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

 

Company Product Description Indication Regulatory status

Cancer

AB Science SA, of Paris AB-8939 Microtubule destabilizing agent Acute myeloid leukemia FDA designated the compound an orphan drug
Affimed NV, of Heidelberg, Germany AFM-24 EGFR modulator; immunoglobulin gamma Fc receptor IIIA agonist EGFR-expressing cancer Phase I/IIa IND application cleared FDA review
Astellas Pharma Inc., of Tokyo Xtandi (enzalutamide) Androgen receptor signaling inhibitor Metastatic castration-resistant prostate cancer after failure of androgen deprivation therapy National Medical Products Administration in China approved the NDA
Astrazeneca plc, of Cambridge, U.K. Calquence (acalabrutinib) BTK inhibitor Chronic lymphocytic leukemia or small lymphocytic lymphoma Approved by FDA for use in adults; granted approval under the FDA’s Real-Time Oncology Review and Project Orbis programs
Astrazeneca plc, of Cambridge, U.K. Imfinzi (durvalumab) PD-L1 inhibitor Small-cell lung cancer FDA accepted sBLA and granted priority review to treat previously untreated extensive-stage disease
Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. Selumetinib MEK 1/2 inhibitor Neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas FDA accepted the NDA with a priority review; PDUFA date in the second quarter of 2020
Autolus Therapeutics plc, of London AUTO-1 CD19 CAR T-cell therapy Acute lymphoblastic leukemia FDA granted orphan designation
Aveo Oncology Inc., of Cambridge, Mass. Tivozanib VEGF receptor tyrosine kinase inhibitor Renal cell carcinoma Plans to submit an updated statistical analysis plan for the final overall survival analysis, followed by an NDA in the first quarter of 2020
Beigene Inc., of Beijing Brukinsa (zanubrutinib) BTK inhibitor Mantle cell lymphoma Approved by FDA for use in adults who have received at least 1 prior therapy
Bio-Path Holdings Inc., of Houston BP-1002 Liposomal Bcl-2 Refractory/relapsed lymphoma and chronic lymphocytic leukemia FDA cleared the IND for a phase I trial
Bristol-Myers Squibb Co., of Princeton, N.J. Opdivo (nivolumab) and Yervoy (ipilimumab) PD-1 inhibitor and CTLA4 inhibitor Advanced hepatocellular carcinoma FDA accepted supplemental BLA and granted breakthrough therapy designation for use of combo in treating patients previously treated with sorafenib; application has priority review, with PDUFA date of March 10, 2020
Canbridge Pharmaceuticals Inc., of Beijing Nerlynx (neratinib) Irreversible tyrosine kinase inhibitor targeting pan-HER family Breast cancer Approved by Department of Health in Hong Kong for extended adjuvant treatment of adults with early stage HR-positive, HER2-overexpressed/amplified disease who completed adjuvant trastuzumab-based therapy less than 1 year ago; marks first Nerlynx approval in greater China
Celgene Corp., of Summit, N.J. Revlimid (lenalidomide) Immunomodulatory agent Follicular lymphoma EMA’s Committee for Medicinal Products for Human Use recommended approval in combination with rituximab for adults with previously treated disease
CNS Pharmaceuticals Inc., of Houston Berubicin for I.V. Anthracycline Glioblastoma multiforme Filed pre-IND meeting request to the FDA’s Administration Division of Oncology Products 2, Center for Drug Evaluation and Research
Compugen Ltd., of Tel Aviv, Israel COM-902 Antibody targeting TIGIT inhibitor Advanced malignancies IND cleared by FDA
Exelixis Inc., of Alameda, Calif., and Ipsen Biopharmaceuticals Canada Inc., part of Paris-based Ipsen SA Cabometyx (cabozantinib) Tyrosine kinase inhibitor Hepatocellular carcinoma Health Canada approved for use in patients who have not been previously treated with sorafenib
Faron Pharmaceuticals Ltd., of Turku, Finland Clevegen (FP-1305) Immunotherapy targeting Clever-1 positive tumor-associated macrophages Metastatic or inoperable solid tumors FDA cleared IND to expand ongoing phase I/II Matins study into the U.S.
Genmab A/S, of Copenhagen, Denmark Darzalex (daratumumab) CD38-targeting monoclonal antibody Multiple myeloma European Commission approved use in combination with lenalidomide and dexamethasone in adults with newly diagnosed disease who are ineligible for autologous stem cell transplant
Hutchison China Meditech Ltd. (Chi-Med), of Hong Kong Surufatinib Angio-immuno kinase inhibitor Pancreatic neuroendocrine tumors FDA granted orphan drug designation
Hutchison China Meditech Ltd. (Chi-Med), of Hong Kong Elunate (fruquintinib) VEGF-1/2/3 receptor antagonist Advanced colorectal cancer Drug included in updated National Reimbursement Drug List by China’s NHSA
Hutchison China Meditech Ltd., of London Surufatinib Oral angio-immuno kinase inhibitor Advanced non-pancreatic neuroendocrine tumors NDA accepted for review by China’s NMPA
Ideaya Biosciences Inc., of South San Francisco IDE-196 Small-molecule inhibitor of PKC Metastatic uveal melanoma Received minutes from end-of-phase I meeting with FDA and plan to start potentially registration-enabling phase II study testing monotherapy in 60 patients, with blinded independent central review determined overall response rate as the primary endpoint
Incyte Corp., of Wilmington, Del. Pemigatinib FGFR inhibitor Cholangiocarcinoma FDA accepted for priority review NDA seeking approval for use in patients with previously treated, locally advanced or metastatic disease with FGFR2 fusions or rearrangements
