Shots:
- PharmaShots’ Designation Report offers a concise overview of the latest drug and device designations granted by major regulatory authorities, including the FDA, EMA, MHLW, Health Canada, and NMPA
- The June 2025 edition covers designations awarded to 43 drugs and 3 medical devices, comprising 17 small molecules, 7 biologics, 8 cell and gene therapies, and 3 medical devices, among others
- Key highlights this month include Sanofi’s Riliprubart, which received Orphan Drug Designation from both the US FDA and Japan’s MHLW for the treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Drugs receiving orphan drug designation by global regulatory bodies echoed as cell & gene therapies, radiopharmaceuticals, small molecules, and biologics. Around 20 drugs received the orphan drug designation across multiple indications
Cell & Gene Therapy: Deramiocel (Capricor Therapeutics) for Becker Muscular Dystrophy, BEAM-101 (Beam Therapeutics) for Sickle Cell Disease, SNUG01 (SineuGene) for Amyotrophic Lateral Sclerosis, CER-1236 (CERo Therapeutics) Acute Myeloid Leukemia, SENTI-202(SentiBio) for r/r Hematologic Malignancies, Small Molecules: Rilzabrutinib (Sanofi) for Sickle Cell Disease, Lumryz (Avadel Pharmaceuticals) for Idiopathic Hypersomnia, VAS-101 (Vascarta) for Sickle Cell Disease, TPST-1120 (Tempest Therapeutics) for Hepatocellular Carcinoma , Biologics: Riliprubart (Sanofi) for Chronic Inflammatory Demyelinating Polyneuropathy, ADoBind MC001 (Medicovestor) for Pancreatic Cancer, DIAG723 (Diagonal Therapeutics) for Hereditary Hemorrhagic Telangiectasia, Lu AG13909 (Lundbeck) for Congenital Adrenal Hyperplasia, Enzyme: JR-446 (MEDIPAL & JCR) for Mucopolysaccharidosis Type IIIB, Protein: Telitacicept (Remegen) for Myasthenia Gravis, Radiopharmaceutical: [18F]florbetaben (Life Molecular Imaging) for Transthyretin (ATTR) Amyloidosis, Polymer: Veverimer (Renibus Therapeutics) for Anti-glomerular Basement Membrane Disease, Peptide: CID-078 (Circle Pharma) for Small Cell Lung Cancer, Vaccine: MVdeltaC (Oncovita) for Pleural Mesothelioma
Around 9 drugs received the Fast Track Designation as gene therapy, small molecules, biologics, radiopharmaceutical and antisense Oligonucleotide
Gene Therapy: VGN-R09b (Shanghai Vitalgen BioPharma) for Parkinson’s Disease, Small Molecule: Mavorixafor (X4 Pharmaceuticals) for Chronic Neutropenia, Nuvisertib (Sumitomo Pharma) for Myelofibrosis, FNP-223 (Ferrer) for Progressive Supranuclear Palsy, SGR-1505 (Schrödinger) for Waldenström Macroglobulinemia, Biologic: Nadunolimab (Cantargia) for Pancreatic Ductal Adenocarcinoma, PHST001 (Pheast Therapeutics) for Ovarian Cancer, Radiopharmaceutical: RAD101 (Radiopharm Theranostics) for Brain Metastases, Antisense Oligonucleotide: AMX0114 (Amylyx Pharmaceuticals) for Amyotrophic Lateral Sclerosis
Regulatory bodies designated six drugs with Breakthrough Therapy Designation, ranging from small molecules and Antisense oligonucleotides to biologics
Small Molecules: Daraxonrasib (Revolution Medicines) for Metastatic Pancreatic Cancer, SC0062 (BioCity Biopharma) for Diabetic Kidney Disease, Iopofosine I 131 (Cellectar) for r/r Waldenstrom Macroglobulinemia, Antisense Oligonucleotide: GTX-102 (Ultragenyx) for Angelman Syndrome, DYNE-101 (Dyne Therapeutics) for Myotonic Dystrophy Type 1, Biologic: Sacituzumab Tirumotecan (Kelun Biotech) for Non-Squamous NSCLC
Six drugs ranging from small molecule to peptide were given priority review
Small Molecules: Zepzelca (Jazz Pharmaceuticals) for Extensive-stage Small Cell Lung Cancer, Zoliflodacin (Innoviva Specialty Therapeutics) for Uncomplicated Gonorrhea, Ziftomenib (Kura Oncology & Kyowa Kirin) for r/r Acute Myeloid Leukemia, Revuforj (Syndax Pharmaceuticals) for R/R mNPM1 Acute Myeloid Leukemia, Azstarys (Shanghai Ark Biopharmaceutical) for Attention-Deficit/Hyperactivity Disorder, Peptide: TransCon CNP (Ascendis Pharma) for Achondroplasia
Two drugs received the rare pediatric disease designation as a gene therapy and small molecule
Small Molecule: QRX003 (Quoin Pharmaceuticals) for Netherton Syndrome, Gene Therapy: VG801 (VeonGen Therapeutics) for ABCA4-mutated Retinal Dystrophy
Three devices received the breakthrough device designation by the US FDA
Shield MCD Test (Guardant Health) for Multi-cancer Screening, Aortic Stenosis ECG Algorithm (Heart Sciences) for Aortic Stenosis Detection, TOBY Test (TOBY health) for Bladder Cancer Detection
A single drug received the regenerative medicine advanced therapy designation
Detalimogene Voraplasmid (enGene Holdings) for Non-Muscle Invasive Bladder Cancer
Related Post: New Drug Designations – May 2025
