Company Product Description Indication Status
Alexion Pharmaceuticals Inc., of Boston Ultomiris (ravulizumab) C5 complement inhibitor Atypical hemolytic uremic syndrome  European Commission granted marketing authorization to treat adults and children with body weight of 10 kg or above 
Aptose Biosciences Inc., of San Diego CG-806 BTK/FLT3 tyrosine kinase inhibitor Acute myeloid leukemia FDA cleared IND to initiate phase Ia/b trial at starting dose of 450 mg twice daily; trial expected to open enrollment in second half of 2020 in individuals with relapsed, resistant or refractory disease
Aquestive Therapeutics Inc., of Warren, N.J. AQST-108 (epinephrine, sublingual soluble film) Adrenergic receptor agonist Anaphylaxis FDA cleared IND for pharmacokinetic trials, expected to begin by year-end 2020; program will be reviewed under 505(b)(2) pathway
Cancer Prevention Pharmaceuticals Inc., of Tucson, Ariz. CPP-1X (eflornithine) + sulindac Cyclooxygenase inhibitor; ornithine decarboxylase inhibitor Familial adenomatous polyposis  NDA submitted to FDA, seeking accelerated approval to treat indication in adults
Cytocom Inc., of Winter Park, Fla. CYTO-201 Inhibits replication of SARS-CoV-2 virus  COVID-19 Completed type B pre-IND meeting with FDA in relation to planned phase II trial; based on agency’s feedback, company plans to revise protocol and quickly initiate enrollment
Emmaus Life Sciences Inc., of Torrance, Calif. Endari  L-glutamine oral powder Sickle cell disease Issued license from Israeli Ministry of Health granting marketing authorization for use in reducing acute complications of sickle cell disease in adult and pediatric patients 5 and older
Genentech, of South San Francisco, a member of the Roche Group Phesgo (pertuzumab/trastuzumab) Fixed-dose combination of HER2-targeting antibodies with hyaluronidase HER2-positive breast cancer FDA approved for use in combination with intravenous chemotherapy for early and metastatic disease; administered via single subcutaneous injection
Goliver Therapeutics SA, of Nantes, France Undisclosed Cell therapy Acute liver failure EMA provided scientific advice on clinical development, pharmaco-toxicological and production strategy plans; company is working with Paul-Brousse Hospital on design and implementation of a phase I/IIa trial 
Heron Therapeutics Inc., of San Diego HTX-011 (bupivacaine + meloxicam) Cyclooxygenase 2 inhibitor; sodium channel inhibitor Management of postoperative pain FDA issued complete response letter, seeking additional nonclinical information; company plans to request type A meeting to obtain agreement with the FDA for NDA resubmission
Incyte Corp., of Wilmington, Del., and Novartis AG, of Basel, Switzerland Tabrecta (capmatinib) Kinase inhibitor that targets MET Non-small-cell lung cancer Japan’s Ministry of Health, Labour and Welfare approved for use in MET exon 14 skipping mutation-positive advanced and/or recurrent unresectable NSCLC; Tabrecta approved for first-line and previously treated patients, regardless of prior treatment type
Intercept Pharmaceuticals Inc., of New York Obeticholic acid FXR agonist Fibrosis due to nonalcoholic steatohepatitis FDA issued complete response letter, recommending submission of additional post-interim analysis of efficacy and safety data from ongoing Regenerate study in support of potential accelerated approval and said long-term outcomes phase of the study should continue
Merz Pharmaceuticals GmbH, of Frankfurt, Germany, and Teijin Pharma Ltd., of Tokyo Xeomin (incobotulinumtoxinA)  Botulinum toxin type A Upper limb spasticity Teijin received marketing approval from Japan’s Ministry of Health, Labor and Welfare 
Nordic Nanovector ASA, of Oslo, Norway Betalutin (177Lu lilotomab satetraxetan) CD37-targeting antibody-radionuclide-conjugate Relapsed or refractory marginal zone lymphoma FDA granted fast track designation for use in adults who have received at least 2 prior therapies
Novartis AG, of Basel, Switzerland Atectura  Fixed-dose combination of indacaterol acetate, mometasone furoate Asthma Approved by Japan’s Ministry of Health, Labour and Welfare
Novartis AG, of Basel, Switzerland Enerzair  Fixed-dose combination of glycopyrronium bromide, indacaterol acetate, mometasone furoate Asthma Approved by Japan’s Ministry of Health, Labour and Welfare
Novartis AG, of Basel, Switzerland Entresto (sacubitril valsartan sodium hydrate) Inhibits neprilysin and blocks angiotensin II type-I receptor Chronic heart failure Approved by Japan’s Ministry of Health, Labour and Welfare
Novartis AG, of Basel, Switzerland Mayzent (siponimod fumaric acid) Sphingosine 1-phosphate receptor modulator Secondary progressive multiple sclerosis Approved by Japan’s Ministry of Health, Labour and Welfare
Novo Nordisk A/S, of Bagsvaerd, Denmark Rybelsus (oral semaglutide) Oral GLP-1 receptor agonist Type 2 diabetes Japan’s Ministry of Health, Labour and Welfare approved for use in adults
PTC Therapeutics Inc., of South Plainfield, N.J. Translarna (ataluren) Protein restoration therapy Duchenne muscular dystrophy EMA’s Committee for Medicinal Products for Human Use recommended by a majority of votes to remove the statement "efficacy has not been demonstrated in non-ambulatory patients”; opinion is subject to final approval by European Commission
Rakuten Medical Inc., of San Mateo, Calif. ASP-1929 Antibody-drug conjugate comprising EGFR-targeting cetuximab and Irdye 700DX, a light activatable dye Recurrent head and neck cancer Japan’s Ministry of Health, Labour and Welfare said application will be reviewed under conditional early approval system; Rakuten submitted Japanese BLA in March 2020 based on phase I/II results
Roche Holding AG, of Basel, Switzerland Enspryng (satralizumab) IL-6-targeting monoclonal antibody Neuromyelitis optica spectrum disorder Japan’s Ministry of Health, Labour and Welfare approved for the prevention of relapses of NMOSD, including NMO, for aquaporin-4 antibody seropositive adults and children
Rocket Pharmaceuticals Inc., of New York RPL-401 Lentiviral vector-based gene therapy Infantile malignant osteopetrosis FDA cleared the IND for a phase I trial to enroll 2 pediatric patients, 1 month of age or older
SFA Therapeutics Inc., of Philadelphia SFA-002 Microbiome-derived metabolite Mild to moderate psoriasis FDA cleared IND for open-label, 30-subject trial

Notes

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

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