Inhibitor Therapeutics Inc., of Tampa, Fla. Suba-itraconazole Lanosterol-14 demethylase inhibitor Metastatic castrate-resistant prostate cancer IND application submitted to FDA for phase IIb trial evaluating drug in combination with docetaxel and prednisone
Janssen Pharmaceutical Cos., unit of Johnson & Johnson, of New Brunswick, N.J. Imbruvica (ibrutinib) BTK protein inhibitor Chronic lymphocytic leukemia; small lymphocytic lymphoma sNDA submitted to FDA for label expansion to include combination with rituximab to treat first-line disease
Merck & Co. Inc., of Kenilworth, N.J. Keytruda (pembrolizumab) Monoclonal antibody targeting PD-1 Metastatic or unresectable recurrent head and neck squamous cell carcinoma expressing PD-L1 European Commission approved the expanded indication as a monotherapy or in combination with platinum and 5-fluorouracil chemotherapy
Merck & Co. Inc., of Kenilworth, N.J. Keytruda (pembrolizumab) Monoclonal antibody targeting PD-1 First-line metastatic squamous non-small-cell lung cancer National Medical Products Administration in China approved the drug in combination with carboplatin and paclitaxel
Merck KGaA, of Darmstadt, Germany Tepotinib MET inhibitor Non-small-cell lung cancer with MET gene alterations Japanese Ministry of Health, Labour and Welfare granted orphan drug designation
Moleculin Biotech Inc., of Houston WP-1066 STAT3 inhibitor Recurrent or refractory malignant brain tumors FDA cleared an IND for a phase I trial
Oryx GmbH & Co. KG, of Munich, Germany Parvoryx Wild-type rat oncolytic parvovirus Recurrent glioblastoma multiforme Following 2 phase I/IIa trials, FDA and EMA will evaluate Oryx's proposed plan for further developmental studies
Roche Holding AG, of Basel, Switzerland Kadcyla (trastuzumab emtansine) HER2-targeting antibody-drug conjugate Breast cancer EMA’s Committee for Medicinal Products for Human Use recommended approval for use in adjuvant treatment of adults with HER2-positive early breast cancer who have residual invasive disease, in the breast and/or lymph nodes, after neoadjuvant taxane-based and HER2-targeted therapy
Roche Holding AG, of Basel, Switzerland Polivy (polatuzumab vedotin) Anti-CD79b antibody-drug conjugate Relapsed or refractory diffuse large B-cell lymphoma EMA’s Committee for Medicinal Products for Human Use recommended approval in combination with bendamustine plus Mabthera (rituximab) for adults who are not candidates for hematopoietic stem cell transplant
Samsung Bioepis Co. Ltd., of Incheon, South Korea SB-8 (bevacizumab biosimilar) VEGF ligand inhibitor Non-small-cell lung cancer FDA accepted BLA for review under 351(k) pathway to treat metastatic or recurrent nonsquamous NSCLC
Seattle Genetics Inc., of Bothell, Wash. Adcetris (brentuximab vedotin) CD30 modulator; tubulin receptor antagonist Systemic anaplastic large-cell lymphoma; peripheral T-cell lymphoma-not otherwise specified; angioimmunoblastic T-cell lymphoma Health Canada approved supplemental new drug submission to expand use to the indications, in combination with cyclophosphamide, doxorubicin and prednisone chemotherapy, in previously untreated adults whose tumors express CD30
Sesen Bio Inc. (formerly Eleven Biotherapeutics Inc.), of Cambridge, Mass. Vicinium (oportuzumab monatox) EpCAM modulator Bladder cancer Agreement reached with FDA during type C meeting on design of postmarketing confirmatory trial enrolling bacillus Calmette-Guérin (BCG)-refractory participants who received less-than-adequate BCG rather than originally proposed BCG-intolerant population; if approved, initial indication will be BCG-unresponsive patients who received adequate BCG
Teneobio Inc., of Newark, Calif. TNB-383B APRIL receptor modulator; CD3 modulator Multiple myeloma FDA granted orphan designation
Transthera Biosciences Co. Ltd., of Nanjing, China TT-00420 Small-molecule spectrum-selective multiple kinase inhibitor Cholangiocarcinoma FDA granted orphan designation
Triumvira Immunologics Inc., of Austin, Texas, and Hamilton, Ontario TAC01-CD19  Genetically engineered, patient-derived T cells to express CD19 T-cell antigen coupler Relapsed or refractory diffuse large B-cell lymphoma The FDA granted fast track designation for use in patients after at least 2 prior systemic therapies
Vascular Biogenics Ltd. (VBL Therapeutics), of Tel Aviv, Israel VB-111 (ofranergene obadenovec) CD95 modulator; TNF receptor modulator Recurrent glioblastoma FDA cleared IND for phase II trial of drug as neo-adjuvant and/or adjuvant therapy in participants undergoing second surgery
Verastem Inc., of Boston Copiktra (duvelisib) Phosphoinositide-3 kinase delta/gamma inhibitor Chronic lymphocytic leukemia/small lymphocytic lymphoma; follicular lymphoma MAA submitted to EMA for treatment of individuals with relapsed or refractory disease in both indications
Viracta Therapeutics Inc., of San Diego Nanatinostat (VRx-3996) Oral HDAC inhibitor Relapsed/refractory Epstein-Barr virus-positive lymphoid malignancies FDA granted fast track designation for use in combination with valganciclovir
Y-mabs Therapeutics Inc., of New York Naxitamab Anti-GD2 monoclonal antibody Relapsed/refractory high-risk neuroblastoma Submitted to FDA first portions of its rolling BLA
Company Product Description Indication Regulatory status

