Company Product Description Indication Regulatory status

Cancer

Affimed N.V., of Heidelberg, Germany AFM-24 Bispecific EGFR/CD16A innate cell engager Cancer Submitted IND for first trial of AFM13 pre-mixed with cord blood-derived allogeneic NK cells in patients with relapsed/refractory CD30-positive lymphoid malignancies; Registration-directed phase II on track to start this quarter
Agilent Technologies Inc., of Santa Clara, Calif. PD-L1 IHC 22C3 Pharmadx assay Locally advanced or metastatic non-small-cell lung cancers Approved by China’s NMPA as a companion diagnostic to identify patients whose tumors express PD-L1 for first-line treatment with single-agent Keytruda (pembrolizumab, Merck & Co. Inc.)
Astellas Pharma Inc., of Tokyo Xospata (gilteritinib) FLT3 inhibitor Relapsed or refractory acute myeloid leukemia European Commission approved as a monotherapy for treating adults whose disease has FLT3 mutation
Astellas Pharma Inc., of Tokyo Xospata (gilteritinib) FLT3 inhibitor Relapsed or refractory acute myeloid leukemia European Commission approved for use in adults with FLT3 mutations detected using Leukostrat CDx FLT3 mutation assay
Astrazeneca Canada, of Mississauga, Ontario Calquence (acalabrutinib) Bruton's tyrosine kinase inhibitor Second-line mantle cell lymphoma Health Canada approved the drug for sale
Astrazeneca plc, of Cambridge, U.K., and Daiichi Sankyo Co. Ltd., of Tokyo [Fam-] trastuzumab deruxtecan (DS-8201) HER2-targeting antibody-drug conjugate HER2-positive metastatic breast cancer FDA accepted the BLA and granted priority review; PDUFA date set for second quarter of 2020
Athenex Inc., of Buffalo, N.Y. Oral paclitaxel Paclitaxel chemotherapy combined P-gp encequidar Soft tissue sarcoma European Commission granted orphan designation
Bergenbio AS, of Bergen, Norway Bemcentinib Axl tyrosine kinase receptor inhibitor Acute myeloid leukemia FDA granted fast track designation to treat elderly patients with relapsed disease
Blueprint Medicines Corp., of Cambridge, Mass. Avapritinib Inhibits KIT and PDGFRA mutant kinases PDGFRA exon 18 mutant gastrointestinal stromal tumors FDA said it intends to split the proposed indications for 2 separate NDAs: 1 for PDGFRA exon 18 mutant GIST, regardless of prior therapy; and 1 for fourth-line GIST; an evaluation extension of the fourth-line indication will likely be required
Bristol-Myers Squibb Co., of New York Opdivo (nivolumab) Monoclonal antibody targeting PD-1 Melanoma with involvement of lymph nodes or metastatic disease European Commission approved the flat dosing schedule of 240 mg infused over 30 minutes every 2 weeks or 480 mg infused over 60 minutes every 4 weeks for patients who have undergone complete resection
Can-Fite Biopharma Ltd., of Petach Tikva, Israel Namodenoson Targets A3 adenosine receptor Hepatocellular cancer During an end-of-phase-II meeting, the FDA agreed with Can-Fite’s proposed design for a phase III study to support approval; study will enroll HCC patients with underlying Child Pugh B7 cirrhosis
Daiichi Sankyo Co. Ltd., of Tokyo Quizartinib Oral FLT3 inhibitor Relapsed/refractory FLT3-ITD acute myeloid leukemia EMA’s Committee for Medicinal Products for Human Use adopted negative opinion for the MAA
Denovo Biopharma LLC, of San Diego DB-102 Inhibits protein kinase C beta Newly diagnosed glioblastoma FDA cleared start of phase IIb trial in combination with radiation and temozolomide
Eagle Pharmaceuticals Inc., of Woodcliff Lake, N.J. Treakisym (bendamustine) PARP modulator Lymphoma; leukemia Said marketing partner Symbio Pharmaceuticals Ltd., of Tokyo, submitted NDA for ready-to-dilute liquid formulation in Japan, covering all approved indications
Epizyme Inc., of Cambridge, Mass. Tazemetostat EZH2 inhibitor Follicular lymphoma Completed pre-NDA meeting with FDA; on track to submit for accelerated approval in December 2019
Flavocure Biotech Inc., of Baltimore Caflanone (FBL-03G) Flavonoid Pancreatic cancer FDA granted orphan drug status
Genmab A/S, of Copenhagen Darzalex (daratumumab) Monoclonal antibody targeting CD38 Multiple myeloma The EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on broadening the existing marketing application to include Darzalex plus lenalidomide and dexamethasone as treatment for newly diagnosed adult patients who are ineligible for autologous stem cell transplant
Glaxosmithkline plc, of London Zejula (niraparib) PARP inhibitor Advanced ovarian, fallopian tube or primary peritoneal cancer FDA approved expanded indication for use in patients who have been treated with 3 or more prior chemotherapy regimens and whose cancer is associated with homologous recombination deficiency-positive status, defined as deleterious or suspected deleterious BRCA mutation or genomic instability with progression more than 6 months after response to last platinum-based chemotherapy
Iaso Biotherapeutics Co. Ltd., of Nanjing, China, and Innovent Biologics Inc., of Suzhou, China CT-103A Lentiviral vector containing CAR with fully human scFv, CD8a hinger and transmembrane, 4-1BB co-stimulatory and CD3z activation domains Relapsed/refractory multiple myeloma China’s NMPA cleared an IND for a phase Ib/II study
I-Mab Biopharma Co. Ltd., of Shanghai TJD-5 CD73 antibody Advanced solid tumors National Medical Products Administration approved the IND to start clinical trials in China
I-Mab Biopharma Co. Ltd., of Shanghai and Morphosys AG, of Martinsried, Germany TJ202/MOR202 Monoclonal antibody targeting CD38 Multiple myeloma National Medical Products Administration of China cleared the IND for phase II and phase III studies
