Cancer

Advanced Accelerator Applications SA, of Saint-Genis-Pouilly, France

Lutathera (lutetium [177Lu] oxodotreotide)

Lutetium Lu 177-labeled somatostatin analogue peptide

Neuroendocrine tumors

Approved by Health Canada for use in unresectable or metastatic, well-differentiated, somatostatin receptor-positive gastroenteropancreatic NETs in adults with progressive disease

2/7/19

Agios Pharmaceuticals Inc., of Cambridge, Mass.

Tibsovo (ivosidenib)

Oral inhibitor of mutant isocitrate dehydrogenase 1

Acute myeloid leukemia

FDA accepted supplemental NDA to treat individuals with newly diagnosed AML with IDH1 mutation who are not eligible for standard therapy, granting priority review and setting PDUFA date of June 21, 2019

2/20/19

Aptose Biosciences Inc., of San Diego

CG-806

FLT3 and BTK kinase inhibitor

Relapsed or refractory B-cell malignancies

Submitted IND application to the FDA

2/25/19

Biolinerx Ltd., of Tel Aviv, Israel

BL-8040

Short synthetic peptide antagonist of CXCR4

Pancreatic cancer

FDA granted orphan status

2/4/19

CASI Pharmaceuticals Inc., of Rockville, Md.

Zevalin (ibritumomab tiuxetan injection)

CD20-directed radiotherapeutic antibody

Non-Hodgkin lymphoma

China's National Medical Products Administration approved clinical trial application for a confirmatory trial to evaluate the efficacy and safety in China

2/19/19

Celgene Corp., of Summit, N.J.

Revlimid (lenalidomide)

Immuno-modulatory drug

Previously treated follicular and marginal zone lymphoma

FDA granted priority review for supplemental NDA seeking approval in combination with rituximab; PDUFA date set for June 27, 2019

2/26/19

Cstone Pharmaceutical Co. Ltd., of Suzhou, China, and Impact Therapeutics Inc., of Nanjing, China

CS-1001 plus IMP-4297

Fully human anti-PD-L1 monoclonal antibody plus PARP inhibitor

Advanced solid tumors

China's National Medical Products Administration accepted the IND for clinical testing of the combination regimen in multiple tumor types

2/7/19

Daiichi Sankyo Co. Ltd., of Tokyo

Pexidartinib

Oral small-molecule inhibitor of CSF-1R

Symptomatic tenosynovial giant cell tumor

FDA accepted the NDA and granted priority review

2/5/19

EMD Serono, a unit of Merck KGaA, of Darmstadt, Germany, and Pfizer Inc., of New York

Bavencio (avelumab)

Anti-PD-L1 antibody

Advanced renal cell carcinoma

FDA accepted for priority review the sBLA, in combination with Inlyta (axitinib, Pfizer), and set a PDUFA date in June

2/11/19

Genentech Inc., of South San Francisco, a unit of Roche Holding AG

Kadcyla (ado-trastuzumab emtansine)

HER2-targeting antibody-drug conjugate

HER2-positive breast cancer

Completed submission of supplemental BLA for adjuvant use in patients with HER2-positive early breast cancer with residual disease after neoadjuvant treatment; FDA reviewing the application under Real-Time Oncology Review and Assessment Aid pilot programs

2/5/19

Genentech, of South San Francisco, a member of the Roche Group

Polatuzumab vedotin

Anti-CD79b antibody-drug conjugate

Relapsed or refractory diffuse large B-cell lymphoma

FDA accepted the BLA and granted priority review; application seeks approval for use in combination with bendamustine plus Rituxan (rituximab); FDA expected to make a decision by Aug. 19, 2019

2/19/19

Genentech, of South San Francisco, a member of the Roche Group

Entrectinib

Blocks ROS1 and NTRK kinase activity

NTRK fusion-positive, locally advanced or metastatic solid tumors

FDA accepted the NDA seeking approval for use in adult and pediatric patients who have either progressed following prior therapies or as initial therapy when there are no acceptable standard therapies, and for the treatment of people with metastatic, ROS1-positive non-small-cell lung cancer; FDA expected to make a decision by Aug. 18, 2019

2/19/19

Genmab A/S, of Copenhagen, Denmark

Darzalex (daratumumab)

Human CD38 monoclonal antibody

Multiple myeloma

FDA approved split dosing regimen for initial infusion

2/12/19

Hemispherx Biopharma Inc., of Ocala, Fla.