Cardiovascular

Actelion Pharmaceuticals Ltd., of Allschwil, part of Johnson & Johnson’s Janssen unit Opsumit (macitentan) Endothelin receptor antagonist Inoperable chronic thromboembolic pulmonary hypertension Decided to voluntarily withdraw European and other health authority filings still under review to extend indication of Opsumit; intends to generate additional data to support future filings in Europe, the U.S. and worldwide
Amarin Corp. plc, of Dublin Vascepa (icosapent ethyl) Ethyl eicosapentaenoic acid Cardiovascular disease FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted 16-0 to recommend approval expanding indication and label to reduce the risk of cardiovascular events in high-risk patients based on results from Reduce-IT cardiovascular outcomes trial
Correvio Pharma Corp., of Vancouver, British Columbia Brinavess (vernakalant hydrochloride) Relatively atrial selective antiarrhythmic agent Recent onset atrial fibrillation to sinus rhythm in adult patients On Dec. 10, the FDA will hold a Cardiovascular and Renal Drugs Advisory Committee meeting to review of the pending NDA
Cynata Therapeutics Ltd., of Melbourne, Australia Cymerus (CYP-002)
Mesenchymoangioblast-derived mesenchymal stem cell therapy
Critical limb ischemia Clinical trial authorization application for proposed phase II trial filed with U.K.'s MHRA
Kiniksa Pharmaceuticals Ltd., Hamilton, Bermuda Rilonacept Fusion protein that blocks IL-1alpha and IL-1beta signaling Recurrent pericarditis FDA granted breakthrough therapy designation
Company Product Description Indication Regulatory status

