Innorna’s mRNA Drug IN015 Earns Orphan Drug Designation for PFIC Treatment
Shenzhen-based Innorna Co., Ltd, a leading platform company specializing in mRNA technology and LNP (lipid...
Rare / orphan disease drugs
CDE Includes Double-Crane’s Hydroxocobalamine and Sanofi Genzyme’s Fitusiran in Rare Disease Care Plan
China’s Center for Drug Evaluation (CDE) has announced the inclusion of China Resources Double-Crane Pharmaceutical...
US FDA Grants ODD to Ractigen’s siRNA Therapy RAG-21 for ALS Treatment
China-based small activating RNA (saRNA) drug developer, Ractigen Therapeutics, has announced that it has received...
Exegenesis Bio’s Gene Therapy EXG110 Earns Orphan Drug Status for Fabry Disease
Exegenesis Bio, a leading provider of epigenetics experimental and analytical services headquartered in Hangzhou, has...
ETERN Therapeutics’ ETS-006 Receives FDA Orphan Drug Designation for Pleural Mesothelioma
ETERN Therapeutics, a Shanghai-based developer of small-molecule anti-tumor drugs, has announced that it has received...
Ascentage Pharma’s Lisaftoclax and AceLink Therapeutics’ AL01211 Receive FDA Designations
The Center for Drug Evaluation (CDE) website has indicated that China-based Ascentage Pharma’s (HKG: 6855)...
AskBio’s AB-1003 Receives Rare Pediatric Disease and Orphan Drug Designations from FDA
Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned subsidiary of Bayer AG (ETR: BAYN), has announced...
Belief BioMed’s BBM-D101 Receives FDA Orphan Drug and Pediatric Rare Disease Designations for DMD
Belief BioMed has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan...
Innorna Co., Ltd Receives FDA Rare Pediatric Disease Designation for mRNA Drug IN015
Shenzhen-based Innorna Co., Ltd has announced that it has received Rare Pediatric Disease Designation (RPDD)...
AstraZeneca’s Fasenra Approved by EU for Eosinophilic Granulomatosis Treatment
UK pharmaceutical giant AstraZeneca Inc. (NASDAQ: AZN) has received marketing approval from the European Commission...
RemeGen’s Telitacicept Receives Priority Review Status from China’s CDE for gMG Treatment
RemeGen (HKG: 9995), a China-based pharmaceutical company, has announced that the Center for Drug Evaluation...
Novo Nordisk’s Alhemo Receives CHMP Recommendation for Haemophilia Prophylaxis in Europe
Novo Nordisk (NYSE: NVO), a Danish pharmaceutical company, has been notified that the Committee for...
Jacobio Pharma’s G12C Inhibitor Gecirasib Earns Orphan Drug Designation in Europe for Pancreatic Cancer
Jacobio Pharma (HKG: 1167), a China-based biopharmaceutical company, has announced that it has received Orphan...
Johnson & Johnson’s Nipocalimab Shows Promise in Treating Adolescent Myasthenia Gravis in Phase II/III Trial
Johnson & Johnson (J&J, NYSE: JNJ), a US pharmaceutical giant, has reported encouraging outcomes from...
Pfizer’s Hympavzi Secures FDA Nod, Offering Novel Once-Weekly Treatment for Hemophilia B
Pfizer Inc. (NYSE: PFE) has scored a significant win with the US Food and Drug...
Biogen’s China Unit Gets Conditional NMPA Nod for Qalsody (Tofersen) in ALS Treatment
Biogen (NASDAQ: BIIB)’s Chinese subsidiary has announced that it has received conditional approval from the...
China’s CDE Announces “Care Plan” to Include Pfizer’s Marstaximab for Rare Disease Drug Development
The National Health Commission (NHC) of China, through its Center for Drug Evaluation (CDE), has...
Beijing Aims to Establish Rare Disease Drug Security Pilot Zone with ‘White List’ System
Beijing is set to follow in the footsteps of Boao Lecheng with the release of...
AstraZeneca’s Fasenra Secures FDA Nod for EGPA Treatment, Strengthening Rare Disease Portfolio
UK pharmaceutical giant AstraZeneca (NASDAQ: AZN, LON: AZN) has announced that the US Food and...
CDE Launches CARE Plan to Streamline Rare Disease Drug Development
The Center for Drug Evaluation has unveiled the ‘Pilot Work Plan for Patient-Centered Action for...