Ampligen (rintatolimod)

Double-stranded RNA with immunostimulatory and antiviral activity

Pancreas tumor

Extended its early access program at the Erasmus Medical Center in the Netherlands until March 9, 2020, to treat pancreatic cancer patients diagnosed with any stage of the serious disease

2/28/19

Idera Pharmaceuticals Inc., of Exton, Pa.

Tilsotolimod

Intratumorally delivered Toll-like receptor 9 agonist

Squamous cell carcinoma of the head and neck and microsatellite stable colorectal cancer

FDA cleared IND for the phase II ILLUMINATE-206 in combination with Yervoy (ipilimumab) and Opdivo (nivolumab); study expected to start in the second quarter of 2019

2/27/19

Imbrium Therapeutics LP, of Stamford, Conn.

Toniribate

Topoisomerase II inhibitor

Relapsed refractory biliary tract cancer

FDA granted orphan drug designation

2/22/19

Innovent Biologics Inc., of Suzhou, China

IBI-318

Recombinant fully human bispecific antibody targeting PD-1 and undisclosed target for a tumor-associated antigen

Hematological and advanced solid tumors

China's National Medical Products Administration cleared the IND for clinical trials

2/7/19

Karyopharm Therapeutics Inc., of Newton, Mass.

Selinexor

Oral selective inhibitor of nuclear transport (SINE) compound

Relapsed/refractory multiple myeloma

FDA's Oncologic Drugs Advisory Committee voted 8-5 in favor of delaying approval until the results of the randomized phase III trial, BOSTON, are available, potentially delaying approval by about two years

2/27/19

Lidds AB, of Uppsala, Sweden

Nanozolid

Docetaxel depot formulation

Cancer

Received approval from Danish Medicines Agency to conduct phase I trial testing intratumoral injections; first patients set to enroll in the NZ-DTX-001 trial in the first quarter of 2019

2/5/19

Merck & Co. Inc., of Kenilworth, N.J.

Keytruda (pembrolizumab)

Anti-PD-1 antibody

Metastatic squamous non-small-cell lung cancer

EMA's Committee for Medicinal Products for Human Use adopted a positive opinion for use in combination with carboplatin and either paclitaxel or nab-paclitaxel as first-line treatment in adults

2/4/19

Merck & Co. Inc., of Kenilworth, N.J.

Keytruda (pembrolizumab)

Anti-PD-1 antibody

First-line advanced renal cell carcinoma

FDA accepted the sBLA for Keytruda plus Inlyta (axitinib, Pfizer Inc.) and granted a priority review; target action date is 6/20/19

2/15/19

Merck & Co. Inc., of Kenilworth, N.J.

Keytruda (pembrolizumab)

Anti-PD-1 antibody

Melanoma

Approved by FDA f or adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection

2/19/19

Merck & Co. Inc., of Kenilworth, N.J.

Keytruda (pembrolizumab)

Anti-PD-1 antibody

Advanced small cell lung cancer

FDA accepted and granted priority review for supplemental BLA as monotherapy to treat SCLC following progression on 2 or more lines of therapy, setting PDUFA date of June 17, 2019

2/20/19

Moleculin Biotech Inc., of Houston

WP-1220

STAT3 inhibitor

Cutaneous T-cell lymphoma

Received approval in Poland to begin clinical trials for the topically administered drug

2/7/19

Moleculin Biotech Inc., of Houston

WP-1066

Immuno-stimulating STAT3 inhibitor

Glioblastoma

FDA granted orphan status

2/5/19

Noxxon Pharma NV, of Berlin

NOX-A12

Targets CXCL12

Brain cancer

Filed clinical trial application with Germany's Federal Institute for Drugs and Medical Devices for a phase I/II trial in combination with radiotherapy in newly diagnosed patients who would not benefit from current standard of care and whose tumor cannot be fully resected by surgery

2/27/19

Orion Corp., of Espoo, Finland, and Bayer AG, of Leverkusen, Germany

Darolutamide

Nonsteroidal androgen receptor antagonist

Non-metastatic castration-resistant prostate cancer

Completed rolling NDA submission with the FDA

2/27/19

Pfizer Inc., of New York

Vizimpro (dacomitinib)

Oral, once-daily, irreversible pan-human EGFR kinase inhibitor

Locally advanced or metastatic non-small-cell lung cancer

EMA's Committee for Medicinal Products for Human Use recommended approval as a monotherapy for first-line treatment in adults with EGFR-activating mutations

