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

Regulatory actions reported in February 2020

Company Product Description Indication Regulatory status


Alx Oncology Inc., of Burlingame, Calif. ALX-148 Next-generation CD47 myeloid checkpoint inhibitor Head and neck squamous cell carcinoma FDA granted fast track status
Alx Oncology Inc., of Burlingame, Calif. ALX-148 Next-generation CD47 myeloid checkpoint inhibitor HER2-positive gastric of gastroesophageal junction carcinoma FDA granted fast track status for second-line treatment
Aptorum Group Ltd., of Hong Kong SACT-1 Repurposed drug; small molecule Neuroblastoma Plans to leverage 505(b)(2) pathway and submit IND to FDA in second half of 2020
Ascentage Pharma Group International, of Suzhou, China APG-1387 Inhibitor of apoptosis proteins inhibitor Advanced pancreatic cancer Received approval from the Center of Drug Evaluation, China NMPA, to start a phase Ib/II study testing the drug in combination with chemotherapy (nab-paclitaxel plus gemcitabine)
Astex Pharmaceuticals Inc., of Pleasanton, Calif., a subsidiary of Otsuka Pharmaceutical Co. Ltd. Oral C-DEC (cedazuridine and decitabine) Fixed-dose combination of cytidine deaminase inhibitor and hypomethylating agent Myelodysplastic syndromes FDA accepted for priority review the NDA seeking approval for use in previously untreated patients with intermediate- and high-risk disease, including chronic myelomonocytic leukemia
Blueprint Medicines Corp., of Cambridge, Mass. Ayvakit (avapritinib) KIT kinase inhibitor Fourth-line gastrointestinal stromal tumor FDA extended PDUFA action date to May 14, 2020, to review data from the ongoing phase III Voyager study
Bold Therapeutics Inc., OF Vancouver, British Columbia BOLD-100 Cancer resistance pathway inhibitor Gastrointestinal cancer Health Canada cleared initiation of phase Ib combination trial with Folfox in gastric, pancreatic, colorectal and bile duct cancers
Bristol-Myers Squibb Co., of New York Lisocabtagene maraleucel Autologous anti-CD19 CAR T-cell immunotherapy Large B-cell lymphoma FDA accepted BLA to treat adults with relapsed/refractory disease after at least 2 prior therapies, granting priority review and setting PDUFA date of Aug. 17, 2020
Bristol-Myers Squibb Co., of Princeton, N.J. Opdivo (nivolumab) Anti-PD-1 antibody Esophageal cancer Japan’s Ministry of Health, Labor and Welfare approved for use in patients with unresectable advanced or recurrent disease that has progressed following chemotherapy
Can-Fite Biopharma Ltd., of Petach Tikva, Israel Namodenoson Adenosine A3 receptor agonist Hepatocellular carcinoma Submitted protocol design and registration plan for pivotal phase III trial to EMA's CHMP, seeking to evaluate efficacy of drug in individuals with advanced disease with underlying Child Pugh B7 cirrhosis whose cancer progressed on first-line therapy; study design of single phase III trial, already endorsed by FDA, would enable concurrent approval in U.S. and Europe
Coimmune Inc., of Durham, N.C. CMN-001 (formerly AGS-003) Dendritic cell-based immunotherapy Advanced metastatic renal cell carcinoma FDA cleared IND for phase IIb trial
Debiopharm Group, of Lausanne, Switzerland Debio-1143 Antagonist of inhibitor of apoptosis proteins Squamous cell carcinoma of the head and neck FDA granted breakthrough therapy designation
Deciphera Pharmaceuticals Inc., of Waltham, Mass. Ripretinib Dual KIT/PDGFR-alpha inhibitor Gastrointestinal stromal tumor FDA accepted NDA under Oncology Center of Excellence Real-Time Oncology Review pilot program, granting priority review and setting Aug. 13, 2020, PDUFA date
Eagle Pharmaceuticals Inc., of Woodcliff Lake, N.J. Pemfexy (pemetrexed for injection; branded alternative to Alimta) Chemotherapy Non-small-cell lung cancer and mesothelioma FDA granted final approval; indications include locally advanced or metastatic nonsquamous NSCLC in combination with cisplatin; locally advanced or metastatic nonsquamous NSCLC patients whose disease has not progressed after 4 cycles of platinum-based first-line chemotherapy, as maintenance treatment; locally advanced or metastatic nonsquamous NSCLC after prior chemotherapy as a single agent; and malignant pleural mesothelioma patients whose disease is unresectable or who are otherwise not candidates for curative surgery in combination with cisplatin
Eli Lilly and Co., of Indianapolis Cyramza (ramucirumab) VEGF receptor 2 antagonist Non-small-cell lung cancer FDA’s Oncology Drugs Advisory Committee voted 6-5 that the drug plus erlotinib demonstrated a favorable benefit/risk profile for patients with untreated metastatic EGFR-positive disease based on results of the phase III Relay study
Epizyme Inc., of Cambridge, Mass. Tazverik (tazemetostat) Methyltransferase inhibitor Relapsed or refractory follicular lymphoma FDA accepted NDA seeking approval for use in patients who have received at least 2 prior lines of systemic therapy; agency granted priority review, with a PDUFA date of June 18, 2020
