AccurEdit Therapeutics’ ART001 Shows Over 90% TTR Reduction in ATTR Amyloidosis Trial
Suzhou-based gene editing specialist AccurEdit Therapeutics has announced positive results from an ongoing investigator-initiated trial...
Rare / orphan disease drugs
Novartis Secures Global Rights to PTC Therapeutics’ Huntington’s Disease Treatment PTC518
Swiss pharmaceutical giant Novartis (NYSE: NVS) has entered into a global license and collaboration agreement...
XellSmart Bio-Pharmaceutical Achieves Milestone with First Patient Enrollment in Global iPSC Clinical Study for ALS
XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd., in collaboration with Shanghai East Hospital (Tongji University Affiliated East...
CDE Expands Care Plan with Approvals for Ractigen’s RAG-17 and Vitalgen’s VGR-R01
In a swift succession of actions, China’s Center for Drug Evaluation (CDE) has added two...
Huadong Medicine’s Arcalyst Receives NMPA Approval for Cryopyrin-Associated Periodic Syndromes
China-based Huadong Medicine Co., Ltd (SHE: 000963) has announced receiving marketing approval from the National...
AstraZeneca’s ALXN1850 (Efzimfotase Alfa) Selected for China’s Rare Disease Care Plan
The Center for Drug Evaluation (CDE) in China has announced its intention to include AstraZeneca’s...
Merck’s Winrevair Shows Positive Results in Phase III ZENITH Study for PAH
US-based pharmaceutical giant Merck & Co., Inc. (NYSE: MRK) has announced positive interim analysis results...
Jiangsu Chia Tai Fenghai’s ALS Treatment FHND1002 Selected for CDE’s Patient-Centered Care Plan
China’s Center for Drug Evaluation (CDE) has announced its intention to include Jiangsu Chia Tai...
Leads Biolabs’ LBL-024 Earns FDA Orphan Drug Designation for Neuroendocrine Cancer
China-based Leads Biolabs Inc. has announced that it has obtained Orphan Drug Designation (ODD) from...
Signet Therapeutics’ SIGX1094 Earns Orphan Drug Designation for Gastric Cancer from FDA
China-based Signet Therapeutics has announced that it has received Orphan Drug Designation (ODD) from the...
China Grants Marketing Approval for MSD’s Welireg for von Hippel-Lindau Disease Tumors
China’s National Medical Products Administration has issued marketing approval for Merck, Sharp & Dohme’s (MSD;...
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...
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)...