2/1/19

Pharmaessentia Corp., of Taiwan

Besremi (ropeginterferon alfa-2b, P-1101)

Single isomer mono-pegylated proline interferon 

Polycythemia vera independent of previous hydroxyurea exposure

European Commission approved

2/22/19

Roche Holding AG, of Basel, Switzerland

Herceptin Hylecta (trastuzumab and hyaluronidase)

Subcutaneous formulation using Halozyme Therapeutics Inc.'s Enhanze delivery technology

Breast cancer

Received FDA approval for treatment of certain people with HER2-positive early breast cancer in combination with chemotherapy and HER2-positive metastatic breast cancer in combination with paclitaxel or alone in people who have received 1 or more chemotherapy regimens

2/28/19

Shanghai Henlius Biotech Inc., of Shanghai

Hanlikang (HLX-01)

Biosimilar to rituximab

Non-Hodgkin lymphoma

Approved by China's National Medical Products Administration

2/25/19

Taiho Oncology Inc., of Princeton, N.J.

Lonsurf (trifluridine/tipiracil)

Nucleoside analogue and the thymidine phosphorylase

Metastatic gastric or gastroesophageal junction adenocarcinoma

FDA approved drug for treatment of patients previously treated with at least 2 prior lines of chemotherapy that included a fluoropyrimidine, a platinum, either a taxane or irinotecan, and if appropriate, HER2/neu-targeted therapy

2/25/19

Tyme Technologies Inc., of New York

SM-88

Dysfunctional tyrosine derivative

Third-line pancreatic cancer

At a type C meeting with the FDA, the company received feedback on the design of a pivotal trial that will use overall survival as the primary endpoint

2/25/19

Cardiovascular

Arca Biopharma Inc., of Westminster, Colo.

Gencaro (bucindolol hydrochloride)

Beta 1/2 adrenoceptor antagonist

Atrial fibrillation

Company reached agreement with the FDA regarding SPA on the design of pivotal phase III trial, PRECISION-AF, to assess safety and efficacy of the genetically targeted AF treatment in heart failure patients with midrange ejection fraction; the SPA provides that the design, trial population (400 participants) and statistical analyses of global study comparing Gencaro with Toprol-XL (metoprolol succinate) to prevent AF recurrence or all-cause mortality would support NDA filing

2/20/19

Hemostemix Inc., of Calgary, Alberta

ACP-01

Allogeneic cell therapy

Critical limb ischemia

Submitted an application to FDA for orphan status

2/4/19

Omeros Corp., of Seattle

OMS-721 

MASP-2 inhibitor

Hematopoietic stem cell transplant-associated thrombotic microangiopathy

After meeting with the FDA, the company decided to use a response-based analysis with the exact primary endpoint to be finalized in the near term; FDA to consider regular approval as well as accelerated approval based on data; company plans to submit first nonclinical sections as part of a rolling submission

2/14/19

Sanofi SA, of Paris

Praluent (alirocumab)

Anti-PCSK9 antibody

Reducing cardiovascular risk in patients with established atherosclerotic CV disease

EMA's Committee for Medicinal Products for Human Use recommended approval for a new indication as an adjunct to correction of other risk factors

2/4/19

Dermatologic

Aclaris Therapeutics Inc., of Wayne, Pa.

Eskata (hydrogen peroxide)

Cutaneous solution of hydrogen peroxide

Seborrheic keratoses

Approved by Swedish Medical Products Agency for use in SKs that are not pedunculated and have up to a maximum of diameter of 15 mm

2/28/19

Evolus Inc., of Newport Beach, Calif.

Jeuveau (prabotulinumtoxinA-xvfs)

Neurotoxin

Moderate to severe glabellar lines

Approved by FDA for use in temporary improvement in the appearance of moderate to severe glabellar lines associated with corrugator and/or procerus muscle activity in adults

2/1/19

Inflarx NV, of Jena, Germany

IFX-1

Monoclonal anti-complement factor C5a antibody

Pyoderma gangrenosum

Health Canada approved clinical trial application for a phase IIa trial

2/27/19

Janssen Pharmaceuticals Co., of New Brunswick, N.J.-based Johnson & Johnson

Tremfya (guselkumab)

Monoclonal antibody targeting IL-23

Moderate to severe plaque psoriasis

FDA approved administration via the One-Press single-dose, patient-controlled injector

2/27/19

Endocrine/Metabolic

Amicus Therapeutics Inc., of Cranbury, N.J.