Ipsen Biopharmaceuticals Canada Inc., of Mississauga, Ontario, part of Ipsen SA Cabometyx (cabozantinib) Kinase inhibitor targeting VEGFR, MET and AXL pathways Advanced renal cell carcinoma Approved by Health Canada for first-line treatment in adults
Isofol Medical AB, of Gothenburg, Sweden Arfolitixorin Active metabolite of folate-based drugs Metastatic colorectal cancer Japan's Pharmaceutical and Medical Devices Agency completed its review of the clinical trial notification, clearing the opening of Japanese sites in the ongoing, pivotal, phase III Agent study
Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson Darzalex (daratumumab) CD38-directed antibody Multiple myeloma FDA approved use in combination with bortezomib, thalidomide and dexamethasone for newly diagnosed patients who are eligible for autologous stem cell transplant
Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson Niraparib Poly ADP-ribose polymerase inhibitor Metastatic castration-resistant prostate cancer FDA granted breakthrough therapy designation for use in patients with BRCA1/2 gene-mutated disease after taxane chemotherapy and androgen receptor-targeted therapy
Janssen Pharmaceutical Cos., of Toronto, part of Johnson & Johnson Darzalex (daratumumab) CD38-directed monoclonal antibody Multiple myeloma Health Canada approved under priority review use in combination with lenalidomide and dexamethasone for newly diagnosed patients who are ineligible for autologous stem cell transplant
Kiadis Pharma NV, of Amsterdam, the Netherlands ATIR-101 T-cell-based therapeutic Hematopoietic stem cell transplant in blood cancers Expects EMA’s Committee for Medicinal Products for Human Use to issue negative opinion and recommend against conditional marketing authorization at November meeting; EMA feedback indicates phase II and historical T-cell deplete control data do not provide adequate support for MAA due to the evolution of standard of care with post-transplant cyclophosphamide; phase III study currently enrolling, with interim readout expected in 2021
Mallinckrodt plc, Staines-Upon-Thames, U.K. Uvadex (methoxsalen) Naturally occurring photoactive substance Cutaneous T-cell lymphoma Australia's TGA approved product for extracorporeal administration with the Therakos Cellex Photopheresis System for palliative treatment of skin manifestations of CTCL that is unresponsive to other forms of treatment
Merck & Co. Inc., of Darmstadt, Germany, and Pfizer Inc., of New York Bavencio (avelumab) PD-L1-inhibiting antibody Advanced renal cell carcinoma European Commission approved use in combination with axitinib for first-line treatment of adults
Merck & Co. Inc., of Kenilworth, N.J. Keytruda (pembrolizumab) Anti-PD-1 antibody Locally advanced or metastatic non-small-cell lung cancer Approved by China’s NMPA for use as a monotherapy in first-line treatment of patients whose tumors express PD-L1 as determined by NMPA-approved test, with no EGFR or ALK genomic tumor aberrations
Merck & Co. Inc., of Kenilworth, N.J. Keytruda (pembrolizumab) Monoclonal antibody targeting PD-1 First-line metastatic or unresectable recurrent head and neck squamous cell carcinoma The EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on expanding the marketing application to include Keytruda as a monotherapy or in combination with platinum therapy and 5-fluorouracil in patients whose tumors express PD-L1
Mereo Biopharma Group plc, of London Navicixizumab Delta-like protein ligand 4 inhibitor; VEGF receptor antagonist Ovarian, primary peritoneal or fallopian tube cancer FDA granted fast track designation to treat individuals who received at least 3 prior therapies
Nanjing Iaso Biotherapeutics Co. Ltd. of Nanjing CT-103A Fully-human BCMA CAR T Relapsed or refractory multiple myeloma China regulators granted an IND approval
Oncolytics Biotech Inc., of San Diego, and Adlai Nortye Biopharma Co. Ltd., of Hangzhou, China Pelareorep Non-pathogenic isolate of unmodified reovirus Metastatic breast cancer Adlai received China NMPA approval to start a phase III trial
Puma Biotechnology Inc., of Los Angeles Nerlynx (neratinib) Tyrosine kinase inhibitor targeting HER2/4 Early stage breast cancer FDA approved labeling supplement to include safety information based on interim results from phase II Control study showing the addition of prophylactic treatment with loperamide plus budesonide reduced discontinuation rate due to neratinib-associated diarrhea to 11% vs. discontinuation rate of 18% with loperamide alone
Sophiris Bio Inc., of San Diego Topsalysin (PRX-302) Pore-forming protein Intermediate-risk localized prostate cancer Following end-of-phase II/pre-phase III meeting with FDA, reached agreement on phase III design to enroll about 700 patients to test topsalysin against placebo, with primary endpoint of proportion of patients at 12 months who have failed treatment, defined as histological progression of disease, resulting in the need for alternative therapy
SQZ Biotechnologies Co., of Watertown, Mass. SQZ-PBMC-HPV Dual human papillomavirus E6/E7 protein modulator HPV-positive tumors FDA cleared IND application
Verastem Inc., of Boston Copiktra (duvelisib) Oral PI3K inhibitor T-cell lymphoma FDA granted orphan designation
Ziopharm Oncology Inc., of Boston CD19-specific CAR T therapy CAR T produced using rapid personalized manufacturing Relapsed CD19-positive leukemias and lymphomas FDA cleared an IND for a phase I study
Company Product Description Indication Regulatory status