Fennec Pharmaceuticals Inc., of Research Triangle Park, N.C. Pedmark Sodium thiosulfate Prevention of cisplatin-induced hearing loss (ototoxicity) in children with solid tumors Completed rolling submission of an NDA to the FDA for intravenous use; also submitted an MAA to the EMA
Genentech, of South San Francisco, a member of the Roche Group, and Halozyme Therapeutics Inc., of San Diego Fixed-dose combination of Perjeta (pertuzumab) and Herceptin (trastuzumab) with hyaluronidase HER2-targeted antibodies with hyaluronidase administered by subcutaneous injection HER2-positive breast cancer FDA accepted the BLA seeking approval for use in combination with intravenous chemotherapy
Genentech, of South San Francisco, a unit of Roche AG Tecentriq (atezolizumab) PD-L1-targeting antibody Advanced nonsquamous and squamous non-small-cell lung cancer FDA accepted supplemental BLA and granted priority review; application seeks approval for patients without EGFR or ALK mutations with high PD-L1 expression as determined by biomarker testing; PDUFA date is June 19, 2020
Glaxosmithkline plc, of London Zejula (niraparib) PARP inhibitor First-line maintenance treatment for platinum-responsive advanced ovarian cancer FDA accepted the sNDA under the real-time oncology review pilot program
Glaxosmithkline plc, of London Zejula (niraparib) PARP inhibitor Advanced ovarian cancer EMA validated type II variation for use of drug as maintenance treatment in the first-line setting for women who responded to platinum-based chemotherapy regardless of biomarker status; formal review process for EMA’s CHMP begins
Glaxosmithkline plc, of London Belantamab mafodotin BCMA monoclonal antibody conjugated to auristatin F Multiple myeloma EMA accepted MAA to treat relapsed/refractory disease in individuals whose prior therapy included immunomodulatory agent, proteasome inhibitor and anti-CD38 antibody; accelerated assessment granted by CHMP
Glycomimetics Inc., of Rockville, Md. GMI-1359 Inhibits adhesion molecules E-selectin and CXCR4 Osteosarcoma FDA granted orphan and rare pediatric disease designations
Harbour Biomed Therapeutics Ltd., of Cambridge, Mass. HBM-9167 Humanized IgG1 monoclonal antibody targeting PD-L1 Nasopharyngeal cancer FDA cleared the IND for a phase II study; agency also granted orphan designation
Incyte Corp., of Wilmington, Del. Capmatinib Selective MET inhibitor Non-small-cell lung cancer FDA accepted the NDA and granted priority review; seeking approval for use in locally advanced or metastatic MET exon 14-skipping mutated disease
Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson Darzalex Daratumumab Relapsed/refractory multiple myeloma Submitted an sBLA to the FDA for Darzalex in combination with Kyprolis (carfilzomib) and dexamethasone
Kiadis Pharma NV, of Amsterdam Off-the-shelf natural killer cells from universal donors Off-the-shelf natural killer cells from universal donors Acute myeloid leukemia FDA cleared clinical study by the Ohio State University in relapsed/refractory AML
Kite, of Santa Monica, Calif., a Gilead Co. KTE-X19 CAR T-cell therapy Relapsed or refractory mantle cell lymphoma FDA accepted the BLA and granted priority review designation
Kleo Pharmaceuticals Inc., of New Haven, Conn. KP-1237 CD38-targeting antibody recruiting molecule Multiple myeloma post-transplant FDA cleared IND for study testing the drug in combination with patients' natural killer cells that will begin the first half of 2020
Marker Therapeutics Inc., of Houston MultiTAA MAPP multi-antigen targeted T-cell therapy Post-transplant acute myeloid leukemia FDA lifted the clinical hold on planned trial investigating safety and efficacy
Merck & Co. Inc., of Kenilworth, N.J. Keytruda (pembrolizumab) PD-1-targeting antibody Multiple cancer indications FDA issued complete response letter regarding supplemental BLAs seeking to update dosing frequency to include a 400-mg dose infused over 30 minutes every six weeks
Monopar Therapeutics Inc., of Chicago Camsirubicin Doxorubicin analogue Soft-tissue sarcoma European Commission granted orphan designation
Nanobiotix SA, of Paris NBTXR-3 Designed to destroy tumors through physical cell death when activated by radiotherapy Head and neck squamous cell cancer FDA granted fast track designation for use, with or without cetuximab, in patients with locally advanced disease who are not eligible for platinum-based chemotherapy
Novartis AG, of Basel, Switzerland Capmatinib (INC-280) MET inhibitor METex14 mutated advanced non-small-cell lung cancer FDA granted priority review
Noxopharm Ltd., of Sydney Veyonda (idronoxil) S1P inhibitor Soft tissue sarcomas FDA cleared IND to test combination with doxorubicin