Dermatologic

Lifemax Laboratories Inc., of Palo Alto, Calif. LM-030 Kallikrein-related peptidase inhibitor Netherton syndrome European Commission granted orphan designation
MC2 Therapeutics A/S of Copenhagen Wynzora cream Topical fixed-dose combination of calcipotriene and betamethasone dipropionate Plaque psoriasis FDA accepted NDA for review; PDUFA date of July 20, 2020
Revance Therapeutics Inc., of Newark, Calif. DaxibotulinumtoxinA injectable Acetylcholine receptor antagonist; Botulinum toxin A stimulator Glabellar (frown) lines BLA submitted to FDA
Verrica Pharmaceuticals Inc., of West Chester, Pa. VP-102 (cantharidin 0.7% topical solution) Blistering agent Molluscum contagiosum NDA accepted for filing by FDA; PDUFA date of July 13, 2020
Company Product Description Indication Regulatory status

Endocrine/metabolic

Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Givosiran (injection for subcutaneous use) RNAi candidate targeting aminolevulinic acid synthase Acute hepatic porphyria FDA has approved the new RNAi therapy for the treatment of adults with the rare genetic disorder acute hepatic porphyria
Astrazeneca plc, of Cambridge, U.K. Qtrilmet (metformin hydrochloride, saxagliptin and dapagliflozin) Once-daily formulation comprising metformin, DPP-4 inhibitor and SGLT2 inhibitor Type 2 diabetes Approved by European Commission for use in adults to improve glycemic control
Axovant Gene Therapies Ltd., of New York AXO-AAV-GM1 Gene therapy GM1 gangliosidosis FDA granted orphan designation
Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis Empagliflozin SGLT2 inhibitor Type 1 diabetes FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted 14-2 that the benefits of empagliflozin 2.5 mg do not outweigh the risks to support approval as an adjunct to insulin
Generex Biotechnology Corp., of Miramar, Fla. Altsulin Microencapsulated Sertoli cells Laron syndrome Submitted phase IIa trial protocol to Ecuadorian Ministry of Public Health
Orphazyme A/S, of Copenhagen, Denmark Arimoclomol Hsp70 stimulator Niemann-Pick disease type C FDA granted breakthrough therapy designation
Protalix Biotherapeutics Inc., of Carmiel, Israel, and Chiesi Farmaceutici SpA, of Parma, Italy Pegunigalsidase alfa (PRX-102) Plant cent culture-expressed recombinant alpha-galactosidase-A enzyme Fabry disease Completed type B pre-BLA meeting with FDA and reached alignment on accelerated approval pathway; BLA to include data from completed phase I/II trials and from ongoing phase III Bridge trial; BLA submission expected by April 2020
Recordati SpA, of Milan, Italy Isturisa (osilodrostat) Inhibits 11-bta-hydroxylase Endogenous Cushing’s syndrome EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion recommended approval for use in adults
Company Product Description Indication Regulatory status

Gastrointestinal

Abivax SA, of Paris ABX-464 Binds to cap binding complex Moderate to severe active ulcerative colitis French regulators approved a phase IIb study
Immuron Ltd., of Melbourne, Australia IMM-124E Oral polyclonal antibody therapy Travelers’ diarrhea Following FDA feedback, company plans to file IND application during the first half of 2020 for phase III trial
Innovation Pharmaceuticals Inc., of Beverly, Mass. Brilacidin Defensin peptidomimetic Oral mucositis FDA granted waiver eliminating need for study on prevention of severe oral mucositis in head and neck cancer in pediatric patients receiving chemoradiation, clearing way to focus development on target adult population
Intercept Pharmaceuticals Inc., of New York Obeticholic acid Farnesoid X receptor agonist Fibrosis FDA accepted NDA under accelerated approval to treat fibrosis due to nonalcoholic steatohepatitis and granted priority review, setting PDUFA date of March 26, 2020
Poxel SA, of Lyon, France PXL-065 Deuterium-stabilized R-pioglitazone Nonalcoholic steatohepatitis FDA provided feedback supporting plan to advance drug in NASH using 505(b)(2) regulatory pathway, in part referencing Actos (pioglitazone) label
Company Product Description Indication Regulatory status

