Company Product Description Indication Status
Abbvie Inc., of North Chicago Humira (adalimumab) Monoclonal antibody targeting tumor necrosis factor Moderate to severe active ulcerative colitis or ulcerative colitis Brazil’s ANVISA approved use in pediatric patients, from age of 6, who showed inadequate response to conventional therapy, including corticosteroids and/or 6-mercaptopurine or azathioprine, or who are intolerant or have medical contraindications for those therapies 
Agios Pharmaceuticals Inc., of Cambridge, Mass. Tibsovo (ivosidenib tablets) IDH1 gene inhibitor Cholangiocarcinoma Submitted supplemental NDA to FDA seeking use in patients with previously treated IDH1-mutated disease; Agios requested priority review
Arrowhead Pharmaceuticals Inc., of Pasadena, Calif. ARO-APOC3 RNAi therapeutic Hypertriglyceridemia  Submitted IND to FDA for phase IIb dose-finding study
Astrazeneca plc, of Cambridge, U.K. COVID-19 vaccines Viral vector-based vaccines COVID-19 Health Canada authorized 2 vaccines: 1 is manufactured by Astrazeneca and developed with Oxford University and 1 is Serum Institute of India’s version of the Astrazeneca vaccine
Athenex Inc., of Buffalo, N.Y. Oraxol Oral paclitaxel plus encequidar  Metastatic breast cancer FDA issued complete response letter, indicating concern of safety risk to patients in terms of an increase in neutropenia-related sequelae in oral paclitaxel arm vs. I.V. paclitaxel; FDA also expressed concerns regarding uncertainty over results of primary endpoint of objective response rate at week 19 conducted by blinded independent central review
Avadel Pharmaceuticals plc, of Dublin FT-218 Once-nightly formulation of sodium oxybate Excessive daytime sleepiness and cataplexy in adults with narcolepsy FDA accepted for review the NDA, setting PDUFA date of Oct. 15, 2021
Avrobio Inc., of Cambridge, Mass.  AVR-RD-04 Gene therapy Cystinosis European Commission granted orphan designation
Bridgebio Pharma Inc., of Palo Alto, Calif., and affiliate Origin Biosciences Inc. Nulibry (fosdenopterin) Substrate replacement therapy Molybdenum cofactor deficiency type A Approved by FDA to reduce risk of mortality in patients with MoCD type A
Carsgen Therapeutics Holdings Ltd., of Shanghai CT-041 Claudin 18.2-targeted CAR T product Gastric cancer European Commission granted orphan designation
Cormedix Inc., of Berkeley Heights, N.J. Defencath (taurolidine/heparin catheter lock solution) Small molecule; antibacterial Prevention of catheter-related bloodstream infections in hemodialysis patients FDA issued complete response letter, noting concerns at third-party manufacturing facility; agency also is requiring a manual extraction study 
Flexion Therapeutics Inc., of Burlington, Mass. FX-301 Locally administered NaV1.7 inhibitor formulated for extended release in thermosensitive gel Non-opioid pain relief FDA cleared IND; phase Ib study in patients undergoing bunionectomy expected to start in first half of 2021
Idorsia Ltd., of Allschwil, Switzerland Clazosentan Selective endothelin A receptor antagonist Prevention of cerebral vasospasm, vasospasm-related cerebral infarction and cerebral ischemic symptoms after aneurysmal subarachnoid hemorrhage Submitted NDA to Japanese PMDA
Johnson & Johnson, of New Brunswick, N.J. COVID-19 vaccine Vaccine COVID-19 FDA issued emergency use authorization for the single-dose vaccine in individuals 18 and older
Krystal Biotech Inc., of Pittsburgh KB-407 CFTR gene modulator Cystic fibrosis EMA issued positive opinion on orphan designation
LFB SA, of Les Ulis, France Eptacog beta Recombinant coagulation factor VIIa  Hemophilia EMA accepted for filing the MAA 
Merck & Co. Inc., of Kenilworth, N.J. Gefapixant Selective P2X3 receptor antagonist Refractory chronic cough FDA accepted for review the NDA; PDUFA date of Dec. 21, 2021
Oncopeptides AB, of Waltham, Mass. Pepaxto (melphalan flufenamide) Peptide-drug conjugate  Relapsed or refractory multiple myeloma Approved by FDA for use in adults who have received at least 4 prior lines of therapy and whose disease is refractory to at least 1 proteasome inhibitor, 1 immunomodulatory agent and 1 CD38-directed monoclonal antibody; granted under accelerated approval
Oncorena AB, of Lund, Sweden Orellanine Fungus toxin Metastatic renal cancer Swedish Medical Products Agency approved start of phase I/II trial in patients with metastatic renal cancer in dialysis
Plx Pharma Inc., of Sparta, N.J. Vazalore 325 mg and Vazalore 81 mg Liquid-filled aspirin capsule Platelet inhibition FDA approved supplemental NDAs
Qurebio Ltd., of Shanghai Q-1802 Bispecific antibody targeting PD-L1 and Claudin18.2 Tumors FDA granted IND approval
Reata Pharmaceuticals Inc., of Plano, Texas Bardoxolone methyl Orally administered activator of Nrf2 Chronic kidney disease caused by Alport syndrome Submitted NDA to FDA
Russian Direct Investment Fund, of Moscow Sputnik V COVID-19 vaccine COVID-19 Approved in Republic of Moldova
Sanofi SA, of Paris Sarclisa (isatuximab)   Monoclonal antibody that binds to specific epitope on CD38 receptor Multiple myeloma EMA’s Committee for Medicinal Products for Human Use adopted positive opinion for use in combination with carfilzomib and dexamethasone to treat adults who have received at least 1 prior therapy
Sciwind Biosciences Co. Ltd., of Hangzhou, China XW-003 Biologic; unspecified drug target Nonalcoholic steatohepatitis China’s NMPA approved for clinical trial
Veru Inc., of Miami VERU-111 Tubulin alpha inhibitor; tubulin beta inhibitor COVID-19 FDA agreed in end-of-phase II meeting to advance drug into phase III registration study based on data from phase II study testing once-daily oral dosing vs. placebo in hospitalized COVID-19 patients at high risk for acute respiratory distress syndrome

Notes

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