Pharmamar SA, of Madrid, Spain, and Jazz Pharmaceuticals plc, of Dublin Lurbinectedin Inhibits oncogenic transcription in tumor-associated macrophages Small-cell lung cancer FDA accepted NDA for filing with priority review, seeking accelerated approval for use in patients who have progressed after prior platinum-containing therapy; PDUFA date of Aug. 16, 2020
Puma Biotechnology Inc., of Los Angeles Nerlynx (neratinib) HER2-targeting therapy Breast cancer FDA approved a supplemental NDA for use in combination with capecitabine for treatment of adults with advanced or metastatic HER2-positive disease who have received 2 or more prior anti-HER2-based regimens in the metastatic setting
Roche Holding AG, of Basel, Switzerland Tecentriq (atezolizumab) Anti-PD-L1 antibody Small-cell lung cancer China’s NMPA approved for use in combination with carboplatin and etoposide for first-line treatment of patients with extensive-stage disease
Scancell Holdings plc, of Nottingham, U.K. SCIB-1 Immunoglobulin Fc receptor modulator Melanoma FDA approved IND for U.S. arm of phase II study in 25 individuals with metastatic disease also receiving pembrolizumab (Keytruda, Merck & Co. Inc.), using electroporation delivery device; study will assess whether addition of study drug to checkpoint inhibitor results in improved tumor response rate, progression-free survival and overall survival
Seattle Genetics Inc., of Bothell, Wash. Tucatinib Erbb2 tyrosine kinase receptor inhibitor Breast cancer FDA accepted NDA for study drug, in combination with trastuzumab and capecitabine, to treat locally advanced unresectable or metastatic HER2-positive disease, including brain metastases, in individuals who received at least 3 prior HER2-directed agents; agency granted priority review, setting Aug. 20, 2020, PDUFA date
Seattle Genetics Inc., of Bothell, Wash., and Astellas Pharma Inc., of Tokyo Padcev (enfortumab vedotin-ejfv) Antibody-drug conjugate directed against Nectin-4 Urothelial cancer FDA granted breakthrough therapy designation for use in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) in patients with unresectable locally advanced or metastatic disease who are unable to receive cisplatin-based chemotherapy in the first-line setting
Sellas Life Sciences Group, of New York Nelipepimut-S Targets the Wilms tumor 1 protein Triple-negative breast cancer Based on feedback from type C meeting with FDA, company finalized design for phase III registration-enabling study in TNBC patients in adjuvant setting after standard treatment; if successful, study may be considered basis of BLA filing
Sirnaomics Inc., of Gaithersburg, Md. STP-705 siRNA therapeutic Hepatocellular carcinoma Granted orphan drug designation by the FDA
Steba Biotech SA, of Luxembourg Tookad (padeliporfin dipotassium) Vascular-targeted photodynamic therapy Prostate cancer FDA’s Oncologic Drugs Advisory Committee voted 13-2 against recommending approval of the NDA seeking use in men with localized early stage disease
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan Alunbrig (brigatinib) Anaplastic lymphoma kinase tyrosine kinase inhibitor First-line ALK-positive metastatic non-small-cell lung cancer FDA granted a priority review for the sNDA; PDUFA action date of June 23, 2020
Tessa Therapeutics Ltd., of Singapore CD30 CAR T CD30-directed autologous CAR T-cell therapy Relapsed/refractory CD30-positive classical Hodgkin lymphoma FDA granted regenerative medicine advanced therapy designation
Tetra Bio-Pharma Inc., of Ottawa Qixleef Dried flower botanical cannabinoid Advanced cancer patients with uncontrolled pain In a letter of advice and type B meeting with the FDA, the agency validated the company's nonclinical program; the agency also validated the company's approach to ensuring the quality and lot-to-lot variation across the nonclinical and clinical programs
Ultragenyx Pharmaceutical Inc., of Novato, Calif., and Kyowa Kirin Co. Ltd., of Tokyo Crysvita (burosumab) Recombinant fully human monoclonal IgG1 antibody FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia) FDA accepted for review supplemental BLA seeking use in patients whose disease cannot be curatively resected or localized; agency assigned priority review, with PDUFA date of June 18, 2020
United Therapeutics Corp., of Research Triangle Park, N.C., and Deka Research & Development Corp., of Manchester, N.H. Remunity pump Subcutaneous delivery system for Remodulin (treprostinil) Pulmonary arterial hypertension FDA granted additional 510(k) clearance for drug reservoirs that have been prefilled by specialty pharmacies
Y-mabs Therapeutics Inc., of New York Omburtamab Monoclonal antibody targeting B7-H3 CNS/leptomeningeal metastases from neuroblastoma Completed type B pre-BLA meeting with FDA and reached alignment on accelerated approval pathway, along with rolling BLA; company expects to complete rolling BLA within 10 weeks
Company Product Description Indication Regulatory status


