Shots:    

• PharmaShots’ Designation Report provides a concise overview of the latest drug designations granted by major regulatory authorities, including the FDA, EMA, MHLW, and NMPA  

• The February 2025 report covers designations granted to 53 drugs and 4 medical devices, spanning 16 small molecules, 14 biologics, 18 cell and gene therapies & 4 medical devices among others  

• Significant trends this month show, Gilead’s Lenacapavir secured priority review by both the US FDA & EMA for Pre-Exposure Prophylaxis (PrEP) to prevent HIV in individuals at risk 

Drugs receiving orphan drug designation by global regulatory bodies echoed as cell & gene therapies, radiopharmaceuticals, small molecules, and biologics

A Quick Look

Cell & Gene Therapies: ABO-101 (Arbor Biotechnologies) for Primary Hyperoxaluria Type 1, EXG-34217 (Elixirgen Therapeutics) for Telomere Biology Disorders, EN-374 (Ensoma) for Chronic Granulomatous Disease, Porosome Reconstitution Therapy (Porosome Therapeutics) for Cystic Fibrosis, Small Molecules: OPN-6602 (Opna Bio) for Multiple Myeloma, IL21120033 (iLeadBMS) for Idiopathic Pulmonary Fibrosis, Radiopharmaceutical: 225 Ac-SSO110 (Ariceum Therapeutics) for Small Cell Lung Cancer (SCLC)

About 19 drugs received the fast track designation as small molecules, biologic, protein, radiopharmaceutical, and cell & gene therapy 

A Quick Look

Small Molecule: ERapa (Biodexa) for Familial Adenomatous Polyposis, BR55 (Bracco Imaging) for Crohn's disease, Amezalpat (Tempest Therapeutics) for Hepatocellular Carcinoma, PP-01 (PleoPharma) for Cannabis Withdrawal Symptoms, NNZ-2591 (Neuren) for Pitt Hopkins syndrome, SIGX1094 (Signet Therapeutics) for Gastric Cancer, AUTX-703 (Auron Therapeutics) for Acute Myelogenous Leukemia, Cell & Gene Therapy: ADI-001 (Adicet Bio) for Systemic Sclerosis), ADI-001 (Adicet Bio) for Systemic Lupus Erythematosus, AVB-114 (Avobis Bio) for Crohn's Perianal Fistulas, BRTX-100 (BioRestorative) for Chronic lumbar disc disease, RZ-001 (Rznomics) for Hepatocellular Carcinoma, Troculeucel (NKGen Biotech) for Moderate Alzheimer’s Disease, Biologic: Amlenetug (Lundbeck) for Multiple System Atrophy, IBI363 (Innovent ) for Squamous NSCLC, PYX-201 (Pyxis Oncology) for Recurrent or Metastatic Head and Neck Cancer, CUSP06 (OnCusp Therapeutics) for Platinum-Resistant Ovarian Cancer (PROC), Radiopharmaceutical: Cu-SAR-bisPSMA (Clarity Pharmaceuticals) for Metastatic castration-resistant prostate cancer, Protein: Rezpegaldesleukin (Nektar Therapeutics) for Moderate-to-Severe Atopic Dermatitis

Seven drugs ranging from biologic and small molecule to cell therapy were given breakthrough therapy designation

A Quick Look

Biologic: Petosemtamab (Merus N.V.) for Recurrent or metastatic PD-L1 positive head and neck squamous cell carcinoma, Opdivo + Yervoy (BMS) for MSI-H/dMMR colorectal cancer, Lacutamab (Innate Pharma) for Sézary Syndrome, HS-20093 (Hansoh Pharma) for Osteosarcoma, Small Molecule: Albumin-bound Sirolimus (CSPC Pharmaceutical) for Perivascular Epithelioid Cell Tumor, Radiprodil (GRIN Therapeutics) for GRIN-related Neurodevelopmental Disorder, Cell Therapy: SkinTE (PolarityBio) for Wagner Grade I Diabetic Foot Ulcers (DFU) 

16 Drugs ranging from small molecule and biologic to gene therapy and radiopharmaceutical were given priority review

A Quick Look

Small Molecule: Zongertinib (Boehringer Ingelheim) for ERBB2-mutant NSCLC, Brensocatib (Insmed) for Non-Cystic Fibrosis Bronchiectasis, Troriluzole (Biohaven) for Spinocerebellar Ataxia, Lenacapavir (Gilead Sciences) for HIV, Vatiquinone (PTC Therapeutics) for Friedreich's Ataxia, Dordaviprone (Chimerix) for H3 K27M-mutant Diffuse Glioma, Lenacapavir (Gilead) for HIV Prevention, Biologic: Sintilimab + Ipilimumab (Innovent Biologics) for MSI-H/dMMR Colon Cancer, Xinyue (Hansoh Pharmaceutical) for Immunoglobulin G4-related Disease,  Gamifant (Sobi) for Hemophagocytic Lymphohistiocytosis (HLH)/Macrophage Activation Syndrome (MAS), Keytruda (Merck) for Head and Neck Squamous Cell Carcinoma, Opdivo + Yervoy (BMS) for MSI-H/dMMR colorectal cancer, Dupixent (Regeneron & Sanofi) for Bullous Pemphigoid, Gene Therapy: UX111 (Ultragenyx) for Sanfilippo syndrome type A, PRGN-2012 (Precigen) for Recurrent Respiratory Papillomatosis, Radiopharmaceutical: Zircaix (Telix Pharmaceuticals) for Kidney Cancer Detection  

Three drugs received the rare pediatric disease designation by the US FDA

A Quick Look

Gene Therapy: ABO-101 (Arbor Biotechnologies) for Primary Hyperoxaluria Type 1, EN-374 (Ensoma) for Chronic Granulomatous Disease, Small Molecule: NNZ-2591 (Neuren) for Pitt Hopkins syndrome & Angelman syndrome 

Four devices received the breakthrough device designation by the US FDA 

A Quick Look

Serial CTRS AI Model (Onc.AI) for CT Scans Analysis, Lipiojoint (Guerbet) for Knee Osteoarthritis, ColoSeal (Averto Medical) for Temporary Ostomy, Sensorized Soft Tissue Anchor System (Penderia Technologies) for Orthopedic Soft Tissue Repair 

Seven drugs received the regenerative medicine advanced therapy designation as cell & gene therapies 

A Quick Look

Gene Therapy: Vigil (Gradalis) for Ovarian Cancer, BS01 (Bionic Sight) for Retinitis Pigmentosa, AB-1005 (AskBio) for Parkinson’s Disease, EXG-34217 (Elixirgen Therapeutics) for Telomere Biology Disorders,  Cell Therapy: NXC-201 (Immix Biopharma) for R/R AL Amyloidosis,  Anktiva + CAR-NK (ImmunityBio) for Pancreatic Cancer, RPESC-RPE-4W (Luxa Biotechnology) for Dry Age-Related Macular Degeneration 

For the complete report, reach out to us at connect@pharmashots.com