Cardiovascular

Eton Pharmaceuticals Inc., of Deer Park, Ill. Biorphen (phenylephrine HCI) Alpha-1 adrenergic receptor agonist Hypotension FDA approved ready-to-use injectable formulation to treat hypotension resulting primarily from vasodilation in the setting of anesthesia
Mezzion Pharma Co. Ltd., of Seoul, South Korea Udenafil PDE5 inhibitor Single ventricle congenital heart disease with Fontan physiology Conducted type C meeting with U.S. FDA and is taking necessary steps to prepare and submit NDA based on results of Fuel phase III trial
Omeros Corp., of Seattle Narsoplimab Human monoclonal antibody targeting MASP-2 Hematopoietic stem cell transplant-associated thrombotic microangiopathy EMA issued positive opinion for pediatric investigation plan
Company Product Description Indication Regulatory status

Dermatologic

Amryt Pharma plc, of London AP-101 (Oleogel-S10) Cyclooxygenase 2 stimulator Epidermolysis bullosa FDA granted fast track designation
Arcutis Biotherapeutics Inc., of Westlake Village, Calif. ARQ-151 Topical formulation of PDE4 inhibitor roflumilast Plaque psoriasis Following an end-of-phase-II meeting with the FDA, the company announced plans to run a phase III study starting in the first half of 2020
Cabaletta Bio Inc., of Philadelphia DSG3-CAART Desmoglein-3 modulator Mucosal pemphigus vulgaris FDA cleared IND application to initiate first-in-human trial, expected to begin enrolling in 2020
Evolus Inc., of Newport Beach, Calif. Nuceiva (prabotulinumtoxinA) 900 kDa purified botulinum toxin type A formulation Glabellar lines European Commission approved for use to temporarily improve appearance of moderate to severe vertical lines between the eyebrows; decision application to all 28 EU member states plus Iceland, Norway and Liechtenstein
Foamix Pharmaceuticals Ltd., of Rehovot, Israel FMX-103 (minocycline, 1.5% topical foam) Tetracycline antibiotic Rosacea FDA accepted for review NDA for treatment of moderate to severe papulopustular rosacea in adults, setting June 2, 2020, PDUFA date
Foamix Pharmaceuticals Ltd., of Rehovot, Israel Amzeeq (FMX-101) Minocycline topical foam 4% Inflammatory lesions of non-nodular moderate to severe acne vulgaris Approved by FDA for use in adults and pediatric patients, 9 and older
Galderma SA, of Lausanne, Switzerland Aklief (trifarotene) Topical cream targeting retinoic acid receptor gamma Acne Approved by FDA
Janssen Pharmaceutical Cos., unit of Johnson & Johnson, of New Brunswick, N.J. Stelara (ustekinumab) Dual IL-12/IL-23 receptor antagonist Plaque psoriasis Submitted sBLA to FDA to treat moderate to severe disease in 6- to 11-year-olds
Krystal Biotech Inc., of Pittsburgh KB-105 Gene therapy Transglutaminase-1-deficient autosomal recessive congenital ichthyosis EMA’s Committee for Orphan Medicinal Products issued a positive opinion for orphan designation
Company Product Description Indication Regulatory status

Endocrine/metabolic

Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Patisiran RNAi therapeutic Hereditary transthyretin-mediated amyloidosis with polyneuropathy Filed marketing application with Brazilian regulatory agent ANVISA, which granted priority review; decision expected in first half of 2020
Ascendis Pharma A/S, of Copenhagen Transcon growth hormone Growth hormone ligand Pediatric growth hormone deficiency European Commission granted orphan designation as long-acting therapy for once-weekly treatment
Assertio Therapeutics Inc., of Lake Forest, Ill. Long-acting cosyntropin Synthetic adrenocorticotropic hormone Diagnostic drug to screen for adrenocortical insufficiency FDA issued a complete response letter; primary focus relates to agency determination that certain pharmacodynamic parameters were not adequately achieved
Astrazeneca plc, of Cambridge, U.K. Farxiga (dapagliflozin) SGLT2 inhibitor Type 2 diabetes FDA approved expanded use to reduce risk of hospitalization for heart failure in adults with type 2 diabetes and established cardiovascular disease or multiple cardiovascular risk factors
Avrobio Inc., of Cambridge, Mass. AVR-RD-02 Gene therapy expressing glucocerebrosidase Gaucher disease FDA granted orphan drug designation
Clinuvel Pharmaceuticals Ltd., of Melbourne, Australia Scenesse (afamelanotide) Melanocortin-1 receptor agonist Erythropoietic protoporphyria Approved by FDA to increase pain-free light exposure in adults with history of phototoxic reactions
Intarcia Therapeutics Inc., of Boston ITCA-650 (exenatide implant) Long-acting glucagon-like peptide-1 receptor agonist Type 2 diabetes mellitus FDA accepted the resubmitted NDA as a class 2 response; PDUFA date of March 9, 2020
Moderna Inc., of Cambridge, Mass. mRNA-3927 2 mRNAs encoding for alpha and beta subunits of mitochondrial enzyme propionyl-CoA carboxylase, encapsulated on lipid nanoparticle Propionic acidemia FDA granted fast track designation
Novo Nordisk A/S, of Bagsværd, Denmark Fiasp (insulin aspart injection) Insulin ligand; insulin receptor agonist Type 1 and 2 diabetes FDA expanded label for the drug at 100 u/mL to include use in insulin infusion pumps to improve glycemic control in adults
Provention Bio Inc., of Oldwick, N.J. PRV-031 (teplizumab) Anti-CD3 monoclonal antibody Prevention or delay of clinical type 1 diabetes EMA granted PRIME (priority medicines) designation for use in people at risk of developing the disease
Ultragenyx Pharmaceutical Inc., of Novato, Calif. UX007 (triheptanoin) Medium-chain triglyceride Long-chain fatty acid oxidation disorders FDA accepted the NDA and assigned a PDUFA action date of July 31, 2020
Visen Pharmaceuticals, of Shanghai Transcon Human growth hormone Growth hormone deficiency China’s NMPA cleared IND for a phase III study in childhood GHD
Company Product Description Indication Regulatory status