AT-GAA

Recombinant human acid alpha-glucosidase plus a pharmacological chaperone

Pompe disease

FDA granted breakthrough designation

2/25/19

Astrazeneca plc, of Cambridge, U.K.

Forxiga (dapagliflozin)

Selective SGLT2 inhibitor

Type 1 diabetes

EMA's Committee for Medicinal Products for Human Use recommended a new indication, for use as an oral adjunct treatment to insulin in adults

2/1/19

Astrazeneca plc, of Cambridge, U.K.

Farxiga (dapagliflozin)

SGLT2 inhibitor

Type 2 diabetes

FDA approved label update expanding use in patients with moderate renal impairment (chronic kidney disease with an estimated glomerular filtration rate [eGFR] of 45-59 mL/min/1.73 m2)

2/27/19

Astrazeneca plc, of Cambridge, U.K.

Xigduo XR (dapagliflozin and metformin HCl extended-release)

SGLT2 inhibitor and antihyperglycemic agent

Type 2 diabetes

FDA approved label update expanding use in patients with moderate renal impairment (chronic kidney disease with an estimated glomerular filtration rate [eGFR] of 45-59 mL/min/1.73 m2)

2/27/19

Cerecor Inc., of Rockville, Md.

CERC-801

Ultra-pure, oral formulation of D-galactose

Phospho-glucomutase 1 deficiency (PGM1-CDG)

Received FDA fast track designation

2/19/19

Daiichi Sankyo Ltd., of Tokyo

Bempedoic acid and Bempedoic acid/ezetimibe fixed-dose combination tablet

Oral, once-daily ATP citrate lyase inhibitor

Hyperchol-esterolemia

EMA validated the marketing authorization application

2/28/19

Diurnal Group plc, of Cardiff, U.K., and Medison Pharma Ltd., of Petach Tikva, Israel

Alkindi (hydrocortisone)

Cortisol hormone granules in capsules for opening

Pediatric adrenal insufficiency

Ministry of Health in Israel validated the MAA; potential approval toward the end of 2019 and launch in Israel in 2020

2/25/19

Jiangsu Aosaikang Pharmaceutical Co. Ltd., of China

Saxagliptin generic

Dipeptidyl peptidase-4 inhibitor

Type 2 diabetes

Chinese drug regulators have granted marketing clearance

2/4/19

Modis Therapeutics Inc., of Oakland, Calif.

MT-1621

Deoxynucleoside combination therapy

Thymidine kinase 2 deficiency

FDA granted breakthrough therapy designation

2/19/19

Sanofi SA, of Paris

Soliqua 100/33 (insulin glargine and lixisenatide injection)

Insulin and GLP-1 receptor agonist

Type 2 diabetes

FDA approved expanded use as an add-on to diet and exercise in adults who are uncontrolled on long-acting insulin or lixisenatide

2/27/19

Translate Bio Inc., of Lexington, Mass.

MRT-5201

Delivers mRNA encoding fully functional OTC enzyme to liver

Ornithine transcarbamylase deficiency

Received formal communication from FDA regarding IND clinical hold; agency is requiring additional preclinical toxicology data to assess potential for adverse effects related to clearance time of the drug; company plans to complete those studies and submit a response in the fourth quarter of 2019

2/27/19

Gastrointestinal

Calliditas Therapeutics AB, of Stockholm, Sweden

Nefecon

Modified release version of budesonide

Primary biliary cholangitis

FDA granted orphan drug designation

2/12/19

Koutif Therapeutics LLC, of Cleveland

KT-1002

Selective small-molecule inhibitor of Fbxo3

Inflammatory bowel disease, including ulcerative colitis and Crohn's disease

Cleared by FDA to begin phase I trial

2/20/19

RDD Pharma Inc., of New York

RDD-0315

Alpha-agonist

Fecal incontinence in spinal cord injury

FDA granted fast track designation

2/14/19

Shionogi & Co. Ltd., of Osaka, Japan

Rizmoic (naldemedine)

Opioid antagonist

Opioid-induced constipation

European Commission approved for use in adults who have previously been treated with a laxative

2/22/19

Genitourinary/Sexual function

Adamis Pharmaceuticals Corp., of San Diego

APC-8000 (fast-disintegrating sublingual tadalafil)

PDE5 inhibitor

Erectile dysfunction

Received a refuse-to-file letter from FDA regarding the NDA; agency determined that application was not sufficiently complete to permit substantive review and requested longer real-time stability data and additional dissolution data for both clinical and registration batches

2/26/19

Cormedix Inc., of Berkeley Heights, N.J.

