Rare / orphan disease drugs

Sanofi’s Wayrilz (Rilzabrutinib) Secures Japanese Approval for Immune Thrombocytopenia – Expanding Global Footprint Across Six Major Markets

Sanofi S.A. (NASDAQ: SNY) announced that Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) has approved...

Haisco Pharmaceutical Secures $1.46B Global Licensing Deal with Nuvectis for BRAF and CFB Inhibitor Programs

Fineline Cube Jun 23, 2026

Haisco Pharmaceutical Group Co., Ltd. (SHE: 002653) announced an exclusive global licensing agreement with U.S.-based...

Company Drug

GSK’s Momelotinib Receives Orphan Drug Designation from FDA and EMA for VEXAS Syndrome, Addressing Critical Unmet Need in Rare Myeloid Disorder

Fineline Cube Jun 17, 2026

GlaxoSmithKline plc (GSK) (NYSE: GSK) announced that momelotinib has received Orphan Drug Designation (ODD) from...

Company Drug

Fosun Pharma Secures NMPA Approval for Fumaining in Pediatric Langerhans Cell Histiocytosis – Expands MEK Inhibitor Franchise

Fineline Cube Jun 16, 2026

Fosun Pharmaceutical (Group) Co., Ltd. (SHA: 600196; HKG: 2196) announced that China’s National Medical Products...

Company Drug

Pfizer Secures FDA Approval for Marstacimab Expanded Indication in Hemophilia A and B Across Pediatric and Adult Populations

Fineline Cube Jun 9, 2026

Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug Administration (FDA) has...

Company Drug

Huahai Pharmaceutical Receives NMPA Approval for Dual-Target IPF Therapy HB0056, Adding to Global Clinical Trial Authorizations

Fineline Cube Jun 3, 2026

Zhejiang Huahai Pharmaceutical Co., Ltd. (SHA: 600521) announced it has received clinical trial approval from...

Company Drug

Sanofi’s Venglustat Receives FDA Priority Review and Breakthrough Therapy Designation for Type 3 Gaucher Disease

Fineline Cube May 29, 2026

Sanofi (NASDAQ: SNY) announced that the U.S. Food and Drug Administration (FDA) has granted priority...

Company Drug

AbbVie Secures FDA Approval for DECNUPAZ in Blastic Plasmacytoid Dendritic Cell Neoplasm – First CD123-Targeting ADC Addresses Ultra-Rare Hematologic Malignancy

Fineline Cube May 28, 2026

AbbVie Inc. (NYSE: ABBV) announced on May 27, 2026, that the U.S. Food and Drug...

Company Drug

Changchun High & New Technology Industries Subsidiary Secures NMPA Approval for GenSci144, Novel Oral PKU Treatment Targeting SLC6A19 Transporter

Fineline Cube May 27, 2026

Changchun High & New Technology Industries (Group) Inc. (SHE: 000661) announced that its subsidiary Changchun...

Company Drug

Boehringer Ingelheim’s Ofev Receives CHMP Endorsement for Idiopathic and Progressive Pulmonary Fibrosis

Fineline Cube May 25, 2026

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a...

Company Drug

Lundbeck’s Asedebart Receives Japanese Orphan Drug Designation for Congenital Adrenal Hyperplasia and Cushing’s Disease

Fineline Cube May 19, 2026

Lundbeck (OTCMKTS: HLUBF) announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted...

Company Drug

Pfizer’s Hympavzi Gains European Approval for Hemophilia with Inhibitors – Novel TFPI-Targeting Therapy Offers Weekly Subcutaneous Alternative to Factor Replacement

Fineline Cube May 14, 2026

Pfizer Inc. (NYSE: PFE) announced it has received European Commission (EC) approval for an indication...

Company Drug

Kanghong Pharma’s Gene Therapy KHN921 Receives FDA Approval for Hypertrophic Cardiomyopathy Trial – AAV9 Vector Targets MYBPC3 Mutations with Potential One-Time Treatment

Fineline Cube May 14, 2026

Chengdu Kanghong Pharmaceutical Group Co., Ltd. (SHE: 002773) announced that its subsidiary Chengdu Origen Biotechnology...

Company Deals

Hybio Pharmaceutical Expands Torrent Pharma Partnership with Lanreotide Acetate for U.S. Market

Fineline Cube May 12, 2026

Hybio Pharmaceutical Co., Ltd. (SHE: 300199) announced an expanded strategic partnership with Torrent Pharmaceuticals Inc.,...

Company Drug

Keymed Biosciences Secures NMPA Breakthrough Therapy Designation for CM336 in AL Amyloidosis

Fineline Cube May 9, 2026

Keymed Biosciences Inc. (HKG: 2162) announced that its BCMA×CD3 T cell engager (TCE), CM336 (OM336),...

Company Drug

HutchMed’s Sovleplenib Granted NMPA Priority Review for Warm Antibody Autoimmune Hemolytic Anemia

Fineline Cube Apr 30, 2026

HutchMed (China) Limited (NASDAQ: HCM, HKG: 0013) announced that China’s National Medical Products Administration (NMPA)...

