Cancer | Cardiovascular | Dermatologic | Endocrine/metabolic | Gastrointestinal | Genitourinary/sexual function | Hematologic | Immune | Infection | Inflammatory | Musculoskeletal | Neurology/psychiatric | Ocular | Other/miscellaneous | Respiratory | Toxicity/intoxication |

Regulatory actions reported in March 2020

Company Product Description Indication Regulatory status

Cancer

Abbvie Inc., of North Chicago Venclyxto (venetoclax) Bcl-2 inhibitor Chronic lymphocytic leukemia European Commission approved use in combination with obinutuzumab for previously untreated patients; approval valid in all 27 member states of the EU, as well as Iceland, Liechtenstein, Norway and the U.K.
Agenus Inc., of Lexington, Mass. Balstilimab and zalifrelimab Anti-PD-1 antibody and anti-CTLA4 antibody Relapsed/refractory metastatic cervical cancer FDA granted fast track designation for combination
Anheart Therapeutics Co. Ltd., of Hangzhou, China Taletrectinib ROS1 and NTRK inhibitor Non-small-cell lung cancer Chinese Center for Drug Evaluation cleared IND and issued clinical trial authorizations for 2 phase II trials in China in patients with ROS1 mutations and locally advanced or metastatic solid tumors with NTRK mutations
Ascentage Pharma Group International, of Suzhou, China APG-2575 Bcl-2 selective inhibitor Relapsed/refractory chronic lymphocytic leukemia or small lymphocytic lymphoma FDA cleared IND for phase Ib/II trial testing drug as single agent or in combination
Ascentage Pharma Group International, of Suzhou, China APG-2575 Bcl-2 selective inhibitor Waldenström macroglobulinemia FDA cleared IND for a phase Ib/II trial testing the drug as a single agent or in combination
Ascentage Pharma Group International, of Suzhou, China APG-2575 Bcl-2 selective inhibitor Relapsed/refractory acute myeloid leukemia China's National Medical Products Administration approved start of phase Ib trial testing the drug as a single agent or in combination
Astrazeneca plc, of Cambridge, U.K. Imfinzi (durvalumab) PD-L1-inhibiting antibody Small-cell lung cancer FDA approved use as first-line treatment for adults with extensive-stage disease in combination with standard-of-care chemotherapies etoposide and either carboplatin or cisplatin
Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. Lynparza PARP inhibitor Maintenance treatment of germline BRCA-mutated, curatively unresectable, pancreatic cancer Japanese Ministry of Health, Labor and Welfare granted orphan drug designation
Aveo Pharmaceuticals Inc. (Aveo Oncology), of Cambridge, Mass. Fotivda (tivozanib) VEGF receptor-tyrosine kinase inhibitor Renal cell carcinoma NDA submitted to FDA to treat individuals with relapsed/refractory disease
Ayala Pharmaceuticals Inc., of Rehovot, Israel AL-101 Selective inhibitor of gamma secretase-mediated Notch signaling Recurrent or metastatic adenoid cystic carcinoma FDA granted fast track designation
Bristol Myers Squibb Co., of New York, and Bluebird Bio Inc., of Cambridge, Mass. Idecabtagene vicleucel (bb-2121) CAR T targeting B-cell maturation antigen Multiple myeloma Submitted a BLA to the FDA
Bristol-Myers Squibb Co., of New York Opdivo (nivolumab) PD-1 inhibitor Hepatocellular carcinoma FDA granted accelerated approval, in combination with Yervoy (ipilimumab), to treat individuals previously treated with sorafenib (Nexavar, Bayer AG)
Cstone Pharmaceuticals Co. Ltd., of Suzhou, China Avapritinib Inhibitor of KIT and PDGFRA mutant kinases Gastrointestinal stromal tumor NDA submitted
Daiichi Sankyo Co. Ltd., of Tokyo Enhertu (trastuzumab deruxtecan) Antibody-drug conjugate targeting HER2 HER2-positive unresectable or recurrent breast cancer after prior chemotherapy Japanese Ministry of Health, Labor and Welfare approved the drug under the conditional early approval system
Daiichi Sankyo Co. Ltd., of Tokyo Axicabtagene ciloleucel CAR T-cell therapy Relapsed/refractory diffuse large B-cell lymphoma and related lymphomas Submitted NDA to Japan's Ministry of Health, Labour and Welfare
Eisai Co. Ltd., of Tokyo Denileukin diftitox (genetic recombinant) Fusion protein of the receptor-binding portion of interleukin-2 and diphtheria toxin that binds to the IL-2 receptor Relapsed or refractory cutaneous T-cell lymphoma and peripheral T-cell lymphoma Submitted a marketing authorization application in Japan
EMD Serono, part of Merck KGaA, of Darmstadt, Germany Tepmetko (tepotinib) MET inhibitor Unresectable, advanced or recurrent non-small-cell lung cancer with MET exon 14 skipping alterations Japanese Ministry of Health, Labor and Welfare approved the drug
Epicentrx Inc., of La Jolla, Calif. AIM-001 Oncolytic adenovirus enhanced with TGF-beta trap transgene Cancer FDA cleared the IND; clinical trials to begin this year
Essa Pharmaceuticals Inc., of Vancouver, British Columbia EPI-7386 Inhibits the N-terminal domain of the androgen receptor Metastatic castration-resistant prostate cancer Submitted an IND to the FDA for a phase I study
Exelixis Inc., of Alameda, Calif., and Takeda Pharmaceutical Co. Ltd., of Osaka, Japan Cabometyx (cabozantinib) Kinase inhibitor Curatively unresectable or metastatic renal cell carcinoma Japanese Ministry of Health, Labor and Welfare approved the drug
Hansoh Pharmaceutical Group Co. Ltd., of Shanghai Ameile (almonertinib) EGFR tyrosine kinase inhibitor Metastatic non-small-cell lung cancer National Medical Products Administration in China granted marketing authorization for use in patients with an EGFR T790M mutation after progression on other EGFR TKI