Neutrolin

Catheter lock solution

End stage renal disease undergoing hemodialysis

FDA told company it could request consideration for approval und the limited population pathway for antibacterial and antifungal drugs pathway

2/14/19

Hematologic

Apellis Pharmaceuticals Inc., of Crestwood, Ky.

APL-2

C3 complement inhibitor

Autoimmune hemolytic anemia

FDA granted orphan status

2/5/19

Astrazeneca plc, of Cambridge, U.K.

Fasenra (benralizumab)

Monoclonal antibody binding to IL-5 receptor alpha on eosinophils

Hypereosinophilic syndrome

FDA granted orphan designation

2/6/19

Chugai Pharmaceutical Co. Ltd., of Tokyo, and Roche Holding AG, of Basel, Switzerland

Hemlibra (emicizumab)

Bispecific monoclonal antibody combining factors IXa and X to mimic factor VIII

Hemophilia A

EMA's Committee for Medicinal Products for Human Use adopted a positive opinion for use as routine prophylaxis of bleeding episodes in adults and children with severe hemophilia A without factor VIII inhibitors

2/3/19

Novo Nordisk A/S, of Bagsvaerd, Denmark

Esperoct (turoctocog alfa pegol)

Extended half-life factor VIII molecule

Hemophilia A

FDA approved the BLA, granting use in treating adults and children

2/19/19

Sanofi SA, of Paris

Cablivi (caplacizumab-yhdp)

Caplacicumab-yhdp

Thrombotic thrombocytopenic purpura

FDA granted approval

2/7/19

Immune

Abbvie Inc., of North Chicago

Upadacitinib

Once-daily, oral JAK1 inhibitor

Moderate to severe rheumatoid arthritis

FDA accepted NDA for priority review; regulatory decision expected in third quarter of 2019

2/19/19

Alkermes plc, of Dublin, and Biogen Inc., of Cambridge, Mass.

Diroximel fumarate (BIIB-098)

Stimulates the Nrf2 pathway

Relapsing forms of multiple sclerosis

FDA accepted NDA; target action date is in the fourth quarter of 2019

2/25/19

ARS Pharmaceuticals Inc., of San Diego

ARS-1

Intranasal epinephrine spray

Severe allergic reactions to food, medications and insect bites that could lead to anaphylaxis

FDA granted fast track designation

2/19/19

Calliditas Therapeutics AB, of Stockholm

Nefecon

Locally acting formulation of budesonide

Autoimmune hepatitis

FDA granted orphan designation

2/5/19

DBV Technologies SA, of Montrouge, France

Viaskin Peanut

Epicutaneous immunotherapy

Peanut allergies

Plans to resubmit BLA in the third quarter of 2019

2/13/19

Equillium Inc., of La Jolla, Calif.

EQ-001

CD6-targeting monoclonal antibody

Acute graft-vs.-host disease

FDA granted orphan designations for both prevention and treatment of aGVHD

2/7/19

Incyte Inc., of Wilmington, Del.

Jakafi (ruxolitinib)

JAK inhibitor

Acute graft-vs.-host disease

FDA extended the review period for the supplemental NDA seeking approval for use in patients who have had an inadequate response to corticosteroids; the new PDUFA date is May 24, 2019

2/7/19

Infection

Aradigm Corp., of Hayward, Calif.

Pulmaquin (ciprofloxacin dispersion for inhalation)

Antimicrobial

Non-cystic fibrosis bronchiectasis

Type B meeting notes received from the FDA; the agency stated that based on the third-party evaluation (TPE) information presented by Aradigm it appears the TPE findings are consistent with the original phase III trial results, but the FDA continues to have concerns over the discordance in the results of the pre-specified primary and secondary endpoints between the ORBIT-3 and ORBIT-4 trials.

2/22/19

Astrazeneca plc, of Cambridge, U.K., and biologics R&D arm Medimmune

MEDI-8897

Extended half-life RSV F monoclonal antibody

Prevention of lower respiratory tract infection caused by respiratory syncytial virus

EMA granted access to PRIME (priority medicines) scheme

2/5/19

Astrazeneca plc, of Cambridge, U.K., and biologics R&D arm Medimmune

MEDI-8897

Extended half-life RSV F monoclonal antibody

Prevention of lower respiratory tract infection caused by respiratory syncytial virus

FDA granted breakthrough therapy designation

2/5/19

Entasis Therapeutics Holdings Inc., of Waltham, Mass.

