Company Product Description Indication Status
Aerie Pharmaceuticals Inc., of Durham, N.C. Rhopressa (netarsudil ophthalmic solution) Rho kinase inhibitor Open-angle glaucoma or ocular hypertension FDA approved sterile fill production facility in Athlone, Ireland, for production of Rhopressa for commercial distribution in U.S.
Amryt plc, of Dublin AP-103 Gene therapy Dystrophic epidermolysis bullosa EMA’s Committee for Orphan Medicinal Products adopted a positive opinion for orphan designation
Apellis Pharmaceuticals Inc., of Waltham, Mass. Pegcetacoplan Targeted C3 inhibitor Paroxysmal nocturnal hemoglobinuria  Submitted NDA to the FDA and MAA to the EMA
Ardelyx Inc., of Fremont, Calif. Tenapanor Acts locally in the gut to inhibit sodium hydrogen exchanger 3 Chronic kidney disease FDA accepted NDA seeking approval to control serum phosphorus in adults with CKD on dialysis
Astrazeneca plc, of Cambridge, U.K. Koselugo (selumetinib) Mitogen-activated protein kinase kinases 1 and 2 inhibitor Neurofibromatosis type 1 FDA issued a rare pediatric disease priority review voucher; to be published in Federal Register Sept. 16, 2020
Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis Jardiance (empagliflozin) SGLT2 inhibitor Heart failure FDA granted fast track designation for development to prevent hospitalization for heart failure and reduce risk of mortality in patients, with and without diabetes, who have had an acute myocardial infarction
Bristol Myers Squibb Co., of New York Opdivo (nivolumab) and Yervoy (ipilimumab)  PD-1 inhibitor and CTLA4 inhibitor Malignant pleural mesothelioma EMA validated a type II variation application seeking use in patients with previously untreated, unresectable disease; validation begins the EMA’s centralized review process
Epimab Biotherapeutics Inc., of Shanghai EMB-02 Bispecific antibody targeting PD-1 and LAG-3 Solid tumors Received study may proceed letter from FDA in response to IND
Equillium Inc., of La Jolla, Calif. Itolizumab (EQ-001) Monoclonal antibody targeting CD6-ALCAM pathway COVID-19 Completed pre-IND meeting with FDA under Coronavirus Treatment Acceleration Program; with FDA feedback, company aims to finalize protocol and submit IND for phase III study during fourth quarter of 2020
Gensight Biologics SA, of Paris Lumevoq (lenadogene nolparvovec) Recombinant adeno-associated viral vector, serotype 2, containing cDNA encoding human wild-type mitochondrial NADH dehydrogenase 4 protein Leber hereditary optic neuropathy Submitted MAA to the EMA seeking approval to treat patients with vision loss due to LHON caused by mutation in the ND4 mitochondrial gene
Gilead Sciences Inc., of Foster City, Calif. Magrolimab Anti-CD47 monoclonal antibody Myelodysplastic syndrome FDA granted breakthrough therapy designation for treating newly diagnosed MDS
Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda Mavrilimumab Monoclonal antibody targeting GM-CSFRα Giant cell arteritis FDA granted orphan designation
Mateon Therapeutics Inc., of Agoura Hills, Calif. Artemisinin Purified from Artemisia annua; inhibits TGF-β activity COVID-19 Global study based on ARTI-19 protocol cleared for patient enrollment in India; study being conducted in India as part of co-development agreement with Windlas Biotech Pvt Ltd.
Matinas Biopharma Holdings Inc., of Bedminster, N.J. MAT-9001 Omega-3 therapy Severe hypertriglyceridemia  Minutes from end-of-phase II meeting with FDA confirmed alignment on key next steps for phase III program and registration pathway; company remains on track to start phase III in first half of 2021
Novartis AG, of Basel, Switzerland Beovu (brolucizumab) Humanized single-chain antibody fragment Wet age-related macular degeneration EMA’s Committee for Medicinal Products for Human Use approved update to summary of product characteristics to include additional information regarding retinal vasculitis and retinal vascular occlusion
Plus Therapeutics Inc., of Austin, Texas Rhenium Nanoliposomes Nanoliposome-encapsulated radionuclide Recurrent glioblastoma FDA granted fast track designation
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan Hyqvia (human normal immunoglobulin 10% and recombinant human hyaluronidase) Subcutaneous immunoglobulin replacement therapy Secondary immunodeficiencies EMA approved label update for use in adults, adolescents and children with secondary immunodeficiencies who suffer from severe or recurrent infections, ineffective antimicrobial treatment and either proven scientific antibody failure or serum IgG level of <4 g/l
Zynerba Pharmaceuticals Inc., of Devon, Pa. Zygel  CBD transdermal gel Fragile X syndrome FDA said it will meet with company via teleconference in fourth quarter of 2020 to discuss pivotal Connect-FX data and regulatory path forward

Notes

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

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