Browsing Tag
Orphan Drug Designation
14 posts
Immutep (ASX: IMM) surges 48% on FDA orphan drug designation: Is the STS pivot the new thesis?
Immutep (ASX: IMM) jumped 48% after the FDA granted orphan drug status for eftilagimod alfa in soft tissue sarcoma. Here is what the EFTISARC-NEO data means for the investment case.
How orphan drug status and patent protection shape Ampligen’s commercial and regulatory outlook
Discover how orphan drug status and long-dated patents could shape Ampligen’s regulatory path and commercial value in pancreatic cancer.
Kiniksa’s KPL-387 earns FDA orphan drug nod, advancing next-gen therapy for recurrent pericarditis
Find out how Kiniksa’s KPL-387 won FDA orphan drug status for pericarditis — and what it means for the company’s expanding rare-disease franchise.
Senti Biosciences gets FDA orphan drug status for SENTI-202 in relapsed AML treatment
Senti Biosciences earns FDA orphan drug designation for SENTI-202 in AML. Find out how this logic-gated CAR-NK therapy could reshape leukemia treatment.
CERo Therapeutics gets FDA boost for CER-1236, eyes breakthrough in AML cell therapy
CERo Therapeutics secures FDA orphan drug status for CER-1236 in AML, advancing its novel CER-T immunotherapy platform. Read what this means for investors.
BioArctic advances exidavnemab phase 2a cohorts after safety review
BioArctic expands its Phase 2a trial for exidavnemab after a successful safety review. Read how this impacts Parkinson’s and MSA drug development today.
Dimerix stock up 74% YTD as FSGS asset DMX-200 gets safety green light
Dimerix’s FSGS drug candidate DMX-200 clears key Phase 3 safety review with no concerns. Learn how this ASX biotech aims to reshape kidney disease treatment.
Breakthrough for Wilson disease: Innorna’s IN013 wins dual FDA fast-track approvals
Innorna accelerates mRNA treatment for Wilson Disease with dual FDA designations. Discover how IN013 could transform care for rare genetic disorders!
Game-changing cancer therapy: Imugene’s VAXINIA receives FDA orphan drug status for bile tract cancer
Imugene Limited has received a significant boost with the United States Food and Drug Administration (FDA) granting Orphan…
Telix Pharmaceuticals submits NDA for Pixclara to advance brain cancer imaging
Telix Pharmaceuticals Limited (ASX: TLX) has taken a significant step in the realm of neuro-oncology by submitting a…