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.
Moleculin Biotech, Inc. secures Japanese IP boost as Annamycin enters pivotal Phase 3
Moleculin secures Japanese patent for Annamycin reconstitution. Discover what this means for AML development and global market strategy.
Imviva Biotech secures FDA orphan designation as CD7 CAR-T development faces execution challenges
FDA grants orphan drug designation to Imviva Biotech’s CTD402. Find out what this means for allogeneic CAR-T development in T-cell leukemia.
FDA grants orphan drug designation to Atossa’s (Z)-Endoxifen for Duchenne: What’s next?
Atossa Therapeutics Inc. has received Orphan Drug Designation from the U.S. Food and Drug Administration for its investigational compound (Z)-Endoxifen in the treatment of Duchenne muscular dystrophy (DMD). The designation, disclosed on January 16, 2026, supplements the previously granted Rare Pediatric Disease Designation for the same compound and indication. The move marks a notable regulatory […]