What Medicus Pharma Ltd.’s SkinJect application signals for Hedgehog pathway-driven cancers
Discover how SkinJect’s orphan drug bid could reshape Hedgehog-driven cancer treatment. Read what it means for clinicians, regulators, and investors.
Could Ampligen become a late-stage pancreatic cancer breakthrough after AIM ImmunoTech’s latest clinical push?
Can Ampligen’s Phase 3 push reshape pancreatic cancer treatment? Read how AIM ImmunoTech’s latest clinical progress could change the oncology outlook.
Can Immutep Limited convert orphan designation into a credible late-stage oncology program?
FDA orphan drug designation gives Immutep Limited a new sarcoma pathway. Find out why eftilagimod alfa could reshape its oncology strategy.
Can REYOBIQ improve Plus Therapeutics’ path in pediatric high-grade glioma and ependymoma?
Plus Therapeutics won orphan drug designation for REYOBIQ in pediatric malignant gliomas. Read what it changes for CNS cancer development.
How REYOBIQ’s latest regulatory milestone may reshape the pediatric neuro-oncology landscape
Plus Therapeutics secures orphan designation for REYOBIQ in pediatric malignant gliomas. Read what this means for trials, exclusivity, and market strategy.
Can Abbisko Therapeutics turn irpagratinib into the first approved FGFR4 therapy for hepatocellular carcinoma?
Abbisko Therapeutics won EMA orphan drug designation for irpagratinib in liver cancer. Read what this changes for FGFR4-targeted therapy.
Can Drug Farm turn ALPK1 inhibition into the first real disease-modifying approach for ROSAH syndrome?
Drug Farm won FDA orphan drug designation for DF-003 in ROSAH syndrome. Read what this rare disease milestone could change next.
Sentynl Therapeutics licenses Progerinin for progeria as rare-disease strategy expands beyond Zokinvy
Sentynl Therapeutics Inc., the United States-based rare disease subsidiary of Zydus Lifesciences Limited, has entered into a licensing agreement with South Korean biotechnology company PRG S&T to develop the investigational molecule Progerinin (SLC-D011) for Hutchinson-Gilford Progeria Syndrome, an ultra-rare genetic disorder that causes accelerated aging in children. The therapy has received orphan drug designation from […]
Aisa Pharma’s AISA-021 shows mixed but promising signals in systemic sclerosis-associated Raynaud’s phenomenon
Aisa Pharma’s AISA-021 shows promising Phase 2 signals in systemic sclerosis Raynaud’s. Discover what the RECONNOITER data could mean for future treatment.
No FDA advisory committee for MOLBREEVI: what Savara’s Day 74 Letter means for the August PDUFA review
Savara’s molgramostim BLA faces no FDA adcom. Analysis of what the August 2026 PDUFA date means for autoimmune PAP treatment access. Read more.