ETX-2514SUL

Broad-spectrum inhibitor of class A, C and D beta-lactamases

Pneumonia and bloodstream infections caused by carbapenem-resistant Acinetobacter baumannii

Completed end-of-phase II meeting with FDA and remains on track to initiate phase III trial in the first quarter of 2019

2/5/19

Finch Therapeutics Group Inc., of Somerville, Mass.

CP-101

Encapsulated, orally administered Full-Spectrum Microbiota drug

Recurrent Clostridium difficile infection

FDA granted breakthrough therapy designation

2/8/19

Merck & Co. Inc., of Kenilworth, N.J.

Relebactam

Beta-lactamase inhibitor

Complicated urinary tract infections and complicated intra-abdominal infections caused by certain susceptible gram-negative bacteria

FDA accepted for filing the NDA seeking approval for relebactam in combination with imipenem/cilastatin (MK-7655A) for treatment of adults with limited or no alternative therapies available; agency granted priority review; PDUFA date is July 16, 2019

2/5/19

Merck & Co. Inc., of Kenilworth, N.J.

Zerbaxa (ceftolozane and tazobactam)

Cephalosporin antibacterial and beta-lactamase inhibitor

Nosocomial pneumonia, including ventilator-associated pneumonia caused by certain susceptible gram-negative microorganisms

FDA accepted for priority review a supplemental NDA; PDUFA date is June 3, 2019

2/5/19

Motif Bio plc, of New York

Iclaprim

Antibiotic targeting Gram-positive bacteria

Acute bacterial skin and skin structure infections

FDA issued a complete response letter that requested additional data to further evaluate the risk for liver toxicity

2/14/19

Nabriva Therapeutics plc, of Dublin

Lefamulin

Semisynthetic pleuromutilin antibiotic

Community-acquired bacterial pneumonia

FDA accepted the NDAs and granted priority review for both intravenous and oral formulations; PDUFA date is Aug. 19, 2019

2/19/19

Novartis AG, of Basel, Switzerland

Egaten (triclabendazole)

Binds tubulin

Fascioliasis

FDA approved drug for patients 6 years of age and older

2/13/19

Spero Therapeutics Inc., of Cambridge, Mass.

SPR-994

Oral carbapenem antibiotic

Complicated urinary tract infections

FDA accepted IND for a U.S. enrollment in a global, pivotal phase III trial

2/4/19

Spero Therapeutics Inc., of Cambridge, Mass.

SPR-720

Oral antimicrobial agent

Non-tuberculosis mycobacterial infections

FDA granted qualified infectious disease product designation for treating lung infections caused by NTM and lung infections caused by Mycobacterium tuberculosis

2/26/19

Themis Bioscience GmbH, of Vienna

MV-CHIK

Chikungunya vaccine

Chikungunya prophylaxis

FDA granted fast track designation

2/25/19

VLP Therapeutics Inc., of Gaithersburg, Md.

VLPM-01

Pre-erythrocytic vaccine delivering circumsporozoite antigen on inserted alphavirus VLP vaccine platform

Malaria

FDA cleared an IND for a phase I/IIa trial to assess safety, immunogenicity and efficacy

2/4/19

Xellia Pharmaceuticals AS, of Oslo, Norway

Premixed vancomycin injection in ready-to-use bag

Glycopeptide antibacterial

Septicemia, infective endocarditis, skin and skin structure infections, bone infections and lower respiratory tract infections

Approved by FDA

2/19/19

Inflammatory

Romeg Therapeutics LLC, of Woburn, Mass.

Gloperba (colchicine) oral solution 0.6 mg/5mL

Anti-mitotic drug

Prophylaxis of gout flares

Approved by FDA as first liquid formulation of colchicine for prevention of gout flares in adults

2/26/19

Samumed LLC, of San Diego

SM-04690

Small-molecule inhibitor of the Wnt pathway

Knee osteoarthritis

Completed end-of-phase II meeting with FDA to discuss phase III plan; pivotal studies STRIDES X-ray and STRIDES 1 expected to start in the first half of 2019

2/28/19

Takeda Pharmaceutical Co. Ltd., of Osaka, Japan

Uloric (febuxostat) 

Xanthine oxidase inhibitor

Gout

FDA added boxed warning to label; in-depth review of results from a safety clinical trial found an increased risk of heart-related death and death from all causes

2/22/19

Musculoskeletal

Ascendis Pharma A/S, of Copenhagen

Transcon CNP

Long-acting prodrug of C-type natriuretic peptide

Achondroplasia

FDA granted orphan drug designation

2/28/19

Biogen Inc., of Cambridge, Mass.