Immunomic Therapeutics Inc., of Rockville, Md. ITI-1001 Plasmid DNA vaccine therapy Glioblastoma multiforme Completed a pre-IND meeting with FDA
Immutep Ltd., of Sydney Eftilagimod alpha (IMP-321) HLA class II antigen stimulator Metastatic breast cancer FDA approved IND for planned phase I AIPAC-002 combination study with paclitaxel in 24 participants
Infinity Pharmaceuticals Inc., of Cambridge, Mass. IPI-549 PI3K-gamma inhibitor Advanced urothelial cancer FDA granted fast track designation in combination with Opdivo (nivolumab, Bristol Myers Squib Co.)
Janssen Pharmaceutical Cos., unit of Johnson & Johnson, of New Brunswick, N.J. JNJ-6372 EGFR-mesenchymal epithelial transition factor bispecific antibody Non-small-cell lung cancer FDA granted breakthrough therapy designation to treat metastatic disease in people with EGFR exon 20 insertion mutations whose disease progressed on or after platinum-based chemotherapy
Junshi Biosciences Ltd., of Shanghai Toripalimab Monoclonal antibody targeting PD-1 Mucosal melanoma FDA granted orphan designation for use in combination with axitinib (Pfizer Inc.)
Kura Oncology Inc., of San Diego Tipifarnib Inhibitor of farnesyl transferase T-cell lymphomas Received fast track designation
Morphosys AG, of Planegg, Germany Tafasitamab Anti-CD19 antibody Relapsed or refractory diffuse large B-cell lymphoma BLA accepted by FDA, with agency granting priority review; PDUFA date of Aug. 30, 2020
Mylan NV, of Hertfordshire, U.K. MYL-1402O (bevacizumab biosimilar) VEGF ligand inhibitor Solid tumors FDA accepted BLA for review under 351(k) pathway for first- and second-line metastatic colorectal cancer in combination with fluorouracil-based chemotherapy; first-line use in nonsquamous non-small-cell lung cancer; and in recurrent glioblastoma, metastatic renal cell carcinoma in combination with interferon alfa and persistent, recurrent or metastatic cervical cancer; BsUFA date is Dec. 27, 2020
Nucana plc, of Edinburgh, U.K. Acelarin Derived from nucleoside analogue gemcitabine Biliary tract cancer EMA's Committee for Orphan Medicinal Products issued positive opinion for orphan designation
Oncology Venture A/S, of Horsholm, Denmark Dovitinib Pan-tyrosine kinase inhibitor Renal cell carcinoma Attended a pre-NDA meeting with the FDA, in which the FDA indicated it would accept the NDA filing if submitted; the company's proposal is to seek approval based on noninferiority against the already-approved compound sorafenib (Bayer AG), based on prior phase III trial results
Orion Corp., of Espoo, Finland Nubeqa (darolutamide) Oral androgen receptor inhibitor Non-metastatic castration-resistant prostate cancer European Commission granted marketing authorization
Ryvu Therapeutics SA, of Krakow, Poland SEL-120 Oral, selective inhibitor of CDK8 kinase Acute myeloid leukemia FDA granted orphan designation
Samsung Bioepis Co. Ltd., of Incheon, Korea Ontruzant (trastuzumab-dttb) ERBB2 tyrosine kinase receptor inhibitor Breast/metastatic breast cancer; metastatic gastric cancer; gastroesophageal junction adenocarcinoma FDA approved 420-mg multidose vial of the biosimilar as adjuvant treatment
Sanofi SA, of Paris Sarclisa (isatuximab-irfc) CD38-targeting monoclonal antibody Relapsed multiple myeloma Approved by FDA for use in patients who have received at least 2 prior therapies, including lenalidomide and a proteasome inhibitor
Sanofi SA, of Paris Sarclisa (isatuximab-irfc) CD38-targeting monoclonal antibody Relapsed and refractory multiple myeloma EMA's Committee for Medicinal Products for Human Use adopted a positive opinion
Sumitomo Dainippon Pharma Co. Ltd., of Osaka, Japan Rethio (thiotepa) Alkylating agent Malignant lymphoma Japan's Ministry of Health, Labor, and Welfare approved the partial change in the marketing approval to include conditioning treatment prior to autologous hematopoietic stem cell transplantation for malignant lymphoma
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan Alunbrig (brigatinib) ALK-targeting tyrosine kinase inhibitor ALK-positive non-small-cell lung cancer EMA's Committee for Medicinal Products for Human Use recommended approval for use in patients not previously treated with an ALK inhibitor
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan, Adcetris (brentuximab vedotin) Antibody-drug conjugate targeting CD30 Previously untreated systemic anaplastic large-cell lymphoma EMA's Committee for Medicinal Products for Human Use adopted a positive opinion for the extension of the marketing authorization in combination with cyclophosphamide, doxorubicin and prednisone
TG Therapeutics Inc., of New York Umbralisib Dual PI3K-delta/CK1-epsilon inhibitor Follicular lymphoma FDA granted orphan designation
Tracon Pharmaceuticals Inc., of San Diego Envafolimab Single-domain antibody against PD-L1 Sarcoma Submitted request for a type B meeting with FDA to discuss the trial design for a potential pivotal study
Zai Lab Ltd., of Shanghai Zejula (niraparib) PARP inhibitor Epithelial ovarian, fallopian tube or primary peritoneal cancer China's NMPA accepted the supplemental NDA for use as maintenance treatment of adults with advanced disease who are in complete or partial response to first-line platinum-based chemotherapy
Company Product Description Indication Regulatory status