Esperion Inc., of Ann Arbor, Mich. Nexletol (Bempedoic acid) ATP citrate lyase inhibitor Heterozygous familial hypercholesterolemia (HeFH) or established atherosclerotic cardiovascular disease who require additional lowering of low-density lipoprotein cholesterol (LDL-C) FDA approval for bempedoic acid as an adjunct to diet and maximally tolerated statin therapy
Resverlogix Corp., of Calgary, Alberta Apabetalone BET inhibitor Major adverse cardiac events FDA granted breakthrough therapy designation, in combination with standard of care to include high-intensity statins, for secondary prevention of MACE in individuals with type 2 diabetes and recent acute coronary syndrome
Company Product Description Indication Regulatory status


Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. Berotralstat Plasma kallikrein inhibitor Hereditary angioedema Submitted an NDA to the Japanese Pharmaceuticals and Medical Devices Agency
Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. Berotralstat (BCX-7353) Oral kallikrein inhibitor Prevention of hereditary angioedema attacks FDA accepted the NDA and set PDUFA date of Dec. 3, 2020
Novan Inc., of Morrisville, N.C. SB-206 Nitric oxide-releasing topical gel Molluscum contagiosum FDA granted a type C meeting scheduled for April 1, 2020
Revance Therapeutics Inc., of Newark, Calif. Daxi DaxibotulinumtoxinA Moderate to severe glabellar lines FDA accepted BLA; PDUFA action date of Nov. 25, 2020; agency is not currently planning to hold an advisory committee meeting
Company Product Description Indication Regulatory status


Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Onpattro (patisiran) RNAi therapeutic Hereditary transthyretin-mediated amyloidosis Approved by Brazilian Health Regulatory Agency (ANVISA)
Diurnal Group plc, of Cardiff, U.K. Alkindi Sprinkle (hydrocortisone) Glucocorticoid receptor agonist Adrenal gland hypofunction FDA accepted NDA for review as replacement therapy for adrenal insufficiency in infants, children and adolescents, setting PDUFA date of Sept. 29, 2020
Eli Lilly and Co., of Indianapolis Trulicity (dulaglutide) GLP-1 receptor agonist Reduction of major adverse cardiovascular events in adults with type 2 diabetes FDA approved the expanded indication for patients who have established cardiovascular disease or multiple cardiovascular risk factors
Horizon Therapeutics plc, of Dublin Procysbi (cysteamine bitartrate) delayed-release oral granules Cystine-depleting agent Nephropathic cystinosis FDA approved oral granule dosage for use in adults and children, 1 and older
Logicbio Therapeutics Inc., of Cambridge, Mass. LB-001 Recombinant adeno-associated vector with human methylmalonyl-COA mutase gene Methylmalonic acidemia The FDA placed a clinical hold on the IND submission
Lysogene SA, of Paris LYS-SAF302 Gene therapy designed to deliver functional copy of the SGSH gene to the brain through 1-time direct-to-CNS administration Mucopolysaccharidosis type IIIA FDA granted fast track status
Prevail Therapeutics Inc., of New York PR-001 AAV-based gene therapy Gaucher disease FDA granted orphan designation; agency also granted rare pediatric disease designation for neuronopathic Gaucher disease
Protalix Biotherapeutics Inc., of Carmiel, Israel, and Chiesi Farmaceutici SpA, of Parma, Italy Pegunigalsidase alfa (PRX-102) Alpha-galactosidase stimulator Fabry disease Reached agreement with the FDA on an initial pediatric study plan
Company Product Description Indication Regulatory status