Spinraza (nusinersen)

Antisense oligonucleotide

Spinal muscular atrophy

Approved by China's National Medical Products Association for 5q SMA

2/28/19

Sarepta Therapeutics Inc.., of Cambridge, Mass.

Golodirsen (SRP-4053)

Phosphoro-diamidate morpholino oligomer to skip exon 53 of the DMD gene

Duchenne muscular dystrophy 

FDA accepted NDA filing and assigned a priority review with a target action date of 8/19/19

2/14/19

Neurology/Psychiatric

Alder Biopharmaceuticals Inc., of Bothell, Wash.

Eptinezumab

Calcitonin gene-related peptide binder

Migraine prevention

BLA filed

2/22/19

Alexion Pharmaceuticals Inc., of Boston

Soliris (eculizumab)

C5 complement inhibitor

Neuromyelitis optica spectrum disorder

FDA accepted for review the supplemental BLA seeking approval for use in patients who have anti-acquaporin-4 autoantibodies; agency granted prior review, with PDUFA date of June 28, 2019

2/22/19

Altan Pharma Ltd., of Dublin

Acetaminophen solution for infusion

Analgesic

Mild to moderate pain

Submitted an NDA to the FDA under the 505(b)(2) pathway; agency accepted application for review

2/7/19

Axsome Therapeutics Inc., of New York

AXS-07 (Moseic meloxicam/rizatriptan)

Fixed-dose combination of COX-2 preferential nonsteroidal anti-inflammatory drug and 5-HT1B/D agonist

Acute migraine

Reached agreement with FDA under a special protocol assessment for design of MOMENTUM phase III trial; study, which will enroll patients with history of inadequate response to prior treatments, is expected to start in the first quarter of 2019

2/6/19

Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn.

Verdiperstat (BHV-3241)

Myeloperoxidase inhibitor

Multiple system atrophy

FDA granted orphan designation

2/19/19

Cytogel Pharma LLC, of Darien, Conn.

CYT-1010

Endomorphin 1 analogue

Moderate to severe pain

FDA agreed the firm may proceed with phase Ia/IIa study in healthy volunteers and in subjects with moderate to severe pain after third molar extraction; after completion of dose-ranging study, company can submit data seeking clearance to advance to higher doses

2/5/19

Durect Corp., of Cupertino, Calif.

Posimir (Saber-bupivacaine)

Local anesthetic

Postoperative pain relief

Plans to submit full response to FDA's complete response letter following review of data and regulatory package; submission expected in first half of 2019, and company expects a 6-month FDA review period

2/27/19

Inhibikase Therapeutics Inc., of Atlanta

IkT-148009

Cellular Abelson tyrosine kinase inhibitor

Parkinson's disease and related disorders

Submitted 2 INDs to the FDA; 1 is seeking to treat PD using standard measures such as the Unified Parkinson's Disease Rating Scale as primary readout of treatment benefit, while the second will use diagnostic tolls and natural history patient data to valuate treatment benefit using new primary endpoints in the gastrointestinal tracts; studies expected to begin in the second quarter of 2019

2/26/19

Johnson & Johnson, of New Brunswick, N.J.

Esketamine

Non-competitive N-methyl-D-aspartate receptor antagonist

Treatment-resistant depression

FDA panel voted 14-2 in favor of esketamine 28-mg single-use nasal spray for TRD

2/13/19

Minoryx Therapeutics SA, of Barcelona, Spain

MIN-102

Oral, selective PPAR gamma agonist

Friedreich's ataxia

Received approval from Spanish Agency of Medicines and Medical Devices to start phase II FRAMES trial; expected to initiate recruitment in upcoming weeks in Europe

2/27/19

Pain Therapeutics Inc., of Austin, Texas

Remoxy ER

Abuse-deterrent, extended-release gel formulation of oxycodone

Pain severe enough to require daily, around-the-clock, long-term opioid treatment

Disclosed feedback from FDA meeting aimed to resolve disagreement during agency's 2018 NDA review and, as a result of that meeting, said firm is "no closer today to product approval" than it was a year ago; company said it will remain silent regarding plans or future expectations for the program

2/5/19

Pharnext SA, of Paris

PXT-3003

Oral fixed-dose combination of baclofen, naltrexone and sorbitol

Charcot-Marie-Tooth disease type 1A

FDA granted fast track designation

2/4/19

Satsuma Pharmaceuticals Inc., of South San Francisco

STS-101

5-HT 1d receptor agonist

Acute migraine

Met with FDA to establish key elements of phase III development program to support NDA filing; company plans to initiate randomized, double-blind, placebo-controlled trial in third quarter to evaluate efficacy and safety

2/20/19

SK Life Science Inc., of Fair Lawn, N.J.