Cardiovascular

Cardiol Therapeutics Inc., of Oakville, Ontario Cannabidiol formulation Cannabidiol formulation Heart failure Received no objection letter from Health Canada for a phase I study in healthy volunteers
Company Product Description Indication Regulatory status

Dermatologic

Arch Therapeutics Inc., of Framingham, Mass. AC5 topical gel Hemostatic agent Pressure sores, leg ulcers, diabetic ulcers, surgical wounds FDA cleared the marketing of the gel using an additional supplier and additional manufacturing processes
Athenex Inc., of Buffalo, N.Y. Tirbanibulin (formerly KX2-391 or KX-01) SRC tyrosine kinase inhibitor; tubulin binding agent Actinic keratosis FDA accepted NDA filing, setting PDUFA date of Dec. 30, 2020, with no advisory committee meeting planned
Athenex Inc., of Buffalo, N.Y., and Almirall SA, of Barcelona, Spain Tirbanibulin ointment Src kinase inhibitor Actinic keratosis EMA accepted filing of the MAA
Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. Berotralstat (BCX-7353) Oral, once-daily inhibitor of kallikrein in plasma Prevention of hereditary angioedema attacks EMA validated the marketing authorization application
Eli Lilly and Co., of Indianapolis Baricitinib Oral JAK inhibitor Alopecia areata FDA granted breakthrough therapy designation
Eli Lilly and Co., of Indianapolis Taltz (ixekizumab) Humanized IgG4 monoclonal antibody Moderate to severe plaque psoriasis FDA approved supplemental BLA for treating pediatric patients, ages 6 to under 18, who are candidates for systemic therapy or phototherapy
Inmed Pharmaceuticals Inc., of Vancouver, British Columbia INM-755 (cannabinol) Keratin modulator Epidermolysis bullosa CTA filed in the Netherlands to initiate second phase I trial in healthy volunteers assessing 2 strengths of study drug in 8 adults over 14-day treatment period
Pharming Group NV, of Leiden, the Netherlands Ruconest (conestat alfa) Recombinant human C1 esterase inhibitor Hereditary angioedema FDA approved addition of new Netherlands production facility, expanding product manufacturing capacity
Pharming Group NV, of Leiden, the Netherlands Ruconest (conestat alfa) Recombinant human C1 esterase inhibitor Hereditary angioedema EMA's Committee for Medicinal Products for Human Use recommended approval for treating acute angioedema attacks in children, expanding the age range to children 2 and older
Company Product Description Indication Regulatory status

Endocrine/metabolic

Agios Pharmaceuticals Inc., of Cambridge, Mass. Mitapivat Allosteric activator of wild-type and a variety of mutated pyruvate kinase-R enzymes Pyruvate kinase deficiency EMA granted orphan drug designation
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Givlaari (givosiran) RNAi therapeutic targeting aminolevulinic acid synthase 1 Acute hepatic porphyria European Commission granted marketing authorization in adults and adolescents
APR Applied Pharma Research SA, of Balerna, Switzerland APR-OD031 Extended-release amino acid mix Phenylketonuria FDA granted orphan designation
Astrazeneca plc, of Cambridge, U.K. Lokelma (sodium zirconium cyclosilicate) Potassium-removing medicine Hyperkalemia on stable hemodialysis EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on a dosing and administration label update
Astrazeneca plc, of Cambridge, U.K. Lokelma (sodium zirconium cyclosilicate) Potassium-removing medicine Hyperkalemia Approved for marketing by Japan Ministry of Health
Avrobio Inc., of Cambridge, Mass. AVR-RD-04 CTNS gene stimulator Cystinosis FDA granted orphan drug designation
Cyclo Therapeutics Inc., of Alachua, Fla. Trappsol Cyclo Formulation of hydroxypropyl beta cyclodextrin Niemann-Pick disease type C1 Received feedback from type C meeting with FDA on global pivotal trial design; plans to meet with EMA in the second quarter of 2020
Eli Lilly and Co., of Indianapolis, and Boehringer Ingelheim GmbH, of Ingelheim, Germany Jardiance (empagliflozin) SGLT2 inhibitor Type 1 diabetes FDA issued a complete response letter
Freeline Therapeutics Ltd., of London FLT-190 Liver-directed AAV gene therapy Fabry disease European Commission granted orphan designation
Intrabio Inc., of Oxford, U.K. IB-1001 Modified amino-acid analogues/esters GM2 gangliosidosis FDA granted fast track designation
Intrabio Inc., of Oxford, U.K. IB-1001 Modified amino-acid analogues/esters Niemann-Pick disease type C FDA granted fast track designation
Novartis AG, of Basel, Switzerland Isturisa (osilodrostat) 11?beta?hyrdoxylase inhibitor Cushing's disease FDA approved drug to treat adults who either cannot undergo pituitary gland surgery or have undergone surgery but still have Cushing's disease
Oramed Pharmaceuticals Inc., of New York ORMD-0801 Oral insulin Type 2 diabetes At the end-of-phase-II meeting, the FDA gave feedback on issues for drug product manufacturing; company plans an additional meeting to go over phase III design
Recordati SpA, of Milan, Italy Osilodrostat Oral inhibitor of 11-beta-hydroxylase Cushing's syndrome Submitted NDA to Japan's Ministry of Health, Labor and Welfare
Rhythm Pharmaceuticals Inc., of Boston Setmelanotide Selective agonist of MC? receptor Alstrom syndrome FDA granted orphan designation
Stealth Biotherapeutics Corp., of Boston Elamipretide Mitochondrial-targeted compound Barth syndrome FDA granted rare pediatric disease designation
Zealand Pharma A/S, of Copenhagen Dasiglucagon Hypopal Rescue Pen Glucagon analogue Hypoglycemia in patients with diabetes Submitted an NDA to the FDA
Company Product Description Indication Regulatory status