Genitourinary/sexual function

Agile Therapeutics Inc., of Princeton, N.J. Twirla (levonorgestrel/ethinyl estradiol) Transdermal system containing progestin and estrogen Contraception FDA extended PDUFA data by 3 months to Feb. 16, 2020
Evofem Biosciences Inc., of San Diego Amphora Vaginal pH regulator Prevention of pregnancy NDA resubmitted to the FDA
Knight Therapeutics Inc., of Montreal, and Therapeuticsmd Inc., of Boca Raton, Fla. Bijuva Estradiol and progesterone Moderate to severe vasomotor symptoms associated with menopause New drug submission accepted for review by Health Canada
Tricida Inc., of South San Francisco Veverimer (TRC-101) Non-absorbed orally administered polymer Chronic kidney disease FDA accepted the NDA under accelerated approval program; PDUFA date set for Aug. 22, 2020, and agency plans to hold a Cardiovascular and Renal Drugs Advisory Committee to discuss the application
Company Product Description Indication Regulatory status

Hematologic

Achillion Pharmaceuticals Inc., of Blue Bell, Pa. ACH-5228 Second-generation oral factor D inhibitor Paroxysmal nocturnal hemoglobinuria FDA cleared the IND for a drug-drug interaction study; company plans to start phase II proof-of-concept trial in PNH in first half of 2020
Achillion Pharmaceuticals Inc., of Blue Bell, Pa. Danicopan Complement factor D inhibitor Paroxysmal nocturnal hemoglobinuria EMA granted PRIME designation to treat individuals with PNH not adequately responding to C5 inhibitor
Biomarin Pharmaceutical Inc., of San Rafael, Calif. Valoctocogene roxaparvovec Gene therapy Severe hemophilia A Submitted MAA to EMA; anticipates start of review to commence in January 2020 under accelerated assessment
Celgene Corp., of Summit, N.J., and Acceleron Pharma Inc., of Cambridge, Mass. Reblozyl (Luspatercept-aamt) Erythroid maturation agent Anemia in beta-thalassemia Approved by FDA under priority review for use in patients who require regular red blood cell transfusions
Global Blood Therapeutics Inc., of South San Francisco Oxbryta (voxelotor) Oral drug designed to increase hemoglobin's affinity for oxygen Sickle cell disease FDA approved the drug
Novartis AG, of Basel, Switzerland Crizanlizumab (SEG-101) Humanized anti-P-selectin monoclonal antibody  Sickle cell disease Early FDA approval to reduce the frequency of vaso-occlusive crises (VOCs), or pain crises, in adult and pediatric patients ages 16 and older with sickle cell disease
Octapharma AG, of Aachen, Switzerland Fibryga Purified, dual pathogen-inactivated, lyophilized human fibrinogen concentrate for intravenous infusion Acquired fibrinogen deficiency Received approval to expand use to AFD in 15 European countries
Rigel Pharmaceuticals Inc., of South San Francisco Fostamatinib SYK inhibitor Chronic immune thrombocytopenia EMA’s Committee for Medicinal Products for Human Use recommended approval for use in adults who are refractory to other treatments
Sandoz AG, of Holzkirchen, Germany, a Novartis AG division Ziextenzo (pegfilgrastim-bmez) Biosimilar to pegfilgrastim Neutropenia Approved by FDA for use in patients with non-myeloid malignancies receiving myelosuppressive cancer treatment; Sandoz plans to launch this year
Company Product Description Indication Regulatory status

