News about "Orphan Drug Designation "

Incyte to Acquire Vega Therapeutics, Expanding Hematology Portfolio

Incyte has agreed to acquire Vega Therapeutics, expanding its hematology portfolio into bleeding disorders with the addition of VGA039, a phase 3 investigational monoclonal antibody for von Willebrand disease. The deal includes USD 1.25 billion upfront and up to USD 750 million in additional payments upon achievement of sales milestones.

Orphan Drug Designation | 09/06/2026 | By News Bureau

FDA Grants Priority Review to Sanofi's Venglustat for Type 3 Gaucher Disease

Sanofi’s brain-penetrant investigational therapy venglustat has received FDA priority review after phase 3 data demonstrated positive efficacy and safety outcomes in patients with type 3 Gaucher disease.

Orphan Drug Designation | 29/05/2026 | By News Bureau

Lundbeck Secures Orphan Drug Designation in Japan for Asedebart Targeting Rare Endocrine Disorders

Lundbeck has received orphan drug designation in Japan for asedebart, an investigational ACTH-targeting therapy being developed for congenital adrenal hyperplasia and Cushing’s disease.

Orphan Drug Designation | 18/05/2026 | By News Bureau

SN BioScience Doses First Patient in Phase 1b/2 Clinical Trial of SNB-101

SN BioScience initiates first patient dosing in a global phase 1b/2 trial of SNB-101, a nanoparticle anticancer therapy for Small Cell Lung Cancer (SCLC).

Orphan Drug Designation | 16/05/2026 | By News Bureau

Partner Therapeutics Wins FDA Approval for BIZENGRI in NRG1+ Cholangiocarcinoma

Partner Therapeutics receives US Food and Drug Administration (FDA) approval for BIZENGRI, the first targeted therapy for NRG1 fusion-positive cholangiocarcinoma, expanding use across NSCLC and pancreatic cancer.

Orphan Drug Designation | 12/05/2026 | By News Bureau

Rznomics Secures US FDA RMAT Designation for Liver Cancer Gene Therapy Candidate RZ-001

The FDA has granted Regenerative Medicine Advanced Therapy designation to RZ-001, an RNA-based gene therapeutic for hepatocellular carcinoma, accelerating its clinical development and regulatory review pathway.

Orphan Drug Designation | 11/05/2026 | By News Bureau

ImmunoForge Unveils LMT15 Platform to Transform CNS Drug Delivery

The clinical-stage biotech firm ImmunoForge is advancing its next-generation blood-brain barrier shuttle technology, combining enhanced brain permeability with long-acting formulations to accelerate therapies for CNS and rare diseases.

Orphan Drug Designation | 24/04/2026 | By News Bureau

Abbisko's Irpagratinib Secures EMA Orphan Drug Tag for Liver Cancer

European regulator grants rare disease designation to FGFR4 inhibitor, boosting its clinical development and commercial prospects in hepatocellular carcinoma.

Orphan Drug Designation | 01/04/2026 | By News Bureau |  115

EU Grants Conditional Approval to Rezurock for Chronic GVHD Treatment

European Commission authorises belumosudil for patients aged 12 plus with limited treatment options, marking a significant advance in managing a life-threatening post-transplant condition.

Orphan Drug Designation | 01/04/2026 | By News Bureau

GSK plc Secures Japan Orphan Drug Status for Ris-Rez in Small-Cell Lung Cancer

Japan’s health ministry grants orphan drug designation to GSK’s investigational ADC Ris-Rez for SCLC, supported by early clinical data showing durable responses in advanced-stage patients.

Orphan Drug Designation | 24/03/2026 | By News Bureau |  151