Gastrointestinal

Intercept Pharmaceuticals Inc., of New York Obeticholic acid Binds to farnesoid X receptor Liver fibrosis due to nonalcoholic steatohepatitis FDA notified firm that, due to COVID-19 pandemic, it rescheduled advisory committee; originally set for April 22, 2020, the meeting is now tentatively scheduled for June 9, 2020; PDUFA date for the priority application remains June 26, 2020
Company Product Description Indication Regulatory status

Genitourinary/sexual function

Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis Jardiance (empagliflozin) SGLT2 inhibitor Chronic kidney disease FDA granted fast track designation for use in reducing risk of kidney disease progression and cardiovascular death in adults with CKD
Lipocine Inc., of Salt Lake City Tlando Oral testosterone Hypogonadism FDA acknowledged receipt of resubmitted NDA seeking approval of the TRT candidate for use in adult males for conditions associated with a deficiency of endogenous testosterone
Mallinckrodt plc, of Staines-upon-Thames, U.K. Terlipressin Vasopressin analogue selective for V1 receptors Hepatorenal syndrome type 1 Initiated rolling submission of NDA to FDA; expects to complete submission in the coming months
Mallinckrodt plc, of Staines-upon-Thames, U.K. Terlipressin Vasopressin analogue selective for V1 receptors Hepatorenal syndrome type 1 Completed a rolling submission of an NDA to the FDA
Myovant Sciences Ltd., of Basel, Switzerland Relugolix GNRH receptor antagonist Uterine fibroids MAA submitted to EMA for combination tablet (relugolix 40 mg + estradiol 1 mg + norethindrone acetate 0.5 mg) to treat moderate to severe symptoms
Orphan Technologies Ltd., of Rapperswil, Switzerland OT-58 Modified recombinant enzyme therapy Homocystinuria FDA granted rare pediatric disease designation
Rockwell Medical Inc., of Wixom, Mich. Triferic AVNU (ferric pyrophosphate citrate) Iron maintenance therapy Hemodialysis-dependent chronic kidney disease FDA approved NDA for I.V. formulation to replace iron and maintain hemoglobin in adults
Urovant Sciences, part of New York-based Sumitovant Biopharma Ltd. Vibegron ?3-adrenoreceptor agonist Overactive bladder FDA accepted for review NDA seeking approval to treat patients with symptoms of urge urinary incontinence, urgency and urinary frequency; agency assigned PDUFA date of Dec. 26, 2020, and said it does not currently plan to hold and advisory committee meeting to discuss the application
Zydus Cadila Ltd., of Ahmedabad, India Saroglitazar Targets PPAR alpha and gamma Noncirrhotic nonalcoholic steatohepatitis NDA approved by Drug Controller General of India
Company Product Description Indication Regulatory status

Hematologic

Agios Pharmaceuticals Inc., of Cambridge, Mass. AG-946 Pyruvate kinase-R activator Hemolytic anemias FDA cleared the IND; company plans to start a phase I trial in healthy volunteers in mid-2020
Intellia Therapeutics Inc., of Cambridge, Mass., and Novartis AG, of Basel, Switzerland OTQ-923 CRISPR/Cas9 expressing fetal hemoglobin Sickle cell disease FDA accepted the IND for a phase I/II study
Company Product Description Indication Regulatory status

Immune

Alpine Immune Sciences Inc., of Seattle AlPN-101 Dual T-cell co-stimulation inhibitor Acute graft-vs.-host disease FDA granted 2 orphan designations for prevention and treatment of acute GVHD
Aurinia Pharmaceuticals Inc., of Victoria, British Columbia Voclosporin Next-generation calcineurin inhibitor Lupus nephritis Initiated rolling NDA to FDA; expects to complete submission by end of the second quarter of 2020
Bristol Myers Squibb Co., of New York Zeposia (ozanimod) S1P receptor modulator Relapsing remitting multiple sclerosis EMA's Committee for Medicinal Products for Human Use recommended approval for use in adults with active disease as defined by clinical or imaging features
DBV Technologies SA, of Montrouge, France Viaskin Peanut Epicutaneous immunotherapy Peanut allergy FDA said during its ongoing review of the BLA it identified questions regarding efficacy, including the impact of patch-site adhesion, and therefore, the Allergenic Products Advisory Committee meeting to discuss the BLA will no longer take place on May 15, 2020
Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson Ponesimod Sphingosine-1-phosphate receptor 1 modulator Relapsing multiple sclerosis NDA submitted to the FDA
Janssen Pharmaceuticals Cos., of Allschwil, Switzerland, part of Johnson & Johnson Ponesimod Sphingosine-1-phosphate receptor 1 modulator Relapsing multiple sclerosis Submitted MAA to EMA seeking approval for use in adults
Kadmon Holdings Inc., of New York KD-025 ROCK2 inhibitor Chronic graft-vs.-host disease Held pre-NDA meeting with FDA and anticipates receiving final minutes within next 30 days; remains on track to complete NDA submission in fourth quarter of 2020
Mylan NV, of Hertfordshire, U.K., and Lupin Ltd., of Mumbai, India Nepexto Etanercept biosimilar Rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, axial spondyloarthritis, plaque psoriasis and pediatric plaque psoriasis The EMA's Committee for Medicinal Products for Human Use adopted a positive opinion recommending approval
Orchard Therapeutics plc, of London OTL-101 (simoladagene autotemcel) ADA gene stimulator Severe combined immunodeficiency COVID-19-related clinical and manufacturing impacts expected to delay initiation of rolling BLA to FDA, previously planned for first half of 2020
Shenzhen Chipscreen Biosciences Co. Ltd., of Shenzhen, China CS-112192 Dual JAK1/JAK3 tyrosine kinase inhibitor; serine threonine protein kinase TBK1 inhibitor Autoimmune diseases China NMPA's Center for Drug Evaluation accepted IND
Virpax Pharmaceuticals Inc., of West Chester, Pa. Epoladerm (diclofenac epolamine) Metered-dose topical aerosol of diclofenac epolamine Osteoarthritis of the knee Completed pre-IND meeting with the FDA, which agreed that the 505(b)(2) pathway is a reasonable approach for approval
Company Product Description Indication Regulatory status

