MarketPulse

orphan drug designation

16 articles
BenzingaBenzinga··Atossa Therapeutics Inc

Atossa Therapeutics Advances (Z)-Endoxifen Pipeline With FDA Orphan Designations

Atossa Therapeutics reported Q1 2026 expenses of $9.9M, secured FDA orphan drug designation for lead candidate (Z)-endoxifen in rare diseases.
ATOSclinical developmentbreast cancer
GlobeNewswire Inc.GlobeNewswire Inc.··Orion Corporation

Orion Surges on Strong Q1 Results, Raises 2026 Outlook Amid Pipeline Advances

Orion posts strong Q1 with 17.8% revenue growth, 47.3% operating profit surge, and raises 2026 outlook. Pipeline advances boost investor confidence.
BAYRYclinical trialsorphan drug designation
GlobeNewswire Inc.GlobeNewswire Inc.··Orion Corporation

Orion Lifts 2026 Outlook After Strong Q1 Surge Driven by Nubeqa Royalties

Finnish pharma $ORION reported 17.8% Q1 revenue growth to €417.7M, raised full-year guidance citing Nubeqa royalties and pipeline progress.
OECrevenue growthclinical trial
GlobeNewswire Inc.GlobeNewswire Inc.··Immutep Limited

Immutep Wins FDA Orphan Drug Nod for Sarcoma Treatment Eftilagimod Alfa

Immutep gains FDA orphan drug designation for eftilagimod alfa in soft tissue sarcoma, with Phase II trial results exceeding efficacy targets by 47%.
MRKIMMPFDA approvalorphan drug designation
BenzingaBenzinga··Soligenix, Inc.

Soligenix Burns Through Cash as Rare Disease Pipeline Awaits Make-or-Break 2026 Trials

Soligenix reported zero 2025 revenue and $11.1M net loss. Company has $7.9M cash, seeking partnerships to fund operations through Q4 2026 pivotal trials.
SNGXPhase 3 clinical trialbiopharmaceutical
BenzingaBenzinga··Na

Galapagos and Gilead Partner on T Cell Therapy, Splitting $1.7B Ouro Deal

Galapagos and Gilead form binding collaboration on gamgertamig, an autoimmune T cell engager, with Galapagos receiving 50% of Gilead's $1.675B Ouro Medicines acquisition.
GILDGLPGclinical developmentautoimmune diseases
BenzingaBenzinga··Prnewswire

Soligenix Wins EU Orphan Status for Behçet's Disease Drug SGX945

Soligenix's dusquetide (SGX945) receives EU orphan drug designation for Behçet's Disease, backed by Phase 2a data showing 40% ulcer reduction with no adverse events.
SNGXrare diseaseclinical trial
BenzingaBenzinga··Vandana Singh

Gilead Bolsters Inflammation Arsenal With $1.675B Ouro Medicines Deal

Gilead acquires Ouro Medicines for $1.675B upfront, targeting inflammation pipeline expansion. Galapagos NV partners to co-develop lead asset OM336.
GILDACLXGLPGacquisitionorphan drug designation
GlobeNewswire Inc.GlobeNewswire Inc.··Na

Galapagos and Gilead Team Up on Breakthrough T Cell Engager for Autoimmune Diseases

Galapagos and Gilead plan to jointly develop an Ouro T cell engager for autoimmune diseases, splitting acquisition costs while Galapagos leads development and Gilead commercializes, freeing $500M in Galapagos cash.
GILDGLPGautoimmune diseasesorphan drug designation
GlobeNewswire Inc.GlobeNewswire Inc.··Na

Galapagos, Gilead in Advanced Talks on T-Cell Therapy Partnership

Galapagos and Gilead are in advanced partnership talks for a first-in-class T-cell engager for autoimmune diseases, with 50% cost-sharing and €500 million capital freed for Galapagos.
GILDGLPGstrategic partnershipclinical development
BenzingaBenzinga··Soligenix, Inc.

Soligenix's HyBryte Shows Promise Against Cutaneous Lymphoma at 2026 Workshop

Soligenix's HyBryte™ cutaneous lymphoma therapy shows positive results ahead of 2026 presentation, with FDA awarding $2.6M development grant.
SNGXPhase 3 clinical trialFDA approval
BenzingaBenzinga··Nabaparna Bhattacharya

AIM ImmunoTech Surges 73% on Japanese Patent Victory for Cancer Therapy

AIM ImmunoTech ($AIMT) surged 73.66% after securing Japanese patent approval for Ampligen cancer therapy through 2039, strengthening its immuno-oncology portfolio.
AIMintellectual propertyorphan drug designation
GlobeNewswire Inc.GlobeNewswire Inc.··Telix Pharmaceuticals Limited

Telix Resubmits FDA Application for Brain Cancer Imaging Drug Pixclara

Telix resubmits FDA application for Pixclara, a brain cancer imaging agent with Orphan Drug and Fast Track designations, addressing a regulatory Complete Response Letter.
TLXorphan drug designationradiopharmaceuticals
GlobeNewswire Inc.GlobeNewswire Inc.··Pmv Pharmaceuticals, Inc.

PMV Pharma's Rezatapopt Shows Promise, But Cash Clock Ticks Toward 2027

PMV Pharmaceuticals reports 50% response rate for ovarian cancer drug rezatapopt, but faces funding pressure with cash runway extending only to Q2 2027.
PMVPclinical trial resultsorphan drug designation
GlobeNewswire Inc.GlobeNewswire Inc.··Can-Fite Biopharma Ltd.

Can-Fite's Namodenoson Clears Safety Hurdle in Pancreatic Cancer Study

Can-Fite's namodenoson meets primary safety endpoint in Phase IIa pancreatic cancer trial with no new safety signals; survival data pending.
CANFclinical developmentorphan drug designation
BenzingaBenzinga··Prnewswire

Soligenix Wins EMA Backing for SGX945 Orphan Drug Status in Behçet's Disease

Soligenix wins EMA orphan drug designation backing for SGX945 treating Behçet's Disease, following positive Phase 2a results and prior FDA support.
SNGXrare diseaseclinical trial