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 April 2025 report covers designations granted to 36 drugs and 6 medical devices, spanning 14 small molecules, 3 biologics, 11 cell and gene therapies & 6 medical devices among others
- Significant trends this month show, Allogene Therapeutics’ ALLO-329 secured fast track designation for active refractory moderate-to-severe systemic lupus erythematous (SLE), active severe/refractory idiopathic inflammatory myopathy (IIM) and active refractory diffuse systemic sclerosis (SSc)
Drugs receiving orphan drug designation by global regulatory bodies echoed as cell & gene therapies, RNAi Therapy, small molecules, antisense oligonucleotide, and biologics. Around 14 drugs received the orphan drug designation across multiple indications.
Cell & Gene Therapies: CK0801 (Cellenkos) for Aplastic Anemia, RVB-003 (ResVita Bio) for Netherton Syndrome, PS-002 (Purespring Therapeutics) for Primary IgA Nephropathy; mRNA Therapy: IN013 (Innora) for Wilson Disease; Small Molecules: Rilzabrutinib (Sanofi) for Warm Autoimmune Hemolytic Anemia and IgG4-related disease, NS-229 (NS Pharma) for Eosinophilic Granulomatosis with Polyangiitis, TPST-1495 (Tempest Therapeutics) for Familial Adenomatous Polyposis, BA-102 (NeuroNOS) for Phelan-McDermid syndrome, Gloperba (Scilex) for Pericarditis, Deucrictibant (Pharvaris) for Bradykinin-mediated Angioedema, AB8939 (AB Science) for Acute Myeloid Leukemia; Biologic: Del-desiran (Avidity Bioscience) for Myotonic Dystrophy Type 1; RNAi Therapy: CBP-4888 (Comanche Biopharma) for sFlt1-mediated Preterm Preeclampsia; Antisense Oligonucleotide: DYNE-251 (Dyne Therapeutics) for Duchenne Muscular Dystrophy
About 12 drugs received the fast track designation as small molecules, biologics, vaccine, antisense oligonucleotides and cell & gene therapies.
Cell & Gene Therapy: VERVE-102 (Verve Therapeutics) for Hyperlipidemia and High Lifetime CV risk, ALLO-329 (Allogene Therapeutics) for Systemic Lupus Erythematous, Idiopathic Inflammatory Myopathy and Diffuse Systemic Sclerosis, PBGENE-HBV (Precision Biosciences) for Hepatitis B; Small Molecule: ATX-295 (Accent Therapeutics) for Advanced/Metastatic Platinum-Resistant or Refractory Ovarian Cancer, ATX-559 (Accent Therapeutics) for Unresectable/Metastatic dMMR/MSI-H Colorectal Cancer, PTX-100 (Prescient Therapeutics) for R/R Mycosis Fungoides, BI-1808 (Bioinvent) for R/R Mycosis Fungoides and Sézary syndrome, INX-315 (Incyclix Bio) for CCNE1-amplified platinum-resistant/refractory Ovarian Cancer; Biologic: Emactuzumab (SynOx Therapeutics) for Tenosynovial Giant Cell Tumours, Bempikibart (Q32 Bio) for Alopecia Areata; Vaccine: ARCT-2304 (Arcturus Therapeutics) for Influenza A H5N1 subtype; Antisense Oligonucleotide: BIIB080 (Biogen) for Alzheimer’s disease
One gene therapy and one cell therapy were given breakthrough therapy designation by the US FDA.
Cell & Gene Therapy: AMT-130 (Uniqure) for Huntington’s disease, BCB-276 (BrainChild Bio) for Diffuse Intrinsic Pontine Glioma
Three drugs ranging from small molecule and protein to peptide were given priority review.
Small Molecule: Agamree (Catalyst Pharmaceuticals) for Duchenne Muscular Dystrophy; Protein: Eylea HD (Regeneron) for Macular Edema from Retinal Vein Occlusion (RVO) & Dosing Expansion; Peptide: Empaveli (Apellis Pharmaceuticals) for C3 glomerulopathy & Primary Immune Complex Membranoproliferative Glomerulonephritis
One drug received the rare pediatric disease designation by the US FDA.
mRNA Therapy: IN013 (Innora) for Wilson Disease
Six devices received the breakthrough device designation by the US FDA.
Paige PanCancer Detect (Paige) for detection of cancer-suspicious foci across multiple tissues and organs, Selective Cytopheretic Device Therapy (SeaStar Medical) for treatment of systemic inflammatory response in adults and pediatrics undergoing cardiac surgery, vCLASTM Cryoablation System (Adagio Medical) for treatment of drug-refractory, recurrent, sustained monomorphic ventricular tachycardia, Atrioventricular Interval Modulation Therapy (Orchestra Biomed & Medtronic) for uncontrolled hypertension, EvoLiver (Mursla Bio) for early liver cancer detection, VENTANA TROP2 RxDx Test (Roche) for identification of non-squamous NSCLC patients
One drug received the Priority Medicine designation as a small molecule.
Small molecule: Radiprodil (GRIN Therapeutics) for GRIN-related neurodevelopmental disorder
Two cell therapy and one gene therapy received the regenerative medicine advanced therapy designation
Cell & Gene Therapies: ATSN-201 (Atsena Therapeutics) for X-linked Retinoschisis, LYL314 (Lyel Immunopharma) for R/R Large B-cell Lymphoma, FT819 (Fate Therapeutics) for Systemic Lupus Erythematosus
One drug received the Qualified Infectious Disease Product designation
VRP-034 (Venus Remedies) for the treatment of bloodstream infections caused by Polymyxin B
Related Post: New Drug Designations – March 2025