Infection

Abbvie Inc., of North Chicago Maviret (glecaprevir/pibrentasvir) NS3/4A protease inhibitor and NS5A inhibitor Hepatitis C virus European Commission approved change to marketing authorization to shorten once-daily treatment duration from 12 to 8 weeks in treatment-naïve, compensated cirrhotic, chronic HCV patients with genotype 3 infection
Adaptive Phage Therapeutics Inc., of Gaithersburg, Md. Phagebank Personalized bacteriophage therapeutic Multidrug-resistant and complicated bacterial infections FDA cleared IND for phase I/II trial in patients with complicated and chronic recurrent urinary tract infections
Algernon Pharmaceuticals Inc., of Vancouver, British Columbia NP-120 (ifenprodil) NDMA receptor glutamate receptor antagonist specifically targeting NMDA-type subunit 2B Prevention of acute lung injury and acute respiratory distress syndrome associated with COVID-19 Requested pre-IND meeting with FDA, asking for direction regarding use of injectable and slow-release formulation as well as currently available drug supply for a U.S. clinical trial on an emergency basis; filing also includes clarification of expanded access pathway
Allovir Inc., of Cambridge, Mass. Viralym-M (ALVR-105) Multivirus-specific T-cell therapy Prevention of BK virus, cytomegalovirus, human herpes virus-6, Epstein-Barr virus, adenovirus and JC virus EMA granted orphan drug designation
Bellerophon Therapeutics Inc., of Warren, N.J. Inopulse Nitric oxide, inhaled COVID-19 FDA granted emergency expanded access
Can-Fite Biopharma Ltd., of Petach Tikva, Israel Piclidenoson  A3 adenosine receptor agonist COVID-19 Submitted drug to the institutional review board at Rabin Medical Center for a compassionate use program
Cansino Biologics Inc., of Tianjin, China Ad5-nCoV Recombinant coronavirus vaccine COVID-19 prophylaxis Vaccine approved to enter phase I study in China
Cytodyn Inc., of Vancouver, Wash. Leronlimab (PRO-140) CCR5 antagonist COVID-19 Filed modified IND and protocol for phase II trial for patients who experience respiratory complications as result of contracting COVID-19
Cytodyn Inc., of Vancouver, Wash. Leronlimab (PRO-140) CCR5 antagonist COVID-19 FDA granted emergency IND for 2 patients
Cytodyn Inc., of Vancouver, Wash. Leronlimab (PRO-140) CCR5 antagonist COVID-19 FDA cleared the start of a phase II study in patients with mild to moderate indications
Cytodyn Inc., of Vancouver, Wash. Leronlimab (PRO-140) CCR5 antagonist HIV infection Filed another round of modifications to its IND and protocol for a phase II trial
Dare Biosciences Inc., of San Diego DARE-BV1 Thermosetting bioadhesive hydrogel containing clindamycin phosphate 2% Bacterial vaginosis FDA granted fast track designation
F2G Ltd., of Manchester, U.K. Olorofim (formerly F-901318) Dihydroorotate dehydrogenase inhibitor Invasive aspergillosis and lomentospora/scedosporium infections FDA granted orphan drug designation
Genentech, of South San Francisco, a member of the Roche Group Actemra (tocilizumab) Interleukin-6 receptor antagonist COVID-19 pneumonia FDA approved a phase III trial testing the drug in combination with standard of care
Genentech, of South San Francisco, a member of the Roche Group Xofluza Baloxavir marboxil Acute uncomplicated influenza; post-exposure prophylaxis of influenza FDA accepted an NDA for a new formulation as 1-dose granules for oral suspension; the FDA also accepted 2 sNDAs: for acute uncomplicated influenza in children and for post-exposure prophylaxis of influenza in people 1 and older
Gilead Sciences Inc., of Foster City, Calif. Epclusa (sofosbuvir and velpatasvir) HCV NS5B polymerase inhibitor and HCV NS5A inhibitor Hepatitis C virus FDA approved supplemental application to expand use for children, ages 6 and older and weighing at least 37 pounds, with any of the 6 HCV genotypes without cirrhosis or with mild cirrhosis; Epclusa plus ribavirin is indicated for treating pediatric patients, 6 and older and weighing at least 37 pounds, with severe cirrhosis
Gilead Sciences Inc., of Foster City, Calif. Remdesivir RNA polymerase modulator COVID-19 Gilead submitted request to FDA to rescind orphan designation and said it is waiving gall benefits that accompany such designation
Humanigen Inc., of Burlingame, Calif. Lenzilumab Humaneered anti-human GM-CSF monoclonal antibody COVID-19 Submitted initial protocol synopsis to FDA to support plans for a phase III study for the prevention of acute respiratory distress syndrome and/or death in hospitalized patients with pneumonia associated with SARS-CoV-2 infection
I-Mab Biopharma Co. Ltd., of Rockville, Md., and Shanghai TJM-2 Neutralizing antibody against human GM-CSF Cytokine storm Submitted IND to South Korea's Ministry of Food and Drug Safety to evaluate drug in severe and critically ill patients with COVID-19
Paratek Pharmaceuticals Inc., of Boston Nuzyra (omadacycline) Broad-spectrum aminomethylcycline antibiotic Anthrax infection Pre-emergency use authorization application submitted by BARDA to FDA to allow for potential treatment and prophylaxis use during declared emergency
Relief Therapeutics Holding AG, of Zurich, and Neurorx Inc., of Wilmington, Del. RLF-100 (aviptadil) Synthesized peptide Acute and moderate respiratory distress in COVID-19 Filed an IND with the FDA for a phase II trial
Spero Therapeutics Inc., of Cambridge, Mass. SPR-720 DNA gyrase inhibitor; topoisomerase IV inhibitor Nontuberculous mycobacterial infection FDA granted orphan drug designation
Synairgen plc, of Southampton, U.K. SNG-001 Inhaled formulation of interferon-beta-1a COVID-19 Received expedited approvals from U.K.'s Medicines and Healthcare Products Regulatory Agency and Health Research Authority to conduct a clinical trial
Takis Biotech Srl, of Rome, and Applied DNA Sciences Inc., of Stony Brook, N.Y. COVID-19 vaccine DNA vaccine COVID-19 prophylaxis Italy's Ministry of Health approved the start of preclinical testing
Valneva SE, of Saint-Herblain, France VLA-1533 Monovalent, single-dose, live-attenuated vaccine Chikungunya Completed end-of-phase II meeting with FDA and agreed on development plan based on immunology surrogate (accelerated approval pathway); company is preparing for phase III initiation as soon as the COVID-19 situation permits, currently assuming initiation in the fourth quarter of 2020
Valneva SE, of Saint-Herblain, France Ixiaro (IC-51) Cell-derived vaccine Japanese encephalitis virus infection FDA approved extension of product shelf-life from 24 to 36 months; separately, U.S. Department of Defense issued request for proposal covering 3-year period that could lead to supply contract during first half of 2020
Viiv Healthcare Ltd., of London Cabenuva Cabotegravir and rilpivirine extended-release injectable suspensions HIV-1 infection Approved by Health Canada
Viiv Healthcare Ltd., of London Vocabria  Cabotegravir HIV-1 infection Health Canada approved it as a short-term addition to Cabenuva
Company Product Description Indication Regulatory status