Gastrointestinal

Biostage Inc., of Holliston, Mass. Cellspan esophageal implant Combines biocompatible scaffold with a patient's own cells Surgical treatment of esophageal disease Submitted IND to FDA
Biovie Inc., of Santa Monica, Calif. BIV-201 Continuous infusion terlipressin Ascites due to chronic liver cirrhosis Submitted protocol for phase IIb/III study to the FDA; trial to test drug in patients who are refractory to or cannot tolerate diuretic therapy
Cipher Pharmaceuticals Inc., of Oakville, Ontario Trulance (plecanatide) Guanylate cyclase-C agonist Irritable bowel syndrome with constipation Health Canada approved the marketing application
Cytodyn Inc., of Vancouver, Wash. Leronlimab (PRO-140) CCR5 antagonist Nonalcoholic steatohepatitis FDA cleared the firm to start phase II trial
Heron Therapeutics Inc., of San Diego Cinvanti (aprepitant) NK1 receptor antagonist Chemotherapy-induced emesis FDA approved sNDA to expand recommended dosage to include 130-mg single-dose regimen to treat individuals with moderately emetogenic chemotherapy
Janssen Pharmaceutical Cos., of New Brunswick, N.J.-based Johnson & Johnson Stelara (ustekinumab) Targets IL-12 and IL-23 Moderately to severely active ulcerative colitis FDA expanded approval
Company Product Description Indication Regulatory status

Genitourinary/sexual function

Agile Therapeutics Inc., of Princeton, N.J. Twirla (levonorgestrel/ethinyl estradiol transdermal system) Hormonal contraceptive patch Prevention of pregnancy The FDA’s Bone, Reproductive and Urologic Drugs Advisory Committee voted 14 to 1, with 1 abstention, that the benefits of Twirla outweigh the risks to support approval
Amag Pharmaceuticals Inc., of Waltham, Mass. Makena (hydroxyprogesterone caproate injection) 17? hydroxyprogesterone caproate Preterm birth FDA’s Bone, Reproductive and Urologic Drugs Advisory Committee voted 9-7 in favor of recommending FDA withdrawal of Makena from the market
Astellas Pharma Inc., of Tokyo ASP-1128 Selective PPARdelta modulator Moderate to severe acute kidney injury FDA granted fast track status for patients at increased risk of development moderate to severe AKI after coronary artery bypass and/or valve surgery
Calliditas Therapeutics AB, of Stockholm Nefecon Modified-release oral formulation of corticosteroid budesonide IgA nephropathy EMA provided positive guidance on conditional marketing authorization of phase III asset
Cormedix Inc., of Berkeley Heights, N.J. Neutrolin Non-antibiotic antimicrobial solution Hemodialysis Completed interaction with FDA related to CMC package to support NDA; pre-NDA meeting now scheduled
Diamedica Therapeutics Inc., of Minneapolis DM-199 Recombinant human serine protease kallikrein Chronic kidney disease FDA accepted the clinical trial protocol for the phase II study; company plans to start enrollment in the next few weeks
Knight Therapeutics Inc., of Montreal, and Therapeuticsmd Inc., of Boca Raton, Fla. Joyesta (estradiol vaginal inserts) Estradiol softgel vaginal capsule Vulvar and vaginal atrophy Health Canada accepted for review Knight’s new drug submission for use in postmenopausal women who have symptoms due to estrogen deficiency
Medicines360, of San Francisco, and Allergan plc, of Dublin Liletta (levonorgestrel-releasing intrauterine system) Hormone-releasing system Contraception FDA approved supplemental NDA to extend duration of use for up to 6 years
Obseva SA, of Geneva Nolasiban Oral oxytocin receptor antagonist In vitro fertilization FDA cleared firm to start enrolling in phase III Implant 3 trial in women undergoing embryo transfer following IVF
Company Product Description Indication Regulatory status

Hematologic

Amgen Inc., of Thousand Oaks, Calif. Nplate (romiplostim) Thrombopoietin receptor agonist Immune thrombocytopenia FDA approved the supplemental BLA to include newly diagnosed and persistent adult ITP patients who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy
Bluebird Bio Inc., of Cambridge, Mass. Zynteglo (betibeglogene darolentivec ) Autologous CD34+ cells encoding beta-A-T87Q-globin gene Beta-thalassemia EMA approved refined commercial drug product manufacturing specifications
Chong Kun Dang Pharmaceutical Corp., of South Korea CKD-11101 Novel erythropoiesis stimulating protein (NESP) biosimilar Anemia Received approval for its novel erythropoiesis stimulating protein (NESP) biosimilar from Japan's Ministry of Health, Labour and Welfare (MHLW)
Janssen Pharmaceutical Co., of Raritan, N.J., a unit of New Brunswick, N.J.-based Johnson & Johnson Xarleto (rivaroxaban) Factor Xa inhibitor Prevention of venous thromboembolism in hospitalized acutely ill medical patients at risk for thromboembolic complications who are not at high risk of bleeding FDA approved the expanded label
Octapharma USA, of Hoboken, N.J. Wilate Human plasma-derived, sterile, purified, double virus inactivated von Willebrand factor/coagulation factor VIII complex Hemophilia A Approved by FDA for routine prophylaxis to reduce frequency of bleeding episodes and on-demand treatment and control of bleeding episodes
Pharmazz Inc., of Willowbrook, Ill. Centhaquine Increases cardiac output and decreases vascular resistance Hypovolemic shock and excessive blood loss Submitted marketing application to India’s Central Drugs Standard Control Organization, Director General of Health Services, Ministry of Health and Family Welfare
Rigel Pharmaceuticals Inc., of South San Francisco Fostamatinib Spleen tyrosine kinase inhibitor Chronic immune thrombocytopenia The EMA's Committee for Medicinal Products for Human Use adopted a positive trend vote on the MAA; final vote expected at the November meeting
Rocket Pharmaceuticals Inc., of New York RP-L301 Lentiviral vector-based gene therapy Pyruvate kinase deficiency FDA cleared IND application
Spectrum Pharmaceuticals Inc., of Henderson, Nev. Rolontis (eflapegrastim) Granulocyte-colony stimulating factor analogue Neutropenia Submitted updated BLA to FDA based on data from Advance and Recover trials in early stage breast cancer patients with neutropenia due to myelosuppressive chemotherapy
Company Product Description Indication Regulatory status

