Company Product Description Indication Status
Aeglea Biotherapeutics Inc., of Austin, Texas ACN-00177 Cystathionine gamma-lyase stimulator Homocystinuria FDA granted rare pediatric disease designation
Amicus Therapeutics Inc., of Cranbury, N.J. AT-GAA (ATB-200 + miglustat) Enzyme replacement therapy + alpha-glucosidase stimulator Pompe disease Rolling BLA submission to FDA initiated to treat late onset disease
Autobahn Therapeutics Inc., of San Diego ABX-002 Thyroid hormone receptor beta agonist Adrenomyeloneuropathy Completed pre-IND meeting with FDA and gained alignment on dose selection strategy and patient inclusion criteria for phase Ia/b trial and on proposal to conduct single phase IIb/III adaptive trial to support potential registration
Bridgebio Pharma Inc. and QED Therapeutics Inc., both of Palo Alto, Calif. Infigratinib FGFR1-3 inhibitor Cholangiocarcinoma FDA accepted NDA application, granting priority review under Real-Time Oncology Review pilot program
Cerecor Inc., of Rockville, Md. CERC-803 (L-fucose) Substrate replacement therapy Leukocyte adhesion deficiency type II FDA cleared IND for pivotal trial expected to begin by first half of 2021 and to report top-line data in second half of 2021
Genentech, of South San Francisco, a member of the Roche Group Gavreto (pralsetinib)  Metastatic RET fusion-positive non-small-cell lung cancer RET-mutant medullary thyroid cancer  Approved by FDA for treatment of adult and pediatric patients 12 and older with advanced or metastatic disease who require systemic therapy, or with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory 
Gilead Sciences Canada Inc., unit of Gilead Sciences Inc., of Foster City, Calif. Descovy (emtricitabine + tenofovir alafenamide) Antiretroviral therapy HIV pre-exposure prophylaxis  Health Canada granted notice of compliance to reduce risk of sexually acquired HIV-1 infection
Incannex Healthcare Ltd., of Melbourne, Australia IHL-675 (hydroxychloroquine + cannabidiol fixed-dose) Anti-inflammatory COVID-19 infection Requested expedited pre-IND meeting with FDA to pursue emergency use authorization to treat COVID-19-related sepsis-associated acute respiratory distress syndrome
Ipsen SA, of Paris Onivyde (irinotecan sucrosofate) Topoisomerase I inhibitor Small-cell lung cancer  FDA granted fast track designation
Janssen-Cilag International NV, unit of Johnson & Johnson, of New Brunswick, N.J. Ad26.COV2.S (JNJ-78436735) SARS-CoV-2 recombinant adenoviral vector vaccine COVID-19 infection EMA initiated rolling review
Merz Therapeutics, unit of Merz Pharmaceuticals GmbH, of Frankfurt Xeomin (incobotulinumtoxinA) Neuromuscular blocking agent Excessive drooling Health Canada granted notice of compliance to treat condition associated with neurological disorders in adults
Minerva Neurosciences Inc., of Waltham, Mass. Roluperidone 5-HT 2a receptor antagonist; opioid receptor sigma antagonist 2 Schizophrenia In type C meeting minutes, FDA advised that NDA submission based on current data from phase IIb and III studies would be unlikely to be filed and would prompt review issues due to lack of 2 well-controlled trials to support efficacy claims to treat negative symptoms of disease
Moderna Inc., of Cambridge, Mass. mRNA-1273 COVID-19 spike glycoprotein modulator COVID-19 infection FDA scheduled Vaccines and Related Biological Products advisory committee meeting on Dec. 17, 2020, to discuss request for emergency use authorization 
Moderna Biotech Spain SL, unit of Moderna Inc., of Cambridge, Mass.  mRNA-1273 COVID19 spike glycoprotein modulator COVID-19 infection Application for conditional marketing authorization submitted to EMA, requesting accelerated assessment
Moleculin Biotech Inc., of Houston WP-1066 JAK inhibitor; C-myc binding protein inhibitor; STAT-3 inhibitor; hypoxia inducible factor-1 alpha inhibitor Solid tumors FDA granted rare pediatric disease designations to treat diffuse intrinsic pontine glioma, medulloblastoma and atypical teratoid rhabdoid tumor
Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany BNT-162b2 COVID-19 spike glycoprotein modulator COVID-19 infection Application for conditional marketing authorization submitted to EMA, which will proceed under accelerated assessment
Rhizen Pharmaceuticals SA, of La Chaux du Fonds, Switzerland RP-7214 Dihydroorotate dehydrogenase inhibitor COVID-19 infection FDA cleared IND for phase I trial in healthy volunteers
Roche Holding AG, of Basel, Switzerland Xolair (omalizumab) Antibody designed to target and block immunoglobulin E Nasal polyps FDA approved supplemental BLA for add-on maintenance treatment of nasal polyps in adult patients 18 and older with inadequate response to nasal corticosteroids
Shanghai Junshi Biosciences Co. Ltd., of Shanghai JS-006 TIGIT receptor inhibitor Solid tumors China NMPA accepted IND
Tearclear Corp., of Boston Latanoprost PGF2 alpha agonist Glaucoma Completed pre-IND meeting with FDA regarding development plan for preservative-free multidose delivery of proprietary formulation, including design for registration study and IND filing in mid-2021 via 505(b)(2) pathway
TG Therapeutics Inc., of New York Ublituximab Anti-CD20 monoclonal antibody Chronic lymphocytic leukemia Rolling BLA submission to FDA initiated, with completion expected in first half of 2021
VBI Vaccines Inc., of Cambridge, Mass. Sci-B-Vac  Hepatitis B large envelope protein modulator Hepatitis B virus infection BLA submitted to FDA covering 3-antigen prophylactic HBV vaccine candidate

Notes

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