Browsing Tag
Orphan Drug
11 posts
IntraBio files first-ever FDA application for an Ataxia-Telangiectasia drug with AQNEURSA sNDA submission
IntraBio filed the first-ever FDA application for Ataxia-Telangiectasia via an AQNEURSA sNDA. Read what the Phase III data means for patients and the rare disease market.
Zydus (NSE: ZYDUSLIFE) unit Sentynl adds Korean progeria drug Progerinin to rare disease portfolio as Phase 2A data loom
Sentynl, Zydus Lifesciences' US subsidiary, licenses Progerinin from Korea's PRG S&T for progeria. Phase 2A data due H1 2026. Read the full analysis.
GSK to sell linerixibat rights to Alfasigma for up to $690m as rare liver disease drug nears FDA approval
GSK and Alfasigma agree worldwide licence for linerixibat, a rare liver disease itch drug nearing FDA approval. Deal worth up to $690 million. Read more.
Neuren Pharmaceuticals secures third FDA Fast Track for NNZ-2591 as shares rise 4.21%
Find out how Neuren Pharmaceuticals is expanding FDA Fast Track coverage for NNZ‑2591 across rare disorders like PMS, AS, and Pitt Hopkins.
Theravance completes enrollment in Phase 3 CYPRESS trial for ampreloxetine in MSA-related nOH
Theravance completes Phase 3 enrollment for ampreloxetine in MSA-related nOH. Topline data expected in Q1 2026. Could this be a rare disease breakthrough?
Omeros seeks EMA approval for narsoplimab to treat stem cell transplant-related TA-TMA
Omeros Corporation has submitted a Marketing Authorization Application to the EMA for narsoplimab, aiming to treat TA-TMA across Europe. Learn what’s at stake.
JR-446 developed by JCR Pharmaceuticals secures orphan drug designation from the European Commission for MPS IIIB
The European Commission has granted orphan drug status to JR-446 for MPS IIIB, supporting JCR Pharmaceuticals’ global rare disease expansion.
What is HARLIKU and how will Cycle’s FDA-approved AKU drug help patients starting July 2025?
Cycle Pharmaceuticals secures FDA approval for HARLIKU™, the first approved treatment for alkaptonuria (AKU); launch in the U.S. expected July 2025.
Amplia Therapeutics secures FDA fast track status for narmafotinib in pancreatic cancer, stock surges
Amplia Therapeutics Limited (ASX: ATX) has announced that its Focal Adhesion Kinase (FAK) inhibitor, narmafotinib, has been granted…
Longboard Pharmaceuticals shares see spike as FDA recognizes potential of bexicaserin in Dravet syndrome
Shares of Longboard Pharmaceuticals, Inc. surged on Thursday after the United States Food and Drug Administration (FDA) granted…