Immune

Abbvie Inc., of North Chicago Rinvoq (upadacitinib) JAK inhibitor Moderate to severe active rheumatoid arthritis The EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on the marketing application
Asit Biotech SA, of Brussels, Belgium gp-ASIT+TM Pollen-derived peptides based on allergen fragments Grass pollen allergy Received GMP certification for its manufacturing site in Liège, Belgium, from the Federal Agency for Medicines and Health Products, enabling in-house manufacturing of the API for its lead candidate
Biogen Inc., of Cambridge, Mass., and Alkermes plc, of Dublin Vumerity (diroximel fumarate) Oral fumarate that converts to monomethyl fumarate Relapsing forms of multiple sclerosis Approved by FDA; indications include clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease
DBV Technologies SA, of Montrouge, France Viaskin Peanut Immunotherapy delivered via once-daily epicutaneous patch Peanut allergy FDA accepted for review BLA seeking approval to treat peanut-allergic children, ages 4 to 11; PDUFA date is Aug. 5, 2020; FDA plans to hold an advisory committee meeting
Gilead Sciences Inc., of Foster City, Calif. Filgotinib JAK1 inhibitor Rheumatoid arthritis NDA submitted to the Japanese Ministry of Health, Labor and Welfare
Glenmark Pharmaceuticals Inc., of Mumbai, India Dimethyl fumarate delayed-release capsules Activates Nrf2 pathway Multiple sclerosis Received tentative approval from FDA for generic version of Tecfidera (Biogen Inc.)
Mallinckrodt plc, Staines-Upon-Thames, U.K. Uvadex (methoxsalen) Naturally occurring photoactive substance Graft-vs.-host disease Australia's TGA approved product for extracorporeal administration with the Therakos Cellex Photopheresis System for treatment of steroid-refractory and steroid-intolerant chronic GVHD in adults following allogeneic hematopoietic stem cell transplantation
Omeros Corp., of Seattle Narsoplimab (OMS-721) Monoclonal antibody targeting MASP-2 Hematopoietic stem cell transplant-associated thrombotic microangiopathy Submitted first sections of rolling BLA to FDA
Savara Inc., of Austin, Texas Molgradex Inhaled formulation of recombinant human GM-CSF Autoimmune pulmonary alveolar proteinosis In written response from FDA following type C meeting, agency indicated data in briefing package do not provide sufficient evidence of efficacy and safety and recommended company not submit a BLA; Savara is working to determine next steps; shares of the company (NASDAQ: SVRA) sank 56% on the news
Xenikos BV, of Nijmegen, the Netherlands T-Guard Toxin-conjugated monoclonal antibodies that target CD3 and CD7 on T cells and NK cells Steroid-refractory acute graft-versus-host disease following allogeneic stem cell transplantation FDA granted fast track designation
Company Product Description Indication Regulatory status