Ascletis Pharma Inc., of Hangzhou, China ASC-41 Oral thyroid hormone receptor-beta agonist Nonalcoholic steatohepatitis China’s NMPA accepted IND for clinical trials; drug is expected to be used in combination with ASC-40 an oral fatty acid synthase inhibitor
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan Vedolizumab SC Subcutaneous version of alpha4beta7 integrin antagonist Moderately to severely active ulcerative colitis or Crohn’s disease EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion for use as maintenance therapy in adults; Takeda proposes to make therapy available in both a prefilled syringe and a prefilled pen
Vivus Inc., of Campbell, Calif. Pancreaze (pancrelipase) Amylase stimulator; pancreatic lipase stimulator; protease stimulator Exocrine pancreatic insufficiency FDA approved sNDA for improved formulation that extends shelf life to 36 months across all dosages
Company Product Description Indication Regulatory status

Genitourinary/sexual function

Agile Therapeutics Inc., of Princeton, N.J. Twirla (levonorgestrel and ethinyl estradiol) Once-weekly hormonal contraceptive patch Contraception FDA approved for use in women with BMI < 30 kg/m2 for whom a combined hormonal contraceptive is appropriate
Therapeuticsmd Inc., of Boca Raton, Fla. Bijuva (estradiol + progesterone) Estradiol agonist; progesterone receptor agonist Severe vasomotor symptoms NDA prior-approval efficacy supplement submitted to FDA for 0.5 mg/100-mg dosage
Company Product Description Indication Regulatory status


Biomarin Pharmaceutical Inc., of San Rafael, Calif. Valoctocogene roxaparvovec AAV5 gene therapy Hemophilia A FDA accepted the BLA and granted priority review, setting PDUFA date of Aug. 21, 2020; FDA informed company no advisory meeting is currently planned
CTI Biopharma Corp., of Seattle Pacritinib FLT3/IRAK-1/JAK2 kinase inhibitor; CSF-1 antagonist Thrombocytopenia FDA agreed to accelerated approval pathway for treatment of myelofibrosis patients with severe disease (platelet counts <50,000/µL); pivotal phase III Pacifica trial protocol amendment will allow for primary analysis of SVR rates on first 168 participants, which, if met, will trigger company's NDA submission under FDA's subpart H regulations
Fibrogen Inc., of San Francisco Roxadustat Oral, small-molecule hypoxia-inducible factor prolyl hydroxylase inhibitor Anemia of chronic kidney disease FDA completed its filing review of NDA seeking approval for use in both nondialysis-dependent and dialysis-dependent patients and set PDUFA date of Dec. 20, 2020
Forma Therapeutics Inc., of Watertown, Mass. FT-4202 Pyruvate kinase-R activator Sickle cell disease FDA granted fast track and rare pediatric disease designations
Phasebio Pharmaceuticals Inc., of Malvern, Pa. PB-2452 Monoclonal antibody antigen-binding fragment targeting ticagrelor Antiplatelet reversal Granted PRIME designation by the EMA
Company Product Description Indication Regulatory status


