Ascendis Pharma’s Hypoparathyroidism Therapy Yorvipath Wins FDA Nod, Fills Market Gap
This week, the U.S. Food and Drug Administration (FDA) granted market approval to Ascendis Pharma...
Rare / orphan disease drugs
Zelgen Biopharmaceuticals’ ZG006 Earns Orphan Drug Designation from FDA for Small-Cell Lung Cancer
Suzhou Zelgen Biopharmaceuticals Co., Ltd (SHA: 688266), a biopharmaceutical company based in China, has announced...
Insilico Medicine’s ISM6331 Earns FDA Orphan Drug Designation for Mesothelioma Treatment
Insilico Medicine, a China-based biotechnology company at the forefront of generative artificial intelligence (AI), has...
BioNTech’s mRNA Cancer Immunotherapy BNT111 Achieves Primary Endpoint in Phase II Trial
Germany-based BioNTech SE (NASDAQ: BNTX) has announced that its mRNA cancer immunotherapy BNT111, in combination...
Organon’s Liptruzet Launches in China for Hypercholesterolemia Treatment
US pharmaceutical company Organon (NYSE: OGN) has announced the official market launch of its single-tablet...
Alphamab’s KN057 Earns FDA Orphan Drug Designation for Hemophilia B Treatment
Alphamab Oncology (HKG: 9966), a biopharmaceutical company based in China, has received another orphan drug...
Belief BioMed’s Hemophilia B Gene Therapy BBM-H901 Slated for Priority Review in China
The Center for Drug Evaluation (CDE) has indicated on its website that the market application...
Sinqi Pharmaceutical Gets NMPA Green Light for SQ-22031 Eye Drops to Treat Dry Eye Syndrome and Neurotrophic Keratitis
Shenyang Sinqi Pharmaceutical Co., Ltd (SHE: 300573), a pharmaceutical company based in China, has received...
Belief BioMed and Takeda’s Hemophilia B Gene Therapy BBM-H901 Accepted for NMPA Review
Belief BioMed Group (BBM), a Shanghai-based gene therapy specialist, and Takeda have announced that the...
IASO Biotherapeutics’ CAR-T Therapy Fucaso Receives FDA IND Approval for Multiple Sclerosis
China-based IASO Biotherapeutics has announced that it has received Investigational New Drug (IND) approval from...
China’s NMPA Considers Breakthrough Designations for Xiangxue and Innostellar’s Therapies
Xiangxue Pharmaceutical Co., Ltd (SHE: 300147) and Innostellar Biotherapeutics Co., Ltd have both been noted...
AccurEdit Therapeutics Receives CDE Approval for Gene Editing Drug ART001 Targeting Amyloidosis
AccurEdit Therapeutics (Suzhou) Co., Ltd, a leading specialist in gene editing therapies based in China,...
Bain Capital-Backed Tenacia Wins NMPA Approval for Ganaxolone Oral Suspension for CDKL5 Deficiency
Tenacia, a central nervous system (CNS) specialist company incubated by Bain Capital, has announced that...
Mabwell Bioscience Lands Orphan Drug Status for B7-H3 Targeting ADC in Small Cell Lung Cancer
Mabwell (Shanghai) Bioscience Co., Ltd (SHA: 688062) has been granted Orphan Drug Designation (ODD) by...
Johnson & Johnson’s Opsumit Faces Generic Competition with Qilu Pharmaceutical’s Entry
Johnson & Johnson (J&J, NYSE: JNJ)’s pulmonary hypertension treatment, Opsumit (macitentan), is set to encounter...
Staidson Pharmaceutical Gets NMPA Green Light for Neurotrophic Keratitis Eye Drop Clinical Trial
Staidson (Beijing) Pharmaceutical Co., Ltd (SHE: 300204), a biopharmaceutical company based in China, has announced...
Novo Nordisk’s Esperoct Receives NMPA Approval, First Long-Acting Factor VIII for Hemophilia A in China
The National Medical Products Administration (NMPA) has granted approval to Novo Nordisk (NYSE: NVO), a...
CStone Pharmaceuticals Partners with Hengrui for Promotion of Ayvakit in China
CStone Pharmaceuticals (HKG: 2616), a biopharmaceutical company based in China, has announced a licensing agreement...
Staidson Pharmaceutical’s Bemiltenase Alfa Earns Orphan Drug Designation from US FDA for Hemophilia Treatment
Staidson (Beijing) Pharmaceutical Co., Ltd (SHE: 300204), a biopharmaceutical company based in China, has announced...
Johnson & Johnson Achieves Positive Phase III Results for Nipocalimab in Generalized Myasthenia Gravis
Johnson & Johnson (J&J, NYSE: JNJ), a leading US-based healthcare company, has announced positive results...