Immune

Bavarian Nordic A/S, of Copenhagen MVA-BN Filo Multivalent vaccine Ebola virus Marketing authorization application submitted to the EMA
Bio-Thera Solutions Ltd. (Guangzhou) BAT-1406 Humira biosimilar Rheumatoid arthritis, ankylosing spondylitis and plaque psoriasis National Medical Products Administration (NMPA) approved to treat indications such as rheumatoid arthritis, ankylosing spondylitis and plaque psoriasis
Celltrion Healthcare Co. Ltd., of Incheon, South Korea Remsima SC Subcutaneous biosimilar of infliximab Rheumatoid arthritis European Commission approved the drug
Janssen Pharmaceutical Cos., of Beerse, Belgium Ad26.ZEBOV Monovalent vaccine Ebola virus Marketing authorization application submitted to the EMA
Novartis AG, of Basel, Switzerland Mayzent (siponimod) Small-molecule agonist of S1P) receptors S1P1 and S1P5 Secondary progressive multiple sclerosis EMA’s Committee for Medicinal Products for Human Use recommended approval for use in patients with active disease evidenced by relapses or imaging features of inflammatory activity
Pfizer Inc., of New York Abrilada (adalimumab-afzb) Biosimilar of TNF-alpha inhibitor Humira Rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, adults Crohn’s disease, ulcerative colitis, plaque psoriasis Approved by FDA; Pfizer plans to launch in 2023
Sangamo Therapeutics Inc., of Brisbane, Calif. TX-200 Chimeric antigen receptor regulatory T-cell therapy Organ transplant rejection U.K.'s MHRA authorized first-in-human trial to prevent immune-mediated rejection following HLA-A2 mismatched kidney transplantation in end-stage renal disease
Company Product Description Indication Regulatory status

Infection

F2G Ltd., of Manchester, U.K. Olorofim (F-901318) Antifungal Invasive mold infections FDA granted breakthrough therapy designation for treating infections in patients with no or limited treatment options, including aspergillosis refractory or intolerant to currently available therapy, and infections due to Lomentospora prolificans, Scedosporium and Scopulariopsis species
Merck & Co. Inc., of Kenilworth, N.J. Ervebo Vaccine Ebola virus disease European Commission granted conditional marketing authorization for active immunization in individuals 18 and older to protect against disease caused by Zaire Ebola virus
Prokarium Ltd., of London Entervax Bivalent vaccine against enteric fever Enteric fever prophylaxis Clinical trial authorization accepted by the U.K. Medicines and Healthcare products Regulatory Agency; study to begin dosing in the first quarter of 2020
Redhill Biopharma Ltd., of Tel Aviv, Israel, and Raleigh, N.C. Talicia Omeprazole magnesium, amoxicillin and rifabutin H. pylori infection Approved by FDA for use in adults
Sanofi SA, of Paris Fluzone High-dose quadrivalent influenza vaccine Influenza FDA approved a supplemental BLA for use in adults 65 and older
Shionogi & Co. Ltd., of Osaka, Japan Fetroja (cefiderocol) Antibacterial drug Complicated urinary tract infections FDA approved for use in patients 18 and older with complicated UTIs, including kidney infections caused by susceptible gram-negative microorganisms, who have limited to no alternative treatment options
Valneva SE, of Saint Herblain, France Ixiaro Vaccine Prevention of Japanese encephalitis EMA approved extension of shelf-life from 24 months to 36 months
Company Product Description Indication Regulatory status

Musculoskeletal

3DBio Therapeutics, a unit of 3D Bio Holdings Inc., of New York Aurinovo 3D-bioprinted implant seeded with autologous auricular cartilage cells Microtia FDA granted orphan drug designation to combination candidate
Daewon Pharmaceutical Co. Ltd., of Seoul, South Korea Terrosa Biosimilar teriparatide Osteoporosis Ministry of Food and Drug Safety (MFDS) granted marketing approval in Korea
Solid Biosciences Inc., of Cambridge, Mass. SGT-001 Adeno-associated viral vector-mediated gene transfer candidate Duchenne muscular dystrophy FDA placed phase I/II study on hold
Company Product Description Indication Regulatory status