Amgen Inc., of Thousand Oaks, Calif. Otezla (apremilast) PDE4 inhibitor Behçet’s disease EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion to extend use for treating ulcers in patients with Behçet’s
Aquestive Therapeutics Inc., of Warren, N.J. AQST-108 (sublingual epinephrine) Hormone Anaphylaxis During a pre-IND meeting, the FDA confirmed that the drug would be reviewed under 505(b)(2); plans to start pivotal pharmacokinetic studies by the end of 2020
DBV Technologies SA, of Montrouge, France Viaskin Peanut Epicutaneous immunotherapy Peanut allergy FDA will hold Allergenic Products Advisory Committee meeting May 15, 2020, to discuss BLA
Mesoblast Ltd., of Melbourne, Australia Ryoncil (remestemcel-L) Allogeneic cell therapy Graft-vs.-host disease Completed BLA submitted to FDA for treatment of children with steroid-refractory acute disease, with request for priority review based on fast track designation in indication
Novartis AG, of Basel, Switzerland, and Genmab A/S, of Copenhagen, Denmark Ofatumumab Monoclonal antibody targeting CD20 Relapsing forms of multiple sclerosis FDA accepted the sBLA and gave the application a priority review with a decision expected in June 2020; EMA accepted the MAA with a decision expected by the second quarter of 2021
Company Product Description Indication Regulatory status


Allovir Inc., of Cambridge, Mass. Viralym-M (ALVR-105) Allogeneic, off-the-shelf, multivirus-specific T-cell therapy Infection EMA granted PRIME designation for treating serious infections with BK virus, cytomegalovirus, human herpes virus-6, Epstein-Barr virus and/or adenovirus in allogeneic hematopoietic stem cell transplantation recipients
Cerovene Inc., of Valley Cottage, N.Y. Generic of Daraprim (pyrimethamine) tablets Dihydrofolate reductase inhibitor Toxoplasmosis FDA approved first generic for treating infection caused by Toxoplasma gondii when used with a sulfonamide
Contrafect Corp., of Yonkers, N.Y. Exebacase Lysin Methicillin-resistant Staphylococcus aureus bloodstream infections FDA granted breakthrough therapy designation
Cormedix Inc., of Berkeley Heights, N.J. Neutrolin (citrate + heparin + taurolidine) Antimicrobial catheter-lock solution Catheter-related blood stream infections FDA granted rolling review of NDA for prevention of CRBSIs in hemodialysis patients
Enochian Biosciences Inc., of Los Angeles ENOB-HV-01 Autologous stem cell transplantation HIV infection FDA scheduled an Initial Targeted Engagement for Regulatory Advice on CBER Products (INTERACT) meeting for early June
Merck & Co. Inc., of Kenilworth, N.J. Recarbrio (imipenem + cilastatin + relebactam) Beta lactamase inhibitor; dehydropeptidase-1 inhibitor Hospital-acquired and ventilator-associated bacterial pneumonia FDA accepted review sNDA to treat infections caused by susceptible gram-negative microorganisms, granting priority review and setting PDUFA date of June 4, 2020
Merck & Co. Inc., of Kenilworth, N.J. (also known as MSD outside the U.S. and Canada) Ervebo Ebola Zaire vaccine, live Zaire ebolavirus Confirmed 4 African countries, including Democratic Republic of Congo, approved vaccine
Seqirus Inc., of Holly Springs, N.C., unit of CSL Ltd., of Melbourne, Australia Audenz (influenza A [H5N1] monovalent vaccine) MF-59 adjuvanted cell-culture vaccine Influenza virus infection FDA approved pandemic influenza vaccine to treat individuals 6 months and older
Seqirus Inc., of Summit, N.J., a unit of CSL Ltd., of Melbourne, Australia Fluad Quadrivalent Influenza vaccine plus MF59 adjuvant Influenza infection prophylaxis FDA approved the drug for adults 65 years and older
Shionogi & Co. Ltd., of Osaka, Japan Fetcroja (cefiderocol) Siderophore cephalosporin antibiotic Aerobic gram-negative infections EMA’s Committee for Medicinal Products for Human Use adopted positive opinion for use in treating adults with limited treatment options
Zai Lab Ltd., of Shanghai and San Francisco Omadacycline Tetracycline antibiotic Community-acquired bacterial pneumonia and acute bacterial skin and skin structure infections China’s NMPA accepted the NDA
Company Product Description Indication Regulatory status