Infection

Ansun Biopharma Inc., of San Diego DAS-181 Recombinant sialidase protein Lower respiratory tract parainfluenza virus infection China’s NMPA cleared start of phase III Stop PIV trial in hospitalized, immunocompromised patients
Ansun Biopharma Inc., of San Diego DAS-181 Recombinant sialidase protein Influenza China’s NMPA cleared IND for phase IIb Stop Flu trial in severe hospitalized influenza patients
Aradigm Corp., of Newark, Calif. Linhaliq Inhaled formulation of the antibiotic ciprofloxacin Non-cystic fibrosis bronchiectasis caused by chronic lung infections with Pseudomonas aeruginosa Company withdrew its MAA after learning that the EMA's Committee for Medicinal Products for Human Use was likely to adopt a negative opinion
Basilea Pharmaceuticals Inc., of Basel, Switzerland, and Grupo Biotoscana SL, of Montevideo, Uruguay Cresemba (isavuconazole) Antifungal Invasive aspergillosis and mucormycosis Received approval in Brazil
Bluewillow Biologics Inc., of Ann Arbor, Mich. BW-1010 Intranasal anthrax vaccine Anthrax FDA cleared the IND for a phase I study, set to start enrollment this year; funded by National Institute of Allergy and Infectious Diseases
Emergent Biosolutions Inc., of Gaithersburg, Md. CHIKV VLP Virus-like particle vaccine Chikungunya virus Granted Priority Medicines, or PRIME, designation by EMA’s Committee for Medicinal Products for Human Use
Genentech, of South San Francisco, a member of the Roche Group Xofluza (baloxavir marboxil) Antiviral designed to inhibit cap-dependent endonuclease protein Acute, uncomplicated influenza FDA approved a supplemental NDA for use in people 12 and older who have been symptomatic for no more than 48 hours and who are at high risk of developing flu-related complications
Geovax Labs Inc., of Atlanta AGT103-T Lentiviral-based gene therapy HIV Filed IND with FDA for a phase I trial to test safety, measure biomarkers and explore surrogate markers of efficacy; recruitment anticipated to start in January 2020
Gilead Sciences Inc., of Foster City, Calif. Descovy (emtricitabine/tenofovir alafenamide) Fixed-dose combo of nucleoside reverse transcriptase inhibitors Pre-exposure prophylaxis for HIV-1 infection Approved by FDA for PrEP indication to reduce risk of sexually acquired HIV-1 infections in adults and adolescents weighing at least 35 kg who are HIV-negative and at risk for sexually acquired HIV, excluding individuals at-risk from receptive vaginal sex
Matinas Biopharma Holdings Inc., of Bedminster, N.J. MAT-2203 Oral formulation of the antifungal amphotericin B Cryptococcosis FDA granted orphan drug designation
Medicago Inc., of Quebec City Plant-based quadrivalent virus-like particle influenza recombinant vaccine Vaccine Influenza Health Canada accepted new drug submission for scientific review
Melinta Therapeutics Inc., of Morristown, N.J. Baxdela (delafloxacin) Quinolone antibiotic Community-acquired bacterial pneumonia Supplemental NDA approved by FDA; company is delaying commercial launch to manage its liquidity position
Merck & Co. Inc., of Kenilworth, N.J. Dificid (fidaxomicin) Macrolide antibacterial medicine Clostridioides difficile infections FDA accepted for review both an NDA for oral suspension and supplemental NDS for tablets and oral suspension to treat infections in children 6 months or older; both applications have priority review, with PDUFA dates of Jan. 24, 2020
Merck & Co. Inc., of Kenilworth, N.J. Ervebo (V920) Ebola Zaire vaccine Ebola infection prophylaxis The EMA's Committee for Medicinal Products for Human Use adopted a positive opinion recommending a conditional marketing authorization
Motif Bio plc, of London Iclaprim Selective bacterial dihydrofolate reductase inhibitor Hospital-acquired bacterial pneumonia, including ventilator-associated bacterial pneumonia Received minutes of type B meeting with FDA, confirming single well-designed and well-controlled phase III trial demonstrating efficacy and safety, along with data on potential mechanism of hepatic injury, would enable submission of NDA; company said partner or other entity with lower cost of capital would be needed
Paratek Pharmaceuticals Inc., of Boston Nuzyra (omadacycline) Modernized tetracycline antibiotic Acute bacterial skin and skin structure infections and community-acquired bacterial pneumonia Company withdrew its MAA from the EMA because the agency recommended approval for ABSSSI but not CABP; agency requested a second phase III study for CABP; withdrawing MAA for ABSSSI avoids starting clock for 10-year marketing exclusivity
Porton Biopharma Ltd., of Salisbury, U.K. NE-rPA anthrax vaccine Protein conjugate Bacillus anthracis infection Signed modification to contract with NIH's National Institute of Allergy and Infectious Diseases to advance into clinic its intranasal vaccine that combines Nanovax system from partner Blue Willow Biologics, with PBL’s recombinant protective antigen for anthrax
Vast Therapeutics Inc., of Durham, N.C. BIOC-11 Delivers nitric oxide via macromolecules Chronic pulmonary infections due to Pseudomonas aeruginosa in patients with cystic fibrosis FDA designated the drug a qualified infectious disease product
Company Product Description Indication Regulatory status

Inflammatory

Flexion Therapeutics Inc., of Burlington, Mass. Zilretta (triamcinolone acetonide) Extended-release glucocorticoid Osteoarthritis knee pain FDA needs additional time to review the supplemental NDA requesting revision of the label to include data from a phase IIIb study testing repeat administration of the drug; agency indicated that the review would be complete in the coming weeks and hasn’t requested any additional clinical data; PDUFA action date was Oct. 14, 2019
Flexion Therapeutics Inc., of Burlington, Mass. FX-201 Helper-dependent non-integrating adenovirus containing human IL-1Ra gene Knee osteoarthritis FDA cleared the IND for a phase I study; company expects to treat first patient by year-end
Novus Therapeutics Inc., of Irvine, Calif. OP-0201 Surfactant-based nasal aerosol drug-device combination Chronic otitis media with effusion FDA agreed with proposed patient population, endpoints and statistical analysis plan for phase IIa study
Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. Dupixent (dupilumab) Monoclonal antibody inhibiting IL-4 and IL-13 proteins Chronic rhinosinusitis with nasal polyposis European Commission approved new indication as an add-on therapy with intranasal corticosteroids for the treatment of adults with severe CRSwNP for whom therapy with systemic corticosteroids and/or surgery do not provide adequate disease control
Company Product Description Indication Regulatory status