Neurology/psychiatric

Alexion Pharmaceuticals Inc., of Boston Soliris (eculizumab) C5 complement inhibitor Neuromyelitis optica spectrum disorder Japan’s Ministry of Health, Labour and Welfare approved extension of marketing authorization to include prevention of relapse in patients with AQP4 antibody-positive NMOSD, including neuromyelitis optica
Alkermes plc, of Dublin ALKS-3831 (olanzapine/samidorphan) 5-HT 2 receptor antagonist; dopamine D1/D2/D4 receptor antagonist Schizophrenia; bipolar I disorder Submitted NDA to FDA
Anavex Life Sciences Corp., of New York ANAVEX2-73 (blarcamesine) Targets sigma-1 and muscarinic receptors Rett syndrome FDA granted rare pediatric disease designation
Aquestive Therapeutics Inc., of Warren, N.J. Exservan (riluzole) oral film Glutamate receptor antagonist; sodium channel inhibitor Amyotrophic lateral sclerosis FDA issued full approval in advance of PDUFA date
Bionomics Ltd., of Melbourne, Australia BNC-210  Negative allosteric modulator of the alpha-7 nicotinic acetylcholine receptor Post traumatic stress disorder FDA granted fast track designation
Eip Pharma Inc., of Boston Neflamapimod Brain-penetrant, oral small-molecule inhibitor of p38 MAP kinase alpha Dementia with Lewy bodies FDA granted fast track designation
Genentech Inc., unit of Roche Holding AG, of Basel, Switzerland Risdiplam Survival motor neuron-2 splicing modifier Spinal muscular atrophy FDA accepted NDA and granted priority review, setting PDUFA date of May 24, 2020
Hope Biosciences Inc., of Houston HB-adMSCs Autologous, adipose-derived mesenchymal stem cells Alzheimer’s disease FDA approved the 24-patient phase I/II study
Jazz Pharmaceuticals plc, of Dublin Solriamfetol Dual-acting dopamine and norepinephrine reuptake inhibitor Narcolepsy EMA’s Committee for Medicinal Products for Human Use recommended approval to improve wakefulness and reduce excessive daytime sleepiness (EDS) in adults with narcolepsy (with or without cataplexy) or obstructive sleep apnea (OSA) whose EDS has not been satisfactorily treated by primary OSA therapy, such as continuous positive airway pressure
Minoryx Therapeutics SL, of Barcelona, Spain Leriglitazone (MIN-102) Brain-penetrant, oral selective PPAR? agonist Friedreich’s ataxia European Commission granted orphan designation
Newron Pharmaceuticals SpA, of Milan Sarizotan 5-HT 1a receptor agonist; dopamine D2 receptor agonist; dopamine D3/D4 receptor antagonist Rett syndrome FDA granted rare pediatric disease designation
Orchard Therapeutics Ltd., of London OTL-200 Gene therapy Metachromatic leukodystrophy EMA’s Committee for Medicinal Products for Human Use granted accelerated assessment
OWP Pharmaceuticals Inc., Naperville, Ill. Liquid formulation of topiramate Targets voltage-dependent sodium channels, GABA receptors and glutamate receptors Epilepsy and migraine FDA approved IND
Seelos Therapeutics Inc., of New York SLS-002 Intranasal racemic ketamine Acute suicidal ideation and behavior in major depressive disorder FDA granted fast track designation
Shanghai Green Valley (Group) Co. Ltd. of China GV-971 Marine-derived oligosaccharide Alzheimer's disease Chinese drug regulators granted conditional approval
SK Biopharmaceuticals Co. Ltd., of Pangyo, South Korea Xcopri (cenobamate tablets) Positive allosteric modulator of GABAA Partial-onset seizures Approved by FDA for use in adults
Sunovion Pharmaceuticals Inc., of Marlborough, Mass. Apomorphine sublingual film (APL-130277) Fast-acting version of dopamine agonist Parkinson’s disease Resubmitted NDA seeking approval to treat motor fluctuations (OFF episodes), in response to Jan. 29, 2019, complete response letter; resubmission included information about intended packaging as well as additional analyses of clinical data
Supernus Pharmaceuticals Inc., of Rockville, Md. SPN-812 Serotonin norepinephrine modulating agent Attention deficit hyperactivity disorder Submitted NDA to FDA
Tetra Bio-Pharma Inc., of Ottawa, Ontario Qixleef (PPP-001) Cannabinoid Pain FDA authorized advancement of previously discontinued 4-week phase II Plenitude trial of inhaled cannabis candidate in individuals with advanced cancer
Tonix Pharmaceuticals Holding Corp., of New York Tonmya (TNX-102 SL) Cyclobenzaprine sublingual tablets Post-traumatic stress disorder Received official minutes from type B meeting with FDA; phase III study design changes are being implemented after agency indicated importance of showing persistence of treatment effect at week 12 in a pivotal study; ongoing Recovery trial has more than 50% target enrollment, with interim analysis expected in first quarter of 2020 and top-line data in second quarter of 2020
Zogenix Inc., of Emeryville, Calif. Fintepla (ZX-008, fenfluramine oral solution) 5-HT 1d/2a/2c receptor modulator Dravet syndrome FDA accepted NDA filing for treatment of seizures associated with Dravet syndrome, granting priority review and setting PDUFA date of March 25, 2020
Zosano Pharma Corp., of Fremont, Calif. Qtrypta Formulation of the triptan zolmitriptan Acute migraine Completed pre-NDA meetings with the FDA; plans to submit NDA in December 2019
Company Product Description Indication Regulatory status