Company Drug

Insilico Medicine Secures Chinese Clinical Clearance for AI-Discovered Rentosertib Inhalation in Pulmonary Fibrosis

Fineline Cube Apr 29, 2026

Insilico Medicine (HKG: 3696), a generative artificial intelligence (AI)-driven biotechnology company, announced it has received...

Company Drug

Santo Therapeutics Secures Dual FDA Designations for ST002 Gene Therapy in NF2-Related Schwannomatosis – Advancing In Vivo Lentiviral Platform

Fineline Cube Apr 28, 2026

Santo Therapeutics, a Hangzhou-based in vivo gene therapy developer, announced it has received both Orphan...

Company Drug

Johnson & Johnson’s Imaavy (nipocalimab) Receives FDA Priority Review for Warm Autoimmune Hemolytic Anemia – Expanding FcRn Blocker Franchise

Fineline Cube Apr 28, 2026

Johnson & Johnson (J&J, NYSE: JNJ) announced that its supplemental Biologics License Application (sBLA) for...

Company Drug

Prothena’s Coramitug Receives FDA Fast Track Designation for ATTR-CM – Advancing Novo Nordisk’s $1.2B Amyloidosis Pipeline

Fineline Cube Apr 28, 2026

Prothena Corporation plc (NASDAQ: PRTA) announced that the U.S. Food and Drug Administration (FDA) has...

Rare / orphan disease drugs

Sanofi’s Wayrilz (Rilzabrutinib) Secures Japanese Approval for Immune Thrombocytopenia – Expanding Global Footprint Across Six Major Markets

Haisco Pharmaceutical Secures $1.46B Global Licensing Deal with Nuvectis for BRAF and CFB Inhibitor Programs

GSK’s Momelotinib Receives Orphan Drug Designation from FDA and EMA for VEXAS Syndrome, Addressing Critical Unmet Need in Rare Myeloid Disorder

Fosun Pharma Secures NMPA Approval for Fumaining in Pediatric Langerhans Cell Histiocytosis – Expands MEK Inhibitor Franchise

Pfizer Secures FDA Approval for Marstacimab Expanded Indication in Hemophilia A and B Across Pediatric and Adult Populations

Huahai Pharmaceutical Receives NMPA Approval for Dual-Target IPF Therapy HB0056, Adding to Global Clinical Trial Authorizations

Sanofi’s Venglustat Receives FDA Priority Review and Breakthrough Therapy Designation for Type 3 Gaucher Disease

AbbVie Secures FDA Approval for DECNUPAZ in Blastic Plasmacytoid Dendritic Cell Neoplasm – First CD123-Targeting ADC Addresses Ultra-Rare Hematologic Malignancy

Changchun High & New Technology Industries Subsidiary Secures NMPA Approval for GenSci144, Novel Oral PKU Treatment Targeting SLC6A19 Transporter

Boehringer Ingelheim’s Ofev Receives CHMP Endorsement for Idiopathic and Progressive Pulmonary Fibrosis

Lundbeck’s Asedebart Receives Japanese Orphan Drug Designation for Congenital Adrenal Hyperplasia and Cushing’s Disease

Pfizer’s Hympavzi Gains European Approval for Hemophilia with Inhibitors – Novel TFPI-Targeting Therapy Offers Weekly Subcutaneous Alternative to Factor Replacement

Kanghong Pharma’s Gene Therapy KHN921 Receives FDA Approval for Hypertrophic Cardiomyopathy Trial – AAV9 Vector Targets MYBPC3 Mutations with Potential One-Time Treatment

Hybio Pharmaceutical Expands Torrent Pharma Partnership with Lanreotide Acetate for U.S. Market

Keymed Biosciences Secures NMPA Breakthrough Therapy Designation for CM336 in AL Amyloidosis

HutchMed’s Sovleplenib Granted NMPA Priority Review for Warm Antibody Autoimmune Hemolytic Anemia

Insilico Medicine Secures Chinese Clinical Clearance for AI-Discovered Rentosertib Inhalation in Pulmonary Fibrosis

Santo Therapeutics Secures Dual FDA Designations for ST002 Gene Therapy in NF2-Related Schwannomatosis – Advancing In Vivo Lentiviral Platform

Johnson & Johnson’s Imaavy (nipocalimab) Receives FDA Priority Review for Warm Autoimmune Hemolytic Anemia – Expanding FcRn Blocker Franchise

Prothena’s Coramitug Receives FDA Fast Track Designation for ATTR-CM – Advancing Novo Nordisk’s $1.2B Amyloidosis Pipeline

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Gilead’s Trodelvy (Sacituzumab Govitecan) Secures European Commission Approval for First-Line Metastatic TNBC – Expanding ADC Access to Treatment-Naïve Patients

Henlius and Organon Canada Launch Denosumab Biosimilars BILDYOS and TUZEMTY – Expanding Access to Bone-Targeted Therapies Across All Canadian Indications

AbbVie’s SKYRIZI (Risankizumab) Secures European Commission Approval for Pediatric Plaque Psoriasis – First IL-23 Inhibitor Authorized for Children as Young as Six

YHLO Biotech and Jingyi Bio Forge Strategic Alliance to Advance Neuroimmunology Diagnostics – Expanding Autoimmune Testing into Neurological Disorders