Musculoskeletal

AB Science SA, of Paris Masitinib Oral tyrosine kinase inhibitor that targets mast cells and macrophages Indolent systemic mastocytosis Cleared by the French Medicine Agency, ANSM, to start a phase III confirmatory study in patients unresponsive to symptomatic treatment
Allergan plc, of Dublin Botox (onabotulinumtoxinA) Botulinum toxin Lower limb spasticity FDA approved supplemental BLA for use in pediatric patients, ages 2 to 17, excluding treatment for spasticity caused by cerebral palsy
Amgen Canada, of Mississauga, Ontario Evenity (romosozumab) Monoclonal antibody targeting sclerostin Postmenopausal women at high risk for fracture Health Canada approved the drug for sale
Amgen Inc., of Thousand Oaks, Calif., and UCB SA, of Brussels, Belgium Evenity (romosozumab) Monoclonal antibody targeting sclerostin Severe osteoporosis in postmenopausal women at high risk of fracture Following a re-examination procedure, the EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on the marketing application
Janssen Pharmaceutical Cos., of Beerse, Belgium, part of Johnson & Johnson Tremfya (guselkumab) Human monoclonal antibody against p19 subunit of IL-23 Active psoriatic arthritis Submitted type II variation application to the EMA seeking first-in-class approval
Novartis AG, of Basel, Switzerland Cosentyx (secukinumab) Inhibits IL-17A Active ankylosing spondylitis European Commission approved late update for up-titration to 300 mg, based on data from 3-year Measure 3 study
Pfenex Inc., of San Diego PF-708 Parathyroid hormone receptor agonist Osteoporosis FDA approved NDA submitted under 505(b)(2) regulatory pathway, with Forteo (teriparatide, Eli Lilly & Co.) as reference drug, earning Pfenex $2.5M milestone payment from partner Alvogen Inc., of Pine Brook, N.J.
Pfenex Inc., of San Diego PF-708 Teriparatide injection Osteoporosis Partner Alvogen Inc. said marketing authorization application was submitted to the Kingdom of Saudi Arabia's Saudi Food and Drug Authority
Pfenex Inc., of San Diego PF-708 Portion of human parathyroid hormone Osteoporosis Company submitted data from a human factors study to the FDA to seek designation of PF-708 as a therapeutically equivalent "A" rated drug to Forteo
Reveragen Biopharma Inc., of Rockville, Md. Vamorolone Dissociative steroid Duchenne muscular dystrophy Designated a promising innovative medicine by U.K.’s Medicines and Healthcare Products Regulatory Agency
Company Product Description Indication Regulatory status

Neurology/psychiatric

Durect Corp., of Cupertino, Calif. Posimir Bupivacaine extended-release solution Postoperative pain FDA notified firm the class 2 NDA resubmission will be discussed at Anesthetic and Analgesic Drug Products Advisory Committee meeting set for Jan. 16, 2020; FDA previously assigned a PDUFA date of Dec. 27, 2019, and a new date has not yet been set
Eicosis LLC, of David, Calif. EC-5026 Small-molecule inhibitor of epoxide hydrolase Pain FDA cleared the IND for phase I trials
Eli Lilly and Co., of Indianapolis Reyvow (lasmiditan) Serotonin (5-HT)1F receptor agonist Migraine FDA approved the drug
Genentech, of South San Francisco, a member of the Roche Group Satralizumab Interleukin-6 receptor antagonist Neuromyelitis optica spectrum disorder FDA accepted the BLA seeking approval for use in adults and adolescents; EMA also validated MAA, granting it accelerated assessment; decision from FDA and recommendation from EMA’s Committee for Medicinal Products for Human Use expected in 2020
Heron Therapeutics Inc., of San Diego HTX-011 Dual-acting, fixed-dose combination of local anesthetic bupivacaine and low dose nonsteroidal anti-inflammatory drug meloxicam Postoperative pain Resubmitted NDA to FDA, based on outcome and final minutes of type A meeting to discuss April 2019 complete response letter; anticipates 6-month review
Intelgenx Corp., of Saint Laurent, Quebec Rizaport Versafilm Oral thin film formulation of 5-HT1 receptor agonist rizatriptan benzoate Acute migraine Resubmitted 505(b)2) NDA was accepted by FDA for review; PDUFA date of March 26, 2020
Intellipharmaceutics International Inc., of Toronto Oxycodone ER Opioid designed to be abuse-deterrent Pain Advised by FDA that product-specific advisory committee meetings, which were previously postponed, are no longer on hold
Janssen Pharmaceutical Co., of Beerse, Belgium, a unit of Johnson & Johnson, of New Brunswick, N.J. Spravato (esketamine) Antagonist of the N-methyl-D-aspartate glutamate receptor Treatment-resistant major depressive disorder The EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on the marketing application for the drug in combination with a selective serotonin reuptake inhibitor or serotonin and norepinephrine reuptake inhibitor
Janssen Pharmaceutical Cos., of Titusville, N.J., part of Johnson & Johnson Spravato (esketamine) Nonselective, noncompetitive antagonist of NMDA receptor Major depressive disorder Submitted supplemental NDA seeking new indication for CIII nasal spray for rapid reduction of depressive symptoms in adults with MDD who have suicidal ideation and intent
Lobsor Pharmaceuticals AB, of Uppsala, Sweden Lecigon Gel formulation of levodopa/carbidopa and entacapone in a 50-ml prefilled container for continuous infusion into small intestine Parkinson’s disease Product approved, and Swedish Medical Products Agency confirmed end of procedure, according to European Mutual Recognition Procedure, including Denmark, Finland and Norway
Minoryx Therapeutics SL, of Barcelona Leriglitazone (MIN-102) PPAR gamma agonist Friedreich’s ataxia FDA granted orphan drug designation
Neuren Pharmaceuticals Ltd., of Melbourne, Australia NNZ-2591 Synthetic analogue of molecule derived from IGF-1 Angelman syndrome FDA granted orphan designation
Neuren Pharmaceuticals Ltd., of Melbourne, Australia NNZ-2591 Analogue of cyclic glycine proline Angelman syndrome; Phelan-McDermid syndrome; Pitt Hopkins syndrome FDA granted orphan designation for all 3 indications
Novartis AG, of Basel, Switzerland AVXS-101 Gene therapy Spinal muscular atrophy type 2 FDA placed partial hold on trials for intrathecal administration based on findings from a small preclinical study in animals showing dorsal root ganglia mononuclear cell inflammation, sometimes accompanied by neuronal cell body degeneration or loss; the hold does not impact marketed Zolgensma or AVSX-101 intravenous trials
Noven Pharmaceuticals Inc., of Miami, a wholly owned subsidiary of Hisamitsu Pharmaceutical Co. Inc. Secuado (asenapine) transdermal system Psychotropic agent delivered via transdermal patch Schizophrenia Approved by FDA for use in adults
Recro Pharma Inc., of Malvern, Pa. Intravenous meloxicam Long-acting preferential COX-2 inhibitor Pain FDA granted appeal of the complete response letter, stating NDA provides “sufficient evidence of effectiveness and safety to support approval”; company working on response to FDA, including proposed product labeling
Tetra Bio-Pharma Inc., of Ottawa Caumz Kit Comprises cannabinoid-derived medicine using synthetic THC and CBD plus vaporizer Pain and inflammation FDA determined product can be evaluated as a drug/device combination; assigned to Center for Drug Evaluation and Research for premarket review and further assignment
Company Product Description Indication Regulatory status