Inflammatory

Bone Therapeutics SA, of Gosselies, Belgium JTA-004 Mix of plasma proteins, hyaluronic acid, a natural component of knee synovial fluid and a fast-acting analgesic Osteoarthritic knee pain Denmark regulatory authorities approved the clinical trial application
Company Product Description Indication Regulatory status

Musculoskeletal

AB Science SA, of Paris Masitinib Tyrosine kinase inhibitor that targets mast cells and macrophages Amyotrophic lateral sclerosis FDA cleared the IND for a phase III study (AB19001); company aims to initiate study as soon as conditions in U.S. clinical sites stabilize post-COVID-19 pandemic
Acer Therapeutics Inc., of Newton, Mass. Edsivo (celiprolol) Beta blocker Vascular Ehlers-Danlos syndrome with a confirmed COL3A1 mutation FDA denied company's appeal of the CRL issued in June 2019
Bone Therapeutics SA, of Gosselies, Belgium Allob Allogeneic cell therapy Difficult tibial fractures Belgian regulatory authorities approved the clinical trial application
Cytokinetics Inc., of South San Francisco Reldesemtiv Fast skeletal muscle troponin activator Amyotrophic lateral sclerosis EMA granted orphan designation
Novartis AG, of Basel, Switzerland Cosentyx Secukinumab; IL-17A inhibitor Non-radiographic axial spondyloarthritis Gained positive opinion from EMA's Committee for Medicinal Products for Human Use for a new indication in the axial spondyloarthritis spectrum
Company Product Description Indication Regulatory status