Cenobamate (YKP-3089)

Antiepileptic believed to modulate GABAA receptors and inhibit sodium current

Partial-onset seizures

FDA accepted for filing the NDA seeking approval for use in adults

2/4/19

Sunovion Pharmaceuticals Inc., of Marlborough, Mass.

APL-130277

Apomorphine sublingual film

Parkinson's disease

FDA issued a complete response letter, requesting additional information and analyses, but no new clinical studies

2/1/19

Teva Pharmaceuticals Industries Ltd., of Jerusalem

Ajovy (fremanezumab)

CGRP-targeting monoclonal antibody

Migraine prophylaxis

EMA's Committee for Medicinal Products for Human Use recommended approval of the pre-filled syringe for use in adults who have at least 4 migraine days per month

2/1/19

Zogenix Inc., of Emeryville, Calif.

Fintepla (ZX-008)

Low-dose fenfluramine

Seizures associated with Dravet syndrome

Submitted an MAA to the EMA

2/6/19

Zogenix Inc., of Emeryville, Calif.

Fintepla (ZX-008)

Low-dose fenfluramine

Seizures associated with Dravet syndrome

Completed a rolling NDA to the FDA

2/6/19

Ocular

Alimera Sciences Inc., of Atlanta

Iluvien (fluocinolone acetonide intravitreal implant)

Sustained-release intravitreal implant

Diabetic macular edema

Product registration received from Ministry of Health in Lebanon, though partner Meagate International FZLLC

2/5/19

Bausch Health Cos. Inc., of Bridgewater, N.J.

Lotemax SM (loteprednol etabonate)

Corticosteroid

Postoperative inflammation and pain following ocular surgery

FDA approved drug

2/25/19

Clearside Biomedical Inc., of Alpharetta, Ga.

Xipere (triamcinolone acetonide, formerly CLS-TA)

Glucocorticoid receptor agonist

Macular edema associated with uveitis

FDA accepted for standard review the NDA, assigning PDUFA date of Oct. 19, 2019, consistent with company's expectations for a 505(b)(2) filing

2/20/19

Eyenovia Inc., of New York

Micropine

Microdose formulation of atropine

Reduce progression of myopia in children

FDA accepted the IND for the CHAPERONE phase III registration study, set to start in the first half of 2019

2/6/19

Ocugen Inc., of Malvern, Pa.

OCU-400

Gene therapy expressing NR2E3

NR2E3 mutation-associated retinal degenerative disease

FDA granted orphan drug designation

2/14/19

Other/Miscellaneous

Assertio Therapeutics Inc., of Lake Forest, Ill.

Long-acting cosyntropin

Synthetic adrenocorticotropic hormone

Diagnostic for screening patients for adrenocortical insufficiency

FDA accepted for filing the 505(b)(2) NDA

2/19/19

Eisai Co. Ltd., of Tokyo

Belviq (lorcaserin) and Belviq XR (lorcaserin)

Serotonin 2C receptor agonist

Obesity

FDA accepted sNDA to include data from CAMELLIA-TIMI 61 study of major adverse cardiovascular events 

2/25/19

Respiratory

Pulmatrix Inc., of Lexington, Mass.

Pulmazole (PUR-1900)

Inhaled Isperse formulation of antifungal itraconazole

Allergic bronchopulmonary aspergillosis in asthma patients

FDA cleared its IND for a phase II trial

2/7/19

Toxicity/Intoxication

Heron Therapeutics Inc., of San Diego

Cinvanti (aprepitant)

Polysorbate 80-free, I.V. formulation of an NK1receptor antagonist

Acute and delayed chemotherapy-induced nausea and vomiting

FDA approved supplemental NDA to expand administration method to include a 2-minute I.V. injection

2/26/19

Nuformix Ltd., of Cambridge, U.K.

NXP-001

Cocrystal version of currently marketed oncology therapy

Chemotherapy-induced nausea and vomiting

U.K.'s MHRA approved start of dosing in open-label study comparing bioavailability to Emend (fosaprepitant, Merck & Co. Inc.) in healthy subjects

2/27/19

Notes

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