Shots:
- PharmaShots’ Designation Report provides a concise overview of the latest drug designations granted by major regulatory authorities, including the FDA, EMA, MHLW, Health Canada and NMPA
- The May 2025 report covers designations granted to 44 drugs and 5 medical devices, spanning 15 small molecules, 7 biologics, 13 cell and gene therapies & 5 medical devices among others
- Significant trends this month show, JCR Pharmaceuticals’ JR-446 secured Orphan drug designation for Mucopolysaccharidosis Type IIIB
Drugs receiving orphan drug designation by global regulatory bodies echoed as Enzyme,Small molecule,Gene therapy,Probiotic,Anti-sense oligonucleotide,RNA based,Cell Therapy. Around 11 drugs received the orphan drug designation across multiple indications.
Cell and Gene Therapies: BEAM-302 (Beam Therapeutics) for Alpha-1 Antitrypsin Deficiency, GIVI-MPCs (IPS Heart) for Becker Muscular Dystrophy, REP-0003(Repair Biotechnologies) for Homozygous Familial Hypercholesterolemia
Small Molecule: POLB 001 (Poolbeg Pharma) for Cytokine Release Syndrome, THE001 (Thermosome) for Soft Tissue Sarcomas, KER-0193 (Kaerus Bioscience) for Fragile X Syndrome, eRapa (JCR Pharmaceuticals) for Familial Adenomatous Polyposis, QRX003 (Quoin Pharmaceuticals) for Netherton Syndrome
Probiotic: INF108(Infinant Health) for Necrotizing Enterocolitis
Enzyme: JR-446 (JCR Pharmaceuticals) for Mucopolysaccharidosis Type IIIB
Anti-sense Oligonucleotide: CRD-002 (Cure Rare Disease) for Spinocerebellar Ataxia
About 9 drugs received the fast-track designation as small molecule, Gene therapy, Peptide and Biologic.
Biologic: TEV-53408 (Teva Pharmaceutical) for Celiac Disease, ISB 2001(Ichnos Glenmark Innovation) for R/R Multiple Myeloma, ADRX-0706 (Adcentrx Therapeutics) for Cervical Cancer, ZL-1310 (Zai Lab) for Extensive-stage Small Cell Lung Cancer
Gene Therapy: CAP-002 (Capsida Biotherapeutics) for Syntaxin-binding Protein 1 Developmental and Epileptic Encephalopathy (STXBP1-DEE)
Small Molecule: Givinostat (Italfarmaco) for Polycythemia Vera, ATH434 (Alterity Therapeutics) for Multiple System Atrophy, Usnoflast (Zydus) for Amyotrophic Lateral Sclerosis
Peptide: Urcosimod(OKYO Pharma) for Neuropathic Corneal Pain
Two Biologics and Three Small Molecule were given breakthrough therapy designation by the US FDA.
Small Molecule: Tinlarebant (Belite Bio) for Stargardt Disease, Irpagratinib (Abbisko Therapeutics) for Hepatocellular Carcinoma, Mesutoclax( InnoCare Pharma) for R/R Mantle Cell Lymphoma
Biologic: Ersodetug (Rezolute) for Tumor Hyperinsulinism, Veligrotug (Viridian Therapeutics) for Thyroid Eye Disease
Seven drugs ranging from small molecule, Biologics, Gene Therapy, Cell Therapy and Peptide were given priority review.
Small Molecule: Orladeyo (BioCryst) for Hereditary Angioedema, Sevabertinib (Bayer) for NSCLC, Zurletrectinib (Innocare) for Advanced Solid Tumors
Biologic: Sibeprenlimab(Otsuka Pharmaceutical) for Immunoglobulin A Nephropathy
Gene Therapy: RGX-121 (NS Pharma and REGENXBIO) for Mucopolysaccharidosis II
Cell Therapy: Satri-cel(CARsgen Therapeutics) for Gastric/Gastroesophageal Junction Adenocarcinoma
Peptide: Semaglutide(Novo Nordisk) for Metabolic Dysfunction-associated Steatohepatitis
Five devices received the breakthrough device designation by the US FDA.
Speech Restoration Device (Neuralink) for Speech Restoration, HART CADhs Test (Prevencio) for Obstructive Coronary Artery Disease, EnVisio X1 In-Body Spatial Intelligence System (Elucent Medical) for Soft-tissue Surgical Guidance, Pulmonary Artery Denervation System (Gradient Denervation Technologies) for Pulmonary Hypertension and Associated Heart Failure, Total Artificial Heart (BiVACOR) for Biventricular Or Univentricular Heart Failure
Four drugs received the rare pediatric disease designation by the US FDA
Gene Therapy: OCU410ST (Ocugen) for ABCA4-associated retinopathies incl. Stargardt disease, Retinitis pigmentosa 19 (RP19), Cone-rod dystrophy 3 (CORD3); Small Molecule: KER-0193 (Kaerus Bioscience) for Fragile X Syndrome; Probiotic: INF108 (Infinant Health) for Necrotizing Enterocolitis; Anesthetic: RLF-TD011 (Relief Therapeutics) for Epidermolysis Bullosa
Seven cell therapy and one gene therapy received the regenerative medicine advanced therapy designation
Cell & Gene Therapies: CAN-2409 (Candel Therapeutics) for Prostate Cancer, OPGx-LCA5 (Opus Genetics) for Leber Congenital Amaurosis, BEAM-302 (Beam Therapeutics) for Alpha-1 Antitrypsin Deficiency, ART001 (AccurEdit Therapeutics) for ATTR Amyloidosis, 4D-150 (4D Molecular Therapeutics) for Diabetic Macular Edema, BCB-276 (BrainChild Bio) for Diffuse Intrinsic Pontine Glioma, and C-CAR168 (AbelZeta) for Systemic Lupus Erythematosus & Lupus Nephritis
Related Post: New Drug Designations – April 2025