Neurology/psychiatric

Acacia Pharma Group plc, of Cambridge, U.K. Byfavo (remimazolam) Ultra-short-acting, reversible intravenous benzodiazepine Sedative/anesthetic use during invasive medical procedures FDA extended the review period by up to 90 days to complete review of additional data submitted in January and February 2020; new PDUFA date of July 5, 2020
Acadia Pharmaceuticals Inc., of San Diego, and Neuren Pharmaceuticals Ltd., of Melbourne, Australia Trofinetide Synthetic analogue of the amino?terminal tripeptide of IGF-1 Rett syndrome FDA granted rare pediatric disease designation
Agex Therapeutics Inc., of Alameda, Calif., and Imstem Biotechnology Inc., of Farmington, Conn. IMS-001 hES-MSC-based allogeneic cell therapy Multiple sclerosis FDA lifted the clinical hold and cleared the IND
Arvelle Therapeutics GmbH, of Zug, Switzerland Cenobamate Voltage-gated sodium channel blocker Focal onset seizures in epilepsy EMA accepted marketing authorization application
Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. Verdiperstat Calcitonin gene-related peptide receptor antagonist Multiple system atrophy FDA granted fast track designation
Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. Vazegepant Calcitonin gene-related peptide receptor antagonist Acute migraine Following a successful end-of-phase-II interaction with the FDA, company plans to start a phase III study with the 10-mg dose
Bristol Myers Squibb Co., of New York Zeposia (ozanimod)  Sphingosine 1-phosphate receptor modulator  Relapsing-remitting forms of multiple sclerosis Approved for marketing by FDA
Cadent Therapeutics Inc., of Cambridge, Mass. CAD-9303 Positive allosteric modulator of NMDA Schizophrenia FDA cleared the IND for a phase I trial in schizophrenia patients and healthy volunteers
GW Pharmaceuticals plc, of Carlsbad, Calif. Epidiolex Cannabidiol oral solution Tuberous sclerosis complex FDA accepted the supplemental NDA with a priority review; PDUFA date of July 21, 2020
GW Pharmaceuticals plc, of London Epidyolex Cannabidiol oral solution Seizures associated with tuberous sclerosis complex Submitted type II variation application to EMA; if approved, it will be the third licensed indication for the drug in Europe
Innocoll Holdings Ltd., of Athlone, Ireland Xaracoll (bupivacaine hydrochloride collagen-matrix implant) Anesthetic Management of postsurgical pain after open inguinal hernia surgery FDA accepted the NDA resubmission and set PDUFA date of Aug. 26, 2020
Intelgenx Technologies Corp., of Saint Laurent, Quebec Rizaport Versafilm Oral thin-film formulation of 5-HT1 receptor agonist rizatriptan Acute migraine FDA issued complete response letter for resubmitted 505(b)(2) NDA, stating the agency cannot approve the application in its present form and requesting additional information, though no new bioequivalence study was requested
Jazz Pharmaceuticals plc, of Dublin JZP-258 Oxybate product Excessive daytime sleepiness FDA accepted NDA for filing with priority review; seeks approval for use in patients 7 and older with narcolepsy; PDUFA date is July 21, 2020
Kempharm Inc., of Celebration, Fla. KP-415 D-methylphenidate prodrug Attention deficit hyperactivity disorder Submitted an NDA to the FDA
Newron Pharmaceuticals SpA, of Milan, Italy Sarizotan 5-HT 1a receptor agonist; selective dopamine receptor modulator Rett syndrome Final minutes from Feb. 5, 2020, meeting with FDA indicated agency agreed with statistical analysis plan, including primary and key secondary efficacy endpoints, for Stars study, expected to report in second quarter of 2020; clinical database remains locked and blinded
Novartis AG, of Basel, Switzerland Mayzent Siponimod Secondary progressive multiple sclerosis with active disease Received Health Canada approval
Novartis AG, of Basel, Switzerland Zolgensma (onasemnogene abeparvovec) Gene therapy expressing SMN Spinal muscular atrophy Japanese Ministry of Health, Labor and Welfare approved the therapy in patients under the age of 2
Novartis AG, of Basel, Switzerland, and its Avexis unit Zolgensma (onasemnogene abeparvovec) Gene therapy expressing SMN Spinal muscular atrophy EMA's Committee for Medicinal Products for Human Use adopted a positive opinion recommending conditional marketing authorization
Orchard Therapeutics plc, of London OTL-200 ARSA gene stimulator Metachromatic leukodystrophy Despite continuing engagement with EMA on submitted MAA under accelerated assessment, decision likely to be postponed due to COVID-19 pandemic; potential launch in Europe now expected in first half of 2021; BLA submission to FDA also postponed to first half of 2021
Pear Therapeutics Inc., of Boston Somryst Digital therapeutic Chronic insomnia FDA granted authorization for use in patients 22 and older
Pfizer Inc., and New York, and Eli Lilly and Co., of Indianapolis Tanezumab NGF-inhibiting monoclonal antibody Chronic pain due to moderate to severe osteoarthritis FDA accepted BLA seeking approval for use in patients who have experienced inadequate pain relief with other analgesics
Pharnext SA, of Paris PXT-3003 Oral fixed-dose combination of baclofen, naltrexone and sorbitol Charcot-Marie-Tooth disease type 1A U.K.'s Medicine and Healthcare products Regulatory Agency granted promising innovative medicine designation for use in patients 16 and older
Prevail Therapeutics Inc., of New York PR-006 AAV-based gene therapy Frontotemporal dementia FDA accepted IND for a phase I/II trial in patients with GRN mutation
Prevail Therapeutics Inc., of New York PR-006 AAV-based gene therapy Frontotemporal dementia FDA granted fast track designation to slow disease progression
Sage Therapeutics Inc., of Cambridge, Mass. Zuranolone Positive allosteric modulator of GABAA receptor Postpartum depression, major depression and major depressive syndrome Following an FDA breakthrough therapy guidance meeting, identified 3 pathways supporting possible filing for approval treat postpartum depression, acute treatment of major depressive disorder when co-initiated with a new antidepressant and as an episodic therapy in major depressive syndrome patients; 3 short-term clinical studies set to start in 2020
Seelos Therapeutics Inc., of New York SLS-005 (I.V. trehalose) Transcription factor EB stimulator Sanfilippo syndrome (mucopolysaccharidosis type III) Written response received from EMA, in lieu of face-to-face meeting, recommended use of natural history data, enabling design of open-label, non-placebo-controlled, pivotal phase IIb/III EU study in Sanfilippo syndrome types A and B
Stoke Therapeutics Inc., of Bedford, Mass. STK-001 Antisense oligonucleotide to up-regulate NaV1.1 protein expression Dravet syndrome FDA confirmed company can start part A of its phase I/IIa Monarch study; the agency placed part B testing a higher dose of the drug on a partial clinical hold, pending additional preclinical toxicology data
Sumitomo Dainippon Pharma Co. Ltd., of Osaka, Japan Latuda (lurasidone hydrochloride) Atypical antipsychotic Schizophrenia and bipolar depression Japanese Ministry of Health, Labor and Welfare approved the drug for both diseases
Teva Pharmaceutical Industries Ltd., of Tel Aviv, Israel Ajovy (fremanezumab) CGRP inhibitor Chronic migraine U.K.'s National Institute for Health and Care Excellence recommended drug in final appraisal document for prevention of migraine in adults with chronic migraine; recommendation is for patients who have not responded to at least 3 prior preventive drug treatments
Trevena Inc., of Chesterbrook, Pa. Oliceridine IV G protein-selective mu-opioid receptor agonist Moderate to severe acute pain PDUFA date of August 7, 2020
Virpax Pharmaceuticals Inc., of Malvern, Pa. NES-100 Nanotechnology for delivery of metabolically labile intranasal peptide Acute and chronic pain Completed a pre-IND application meeting with FDA
Zosano Pharma Corp., of Fremont, Calif. Qtrypta Formulation of zolmitriptan delivered using intracutaneous microneedle system Migraine FDA accepted the NDA under the 505(b)(2) pathway, setting a PDUFA date of Oct. 20, 2020
Company Product Description Indication Regulatory status