Ocular

Alimera Sciences Inc., of Atlanta Iluvien (fluocinolone acetonide intravitreal implant) Corticosteroid delivered via sustained-release intravitreal implant Recurrent non-infectious uveitis affecting posterior segment Approved by regulators in Portugal for use in preventing recurrence
Novartis AG, of Basel, Switzerland Beovu (brolucizumab) Monoclonal antibody targeting VEGF Wet age-related macular degeneration FDA approved the drug
Proqr Therapeutics NV, of Leiden, the Netherlands Sepofarsen RNA-based oligonucleotide Leber’s congenital amaurosis 10 Received rare pediatric disease designation from FDA
Company Product Description Indication Regulatory status

Other/miscellaneous

Algenex SL, of Madrid Undisclosed Vaccine using Baculovirus-based technology for production of recombinant biologics Undisclosed Partner submitted a dossier to the EMA for a recombinant VLP vaccine produced using Algenex’ Crisbio technology
Frequency Therapeutics Inc., of Woburn, Mass. FX-322 Combination of small-molecule drugs designed to activate inner ear progenitor cells to induce hair cell regeneration Sensorineural hearing loss FDA granted fast track designation
Mirum Pharmaceuticals Inc., of Foster City, Calif. Maralixibat Inhibits apical sodium dependent bile acid transporter Pruritus associated with Alagille syndrome FDA granted breakthrough therapy designation for use in patients, 1 and older
Solasia Pharma KK, of Tokyo Episil (SP-03) Oral liquid Oral mucositis Approved by the National Institute of Medical Device Safety Information in South Korea for management of pain and relief of pain in oral cavity caused by chemotherapy and/or radiotherapy
Vivus Inc., of Campbell, Calif. Qsymia (phentermine and topiramate) Enhances satiety Obesity European regulatory agencies in Sweden, Denmark, Finland, Iceland, Norway and Poland accepted the MAA on a decentralized basis; Sweden is acting as the lead concerned member state; marketing authorizations, if approved, are expected in the second half of 2020
Company Product Description Indication Regulatory status

Respiratory

Aradigm Corp., of Newark, Calif. Linhaliq (ciprofloxacin sustained-release inhalant nebulizer) DNA gyrase inhibitor; topoisomerase IV inhibitor Bronchiectasis Received feedback from EMA's Committee for Medicinal Products for Human Use that negative opinion will likely be issued next month on centralized MAA to treat individuals with non-cystic fibrosis bronchiectasis and chronic lung infections with Pseudomonas aeruginosa
Astrazeneca plc, of Cambridge, U.K. PT-010 (budesonide/glycopyrronium/formoterol fumarate) Fixed-dose combination of corticosteroid/long-acting muscarinic agonist/long-acting beta2-agonist Chronic obstructive pulmonary disease FDA issued complete response letter to NDA
Astrazeneca plc, of Cambridge, U.K. Fasenra Pen (benralizumab) Monoclonal antibody targeting IL-5 receptor alpha Severe eosinophilic asthma FDA approved the self-administration of the drug in a prefilled, single-use auto injector
Boehringer Ingelheim GmbH, of Ingelheim, Germany Ofev (nintedanib) Antifibrotic Chronic fibrosing interstitial lung diseases with progressive phenotype FDA granted breakthrough therapy designation; application under review
Glaxosmithkline plc, of London Nucala (mepolizumab) Anti-IL-5 biologic Severe eosinophilic asthma or eosinophilic granulomatosis with polyangiitis Approved in Canada in 2 new formats: prefilled autoinjector and prefilled safety syringe
Glaxosmithkline plc, of London, and Innoviva Inc., of Brisbane, Calif. Trelegy Ellipta Once-daily, single-inhaler comprising fluticasone furoate, umeclidinium and vilanterol Asthma Submitted supplemental NDA to FDA seeking additional indication
United Therapeutics Corp., of Silver Spring, Md. Orenitram (treprostinil) extended-release tablets Prostacyclin mimetic Pulmonary arterial hypertension FDA approved supplemental NDA reflecting data from Freedom-EV study, to include that Orenitram delays disease progression when used in conjunction with approved oral background PAH therapy
Vertex Pharmaceuticals Inc., of Boston Kalydeco (ivacaftor) Affects the gating of cystic fibrosis transmembrane conductance regulator Cystic fibrosis The EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on expanding the marketing application to include infants ages 6 months to less than 12 months with specified mutations in the CFTR gene
Vertex Pharmaceuticals Inc., of Boston Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) Dual CFTR modulator/stimulator Cystic fibrosis FDA approved drug to treat people 12 and older with at least 1 F508del mutation in the CFTR gene
Vertex Pharmaceuticals Inc., of Boston VX-445 (elexacaftor), tezacaftor and ivacaftor Triple-combination regimen comprising 3 CFTR modulators Cystic fibrosis EMA validated the MAA seeking approval for use in people with 1 F508del mutation and 1 minimal function mutation and in people with 2 F508del mutations
Company Product Description Indication Regulatory status

Toxicity/intoxication

Adial Pharmaceuticals Inc., of Charlottesville, Va. AD-04 5-HT 3 receptor antagonist Alcohol use disorder CTA filed with Swedish Medical Products Agency for phase III trial in genetically targeted participants

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