Eli Lilly Canada, a unit of Eli Lilly and Co., of Indianapolis Taltz (ixekizumab) IL-17 antagonist; interleukin 17A ligand inhibitor Ankylosing spondylitis Health Canada granted approval for individuals who responded inadequately or are intolerant to conventional therapy
Mereo Biopharma Group plc, of London Setrusumab Anti-sclerostin antibody Osteogenesis imperfecta Completed type B end-of-phase II meeting with FDA, which agreed to design of phase III pediatric study prior to submission of a BLA
Company Product Description Indication Regulatory status


Anavex Life Sciences Corp., of New York Anavex2-73 (blarcamesine) Activates sigma-1 receptor Rett syndrome FDA granted fast track designation
Aquestive Therapeutics Inc., of Warren, N.J. Libervant (diazepam) Buccal film version of benzodiazepine Management of seizure clusters FDA accepted NDA and assigned a PDUFA date of Sept. 27, 2020
Avenue Therapeutics Inc., of New York Tramadol (intravenous) Opioid receptor mu agonist Pain FDA accepted NDA for drug to manage moderate to moderately severe pain in adults in medically supervised health care setting, setting Oct. 10, 2020, PDUFA date
Baudax Bio Inc., of Malvern, Pa. Anjeso (meloxicam injection) 24-hour, intravenous COX-2 preferential nonsteroidal anti-inflammatory Management of moderate or severe pain Approved by FDA for use alone or in combination with other non-NSAID analgesics
Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. Nurtec ODT (rimegepant) CGRP receptor antagonist formulated in orally disintegrating tablet Acute migraine Approved by FDA for use in adults
Brainstorm Cell Therapeutics Inc., of New York Nurown Autologous mesenchymal stem cell-neurotrophic factor-producing cell therapy Amyotrophic lateral sclerosis Representatives from FDA's CBER confirmed during planned meeting that fully enrolled trial is collecting relevant data to assess efficacy; agency agreed to work collaboratively to identify regulatory pathway, including opportunities to expedite statistical review of phase III data
Cortexyme Inc., of South San Francisco COR-388 Gingipain inhibitor Alzheimer's disease FDA agreed during type C meeting to interim analysis of ongoing phase II/III Gain trial to assess evidence of overwhelming efficacy after about 100 participants in each of study's 3 arms complete 6 months of dosing, expected by year-end 2020; co-primary endpoints for interim analysis will be change from baseline in ADAS-Cog11 and CDR-SB vs. placebo
Eli Lilly and Co., of Indianapolis Emgality (galcanezumab) Monoclonal antibody targeting CGRP Episodic cluster headache EMA’s Committee for Medicinal Products for Human Use adopted a negative opinion to extend product’s indication for prevention of attacks in adults with episodic cluster headache
Eton Pharmaceuticals Inc., of Deer Park, Ill. ET-105 Oral liquid formulation of lamotrigine Epilepsy FDA requested changes to dosage and administration section of prescribing information to simplify dosing; agency also requested company conduct a human factors validation study with the revised labeling to show intended users can prepare and administer the oral suspension safely and effectively
Glaxosmithkline plc, of London Voltaren (diclofenac sodium topical gel) Nonsteroidal anti-inflammatory treatment Arthritis pain FDA approved 1% gel as over-the-counter product
GW Pharmaceuticals Ltd., of London, and U.S. subsidiary Greenwich Biosciences Inc., of Carlsbad, Calif. Epidiolex (cannabidiol) Dual cannabinoid CB1/CB2 receptor modulator Tuberous sclerosis complex sNDA submitted to FDA to treat seizures associated with TSC
H. Lundbeck A/S, of Valby, Denmark Vyepti (eptinezumab-jjmr) Monoclonal antibody targeting CGRP ligand Migraine prevention FDA approved the drug, which is expected to be available in April 2020
Heron Therapeutics Inc., of San Diego HTX-011 (bupivacaine + meloxicam) Cyclooxygenase 2 inhibitor; sodium channel inhibitor Postoperative pain FDA extended NDA review period by up to 3 months, resetting PDUFA date to June 26, 2020
Innocoll Holdings Ltd., of Athlone, Ireland Xaracoll (bupivacaine HCl collagen-matrix implants) Drug-device anesthetic Postsurgical pain Resubmitted NDA to FDA seeking approval for management of postsurgical pain after open inguinal hernia surgery; company anticipates a 6-month review
Lyndra Therapeutics Inc., of Watertown, Mass. Ultra-long-acting oral therapy Designed to open inside the stomach and deliver drugs over time Schizophrenia Filed an IND with the FDA
Neurosense Therapeutics, of Herzliya, Israel PrimeC Targets neuroinflammation and dysregulation of RNA metabolism Amyotrophic lateral sclerosis FDA granted orphan drug designation
Realta Life Sciences Inc., of Norfolk, Va. PIC-1 Anti-inflammatory, antioxidant Hypoxic-ischemic encephalopathy FDA granted orphan drug designation
Retrotope Inc., of Los Altos, Calif. RT-001 Chemically modified polyunsaturated fatty acid drug Progressive supranuclear palsy FDA granted orphan designation
Trevena Inc., of Chesterbrook, Pa. Oliceridine G protein-selective mu-opioid receptor agonist Moderate to severe acute pain Resubmitted NDA to FDA; company anticipates 6-month review period
Zhittya Genesis Medicine Inc., of Las Vegas Undisclosed Biologic candidate Parkinson’s disease Received approval from COFEPRIS, the Mexican regulator, for a phase I study
Zogenix Inc., of Emeryville, Calif. Fintepla (ZX-008, fenfluramine oral solution) Antiseizure drug Dravet syndrome FDA extended review period for NDA seeking approval to treat seizures associated with Dravet syndrome by 3 months; new PDUFA date is June 25, 2020
Company Product Description Indication Regulatory status


