Company Product Description Indication Status
Abbvie Inc., of North Chicago Atogepant CGRP antagonist Migraine FDA accepted NDA for preventive treatment in adults who meet criteria for episodic migraine; decision expected in late third quarter of 2021
Akebia Therapeutics Inc., of Cambridge, Mass. Vadadustat HIF prolyl hydroxylase inhibitor Anemia NDA submitted to FDA to treat disease in adults with chronic kidney disease, whether on dialysis or not
Amarin Corp. plc, of Dublin Vazkepa (icosapent ethyl) Apolipoprotein B antagonist; phospholipase A2 inhibitor Cardiovascular disease European Commission approved MAA to reduce risk of cardiovascular events in high-risk, statin-treated adults with triglycerides ≥150 mg/dL and either established cardiovascular disease or diabetes and at least 1 additional cardiovascular risk factor
Biom Therapeutics LLC, of Sarasota, Fla. BIO-017 GPCR 55/TRPV1 agonist Angelman syndrome FDA granted orphan drug designation
Curevo Vaccine Inc., of Seattle CRV-101 Varicella zoster virus subunit adjuvant vaccine  Varicella infection South African Health Products Regulatory Authority cleared initiation of phase Ib pediatric trial to prevent disease in immunocompromised HIV-infected children
Eisai Co. Ltd., of Tokyo Lenvima/Kisplyx (lenvatinib) Multiple receptor tyrosine kinase inhibitor Renal cell and endometrial carcinoma  EMA accepted MAAs for combination with anti-PD-1 Keytruda (pembrolizumab, Merck & Co. Inc.) to treat people with advanced disease
Genmab A/S, of Copenhagen, Denmark Kesimpta (ofatumumab) B-lymphocyte antigen CD20 inhibitor Multiple sclerosis European Commission approved MAA from developer Novartis AG to treat adults with relapsing forms of active disease
Horizon Therapeutics plc, of Dublin Tepezza (teprotumumab) Insulin-like growth factor 1 receptor antagonist Thyroid eye disease FDA approved a prior approval supplement to previously approved BLA, giving company authorization to manufacture more Tepezza drug product, resulting in increased number of vials with each manufacturing slot; company plans to resupply the market beginning April 2021, ending supply disruption that began in December 2020 due to prioritizing COVID-19 vaccine manufacturing
I-Mab, of Shanghai, and ABL Bio Inc., of Gaithersburg, Md. TJ-CD4B (ABL-111) CDw137 agonist; claudin 18 modulator Solid tumors FDA cleared IND for phase I trial in people with advanced/metastatic disease
Incyte Corp., of Wilmington, Del. Pemazyre (pemigatinib) FGF1/FGF2/FGF3 receptor antagonist Cholangiocarcinoma European Commission approved MAA to treat adults with locally advanced/metastatic disease with FGFR2 fusion or rearrangement who progressed after at least 1 line of systemic therapy
Intellia Therapeutics Inc., of Cambridge, Mass. NTLA-2001 CRISPR-associated endonuclease Cas9 modulator Transthyretin amyloidosis EMA granted orphan drug designation
JCR Pharmaceuticals Co. Ltd., of Hyogo, Japan JR-171 Alpha-L-iduronidase modulator Mucopolysaccharidosis type I (Hurler syndrome) EMA granted orphan drug designation
Karyopharm Therapeutics Inc., of Newton, Mass. Nexpovio (selinexor) Exportin 1 inhibitor Multiple myeloma European Commission approved MAA, in combination with dexamethasone, to treat adults who received at least 4 prior therapies and whose disease is refractory to at least 2 proteasome inhibitors, 2 immunomodulatory agents and an anti-CD38 monoclonal antibody and who showed disease progression
Merck & Co. Inc., of Kenilworth, N.J. Keytruda (pembrolizumab) Monoclonal antibody targeting PD-1 High-risk early stage triple-negative breast cancer FDA issued a complete response letter for the sBLA, saying that a regulatory decision should be deferred until further data are available from KEYNOTE-522; the next interim analysis will occur in 3Q2021
Merck & Co. Inc., of Kenilworth, N.J. Keytruda (pembrolizumab) Monoclonal antibody targeting PD-1 Locally advanced or metastatic urothelial cancer Received a positive EMA Committee for Medicinal Products for Human Use opinion, recommending that the European label be updated to include results of the phase III KEYNOTE-361 trial
Moleculin Biotech Inc., of Houston Annamycin Next-generation anthracycline Soft tissue sarcoma lung metastases FDA granted fast track designation
Myovant Sciences Ltd., of Basel, Switzerland Relugolix Gonadotropin-releasing hormone receptor antagonist Advanced prostate cancer EMA validated the MAA, confirming that the submission is sufficiently complete to begin the formal review process
PTC Therapeutics Inc., of South Plainfield, N.J. Evrysdi (risdiplam) SMN2-directed RNA splicing modifier Spinal muscular atrophy European Commission granted marketing authorization in patients 2 months and older, with a clinical diagnosis of SMA type 1, type 2 or type 3 with 1 to 4 SMN2 copies
Russian Direct Investment Fund, of Moscow Sputnik V Adenovirus-based vaccine COVID-19 prophylaxis Authorized in Mali
Stoke Therapeutics Inc., of Bedford, Mass. STK-001 Antisense oligonucleotide targeting SCN1A Dravet syndrome MHRA authorized its clinical trial application to initiate a phase I/IIa trial in the U.K.
Tetra Bio-Pharma Inc., of Ottawa ARDS-003 Cannabinoid CB2 receptor agonist COVID-19 patients at risk for developing acute respiratory distress syndrome Health Canada acknowledged that the preclinical safety data are robust and sufficient to file a clinical trial application
Tiziana Life Sciences plc, of New York and London Foralumab Anti-CD3 monoclonal antibody Secondary progressive multiple sclerosis FDA has allowed evaluation of nasal administration in a patient, under an individual patient expanded access program
Vifor Pharma Group, of St. Gallen, Switzerland, and Cara Therapeutics Inc., of Stamford, Conn. Difelikefalin injection Opioid receptor kappa agonist Pruritus associated with chronic kidney disease in hemodialysis patients EMA accepted the MAA for review

Notes

For more information about individual companies and/or products, see Cortellis.