’ REYOBIQ™ for Pediatric Malignant Gliomas FDA Grants Orphan Drug Designation to Compass Therapeutic’s Tovecimig for Biliary Tract Cancer FDA Grants Orphan Drug Status to Plus Therapeutics’ REYOBIQ™ for Pediatric Malignant Gliomas ( Ref ) Plus Therapeutics’ REYOBIQ™ (rhenium Re186 obisbemeda) has been granted orphan drug status for pediatric malignant gliomas, with scope expanded to include pediatric ependymoma Regu

24/03/2026 The Ph3 SENTRY trial of Karyopharm's selinexor + ruxolitinib met SVR35 but not Abs-TSS endpoint in frontline myelofibrosis ReCerise's RCT1213 received the FDA ODD for hepatocellular carcinoma The Ph3 SENTRY trial of Karyopharm's selinexor + ruxolitinib met SVR35 but not Abs-TSS endpoint in frontline myelofibrosis ( Ref ) Karyopharm Therapeutics reported topline results from its Phase 3 SENTRY trial of selinexor 60 mg + ruxolitinib in frontline myelofibrosis. The tr

17/03/2026 CRISM Therapeutics' irinotecan received the FDA ODD for malignant glioma Voro Therapeutics and Daiichi Sankyo sign a research collaboration for Next-Generation Tumor-Activated ADCs Myriad Genetics' MyChoice CDx test received FDA approval as the companion diagnostic for Zejula CRISM Therapeutics' irinotecan received the FDA ODD for malignant glioma ( Ref ) The US FDA granted the Orphan Drug Designation to CRISM Therapeutics Corporation's irinotecan for the treatment

21/01/2026 BioNTech's BNT113 + pembro received the FDA fast track designation for HPV16+ R/M SCCHN with PD-L1 CPS ≥1 Wugen's soficabtagene geleucel received the FDA Breakthrough Therapy Designation for T cell malignancies Opna Bio's zavabresib received the FDA orphan drug designation for myelofibrosis BioNTech's BNT113 + pembro received the FDA fast track designation for HPV16+ R/M SCCHN with PD-L1 CPS ≥1 ( Ref ) The US FDA granted the fast track designation to BioNTech's BNT

18/09/2025 Replimune completed a Type A meeting with the FDA to discuss the CRL for vusolimogene oderparepvec + nivo ( Ref ) Replimune...

08/09/2025 Beyond Air's BA-101 received the Orphan Drug Designation for GBM ( Ref ) The US FDA granted the Orphan Drug Designation to...