Novartis AG, of Basel, Switzerland Beovu (brolucizumab) Anti-VEGF antibody Wet age-related macular degeneration Approved by European Commission
Company Product Description Indication Regulatory status


Ionis Pharmaceuticals Inc., of Carlsbad, Calif. ION-373 Designed to stop a mutated gene from producing excess glial fibrillary acidic protein Alexander disease EMA granted orphan drug designation
Company Product Description Indication Regulatory status


Arrevus Inc., of Raleigh, N.C. ARV-1801 (sodium fusidate) Oral antibiotic Cystic fibrosis FDA granted orphan status
Boehringer Ingelheim GmbH, of Ingelheim, Germany Nintedanib Antifibrotic Systemic sclerosis-associated interstitial lung disease EMA's Committee for Medicinal Products for Human Use adopted a positive opinion recommending granting marketing authorization in adults
Glaxosmithkline plc, of London, and Innoviva Inc., of South San Francisco Trelegy Ellipta (fluticasone furoate/umeclidinium/vilanterol) Combination of inhaled corticosteroid, long-acting muscarinic antagonist and long-acting beta2-adrenergic agonist Asthma EMA accepted the regulatory submission seeking additional indication for the use of once-daily, single-inhaler triple therapy in adults
Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa. INO-3107 DNA medicine Recurrent respiratory papillomatosis FDA accepted the IND for a phase I/II trial
Translate Bio Inc., of Lexington, Mass. MRT-5005 mRNA therapeutic Cystic fibrosis FDA granted fast track designation
Company Product Description Indication Regulatory status


Acacia Pharma Group plc, of Cambridge, U.K. Barhemsys (amisulpride injection) Selective dopamine D2 and D3 antagonist Postoperative nausea and vomiting FDA approved drug for prevention and treatment of PONV in adults
Adamis Pharmaceuticals Corp., of San Diego Zimhi High-dose naloxone injection Opioid overdose During type A meeting with FDA to discuss 2019 complete response letter, company obtained concurrence from agency on CMC information required for resubmission of NDA and discussed potential public health role of drug in opioid epidemic; company said it believes it can generate necessary information and resubmit NDA early in second quarter of 2020
Adial Pharmaceuticals Inc., of Charlottesville, Va. AD-04 (repurposed ondansetron) 5-HT 3 receptor antagonist Alcohol use disorder Bulgarian Drug Agency and Bulgarian Ethics Committee for Clinical Trials approved initiation of sites in Bulgaria for ongoing phase III study in individuals with target genotypes related to serotonin transporter and receptor genes
Bioxcel Therapeutics Inc., of New Haven, Conn. BXCL-501 Alpha 2A adrenoceptor agonist Opioid withdrawal FDA cleared IND for initiation of phase Ib/II Release trial
Fennec Pharmaceuticals Inc., of Research Triangle Park, N.C. Pedmark Formulation of sodium thiosulfate Prevention of cisplatin-induced ototoxicity in pediatric patients Received EMA validation for the MAA and it is now under evaluation
Soligenix Inc., of Princeton, N.J. Rivax Protein subunit vaccine Ricin intoxication FDA granted fast track designation

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