Ocular

Allergan plc, of Dublin Durysta (bimatoprost implant) Prostaglandin analogue for intracameral administration Open-angle glaucoma or ocular hypertension FDA approved NDA
Janssen Pharmaceutical Inc., of Raritan, N.J., part of Johnson & Johnson, and Meiragtx Holding plc, of London AAV-RPGR Gene therapy X-linked retinitis pigmentosa EMA granted both PRIME (Priority Medicines) and advanced therapy medicinal product designations
Nicox SA, of Sophia Antipolis, France NCX-470 Second-generation nitric oxide-donating bimatoprost analogue Open-angle glaucoma or ocular hypertension Completed end-of-phase II meeting with FDA and agreed on design of phase III program, nonclinical and CMC plans for submission of an NDA
Nicox SA, of Sophia Antipolis, France Vyzulta (latanoprostene bunod) PGF2 alpha agonist Ocular hypertension; open-angle glaucoma Partner Bausch & Lomb Inc., a unit of Bausch Health Cos. Inc. received approval in Taiwan for ophthalmic solution (0.024%) to reduce intraocular pressure in either indication
Outlook Therapeutics Inc., of Cranbury, N.J. ONS-5010 Ophthalmic formulation of VEGF inhibitor Wet age-related degeneration and other retinal diseases Received tentative FDA approval for trade name of Lytenava (bevacizumab-vikg), subject to further review at the time of planned BLA filing, expected in 2021
Company Product Description Indication Regulatory status

Other/miscellaneous

Dicerna Pharmaceuticals Inc., of Lexington, Mass. DCR-A1AT RNAi targeting SERPINA1 Alpha-1 antitrypsin deficiency FDA granted orphan drug designation
Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. Lonafarnib Farnesyltransferase inhibitor Progeria and progeroid laminopathies Submitted NDA to FDA
Rhythm Pharmaceuticals Inc., of Boston Setmelanotide Melanocortin-4 receptor agonist Pro-opiomelanotonin deficiency obesity and leptin receptor deficiency obesity Completed rolling NDA to FDA
Company Product Description Indication Regulatory status

Respiratory

Algernon Pharmaceuticals Inc., of Vancouver, British Columbia NP-120 (ifenprodil) N-methyl-D-aspartate receptor glutamate receptor antagonist Idiopathic pulmonary fibrosis and chronic cough Submitted for ethics approval in Australia for its planned phase II study
Bellerophon Therapeutics Inc., of Warren, N.J. Inopulse Pulsatile nitric oxide delivery system Pulmonary hypertension associated with pulmonary fibrosis Finalized, in consultation with FDA, key elements of planned pivotal phase III trial, including use of moderate to vigorous physical activity as the primary endpoint for approval, the population of PF subjects at risk of PH, as well as the dose of iNO45 (45 mcg/kg IBW/hr)
Boehringer Ingelheim GmbH, of Ingelheim, Germany Ofev (nintedanib) Small-molecule kinase inhibitor Chronic fibrosing interstitial lung diseases Approved by FDA for use in patients with a progressive phenotype
Genentech, of South San Francisco, a member of the Roche Group Esbriet Pirfenidone Unclassifiable interstitial lung disease FDA granted breakthrough therapy designation
Translate Bio Inc., of Lexington, Mass. MRT-5005 mRNA therapeutic Cystic fibrosis FDA granted rare pediatric disease designation
Virometix AG, of Schlieren, Switzerland V-306 Vaccine Respiratory syncytial virus Belgian health authority approved clinical trial application for phase I study in healthy volunteers
Company Product Description Indication Regulatory status

Toxicity/intoxication

Beyondspring Inc., of New York Plinabulin Immune and stem cell modulator Chemotherapy-induced neutropenia Initiated rolling submission of NDA to China's NMPA
Helsinn Healthcare SA, of Lugano, Switzerland Akynzeo (fosnetupitant/palonosetron) Netupitant prodrug Chemotherapy-induced nausea and vomiting European Commission approved the intravenous formulation of Akynzeo

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