Company
(location)

Product

Description

Indication

Status

Date

Cancer

AB Science SA, of Paris

AB-8939

Microtubule destabilizer

Acute myeloid leukemia

EMA validated clinical development program through a scientific advice procedure

7/4/19

Agilent Technologies Inc., of Santa Clara, Calif.

PD-L1 IHC 22C3 pharmDx assay

Companion diagnostic

Esophageal squamous cell carcinoma

Approved by FDA to identify patients with recurrent locally advanced or metastatic disease for treatment with PD-1 inhibitor Keytruda (pembrolizumab, Merck & Co. Inc.)

7/31/19

Astellas Pharma Inc., of Tokyo

Xtandi (enzalutamide)

Androgen receptor inhibitor

Metastatic hormone-sensitive prostate cancer

EMA accepted type II variation application for regulatory review

7/24/19

Astrazeneca plc, of Cambridge, Mass.

Imfinzi (durvalumab)

Anti-PD-L1 antibody

Small-cell lung cancer

FDA granted orphan designation

7/12/19

Astrazeneca plc, of Cambridge, U.K.

Imfinzi (durvalumab)

Monoclonal antibody targeting PD-L1

Stage III non-small-cell lung cancer

FDA approved the inclusion of overall survival data from the phase III Pacific study in the drug's label

7/22/19

Bayer AG, of Leverkusen, Germany

Vitrakvi (larotrectinib)

TRK inhibitor

Solid tumors with an NTRK gene fusion without a known acquired resistance mutation

Health Canada issued a notice of compliance with conditions

7/30/19

Bayer AG, of Leverkusen, Germany

Nubeqa (darolutamide)

Androgen receptor inhibitor

Non-metastatic castration-resistant prostate cancer

Approved by FDA under the priority review designation

7/30/19

Beigene Ltd., of Beijing

Tislelizumab

Fc-engineered anti-PD-1 antibody

Previously treated locally advanced or metastatic urothelial carcinoma

China's National Medical Products Administration granted priority review status for sNDA seeking use in patients with previously treated disease

7/7/19

Bexion Pharmaceuticals Inc., of Covington, Ky.

BXQ-350

Lysosomal activator protein saposin C and phosphatidyl-serine

Recurrent glioblastoma multiforme

At a type B meeting with the FDA, the agency provided guidance on the design of a phase II study expected to enroll 55-60 patients; plans to start study in first quarter of 2020

7/30/19

Bioinvent International AB, of Lund, Sweden

Anti-FcyRIIB antibody

Anti-FcyRIIB antibody

Solid tumors

FDA cleared IND for a phase I/IIa trial in combination with an anti-PD-1 antibody

7/2/19

Blueprint Medicines Corp., of Cambridge, Mass.

Avapritinib

Selective KIT and PDGFRA inhibitor

Advanced gastrointestinal stromal tumors

EMA validated marketing application for use in adults with PDGFRα D842V mutant GIST, regardless of prior therapy, and fourth-line GIST

7/18/19

Bristol-Myers Squibb Co., of New York

Empliciti (elotuzumab)

Targets signaling lymphocyte activation molecule family member 7, a cell-surface glycoprotein

Relapsed and refractory multiple myeloma

EMA's Committee for Medicinal Products for Human Use issued a positive opinion for use with Pomalyst (pomalidomide, Celgene Corp.) and low-dose dexamethasone in patients who have received at least 2 prior therapies

7/26/19

Celgene Corp., of Summit, N.J.

Pomalyst (pomalidomide)