Ocular

Aerie Pharmaceuticals Inc., of Durham, N.C. Rhokiinsa (netarsudil ophthalmic solution) 0.02% Rho kinase inhibitor Open-angle glaucoma or ocular hypertension Approved by European Commission for use in reducing elevated intraocular pressure in adults
Proqr Therapeutics NV, of Leiden, the Netherlands QR-1123 Antisense oligonucleotide Autosomal dominant retinitis pigmentosa due to P23H mutation in rhodopsin gene Received orphan designation
Vertical Pharmaceuticals LLC, of Bridgewater, N.J. RVL-1201 Ophthalmic formulation of alpha-adrenergic receptor agonist oxymetazoline Acquired blepharoptosis FDA accepted the NDA; PDUFA action date of July 16, 2020
Company Product Description Indication Regulatory status

Other/miscellaneous

Eagle Pharmaceuticals Inc., of Woodcliff, N.J. Ryanodex (dantrolene sodium) Ryanodine receptor 1 antagonist Exertional heat stroke Company submitted a plan to the FDA to review data from all 41 patients from studies in 2015, 2018 and 2019
Endo International plc, of Dublin Collagenase clostridium histolyticum Enzyme targeting collagen Cellulite in the buttocks FDA accepted the BLA; PDUFA action date of July 6, 2020
Levo Therapeutics Inc., of Chicago LV-101 Intranasal carbetocin Prader-Willi syndrome FDA granted fast track designation
Medexus Pharmaceuticals Inc., of Montreal Gliolan 5-aminolevulinic acid hydrochloride Glioma imaging agent Health Canada granted priority review for new drug submission to guide maximal surgical resection of high-grade disease in adults
X4 Pharmaceuticals Inc., of Cambridge, Mass. Mavorixafor (X4-001) Oral, small-molecule antagonist of chemokine receptor CXCR4 WHIM (warts, hypogammaglobulinemia, infections and myelokathexis) syndrome FDA granted breakthrough therapy designation
Company Product Description Indication Regulatory status

Respiratory

Healios K.K., of Tokyo, and Athersys Inc., of Cleveland Multistem (HLCM-051) Cell therapy Acute respiratory distress syndrome Japan’s Pharmaceutical Affairs and Food Sanitation Council notified companies that the Ministry of Health, Labour and Welfare will grant orphan regenerative medicine designation within the next few weeks
Company Product Description Indication Regulatory status

Toxicity/intoxication

Adamis Pharmaceuticals Corp., of San Diego Zimhi (naloxone injection) Opioid receptor antagonist Opioid overdose FDA issued complete response letter on NDA, with questions generally related to CMC
Tonix Pharmaceuticals Holding Corp., of New York TNX-102 SL Cyclobenzaprine HCl sublingual tablets Alcohol use disorder Received minutes from recent type B pre-IND meeting with FDA; plans to submit IND in first quarter of 2020 to support phase II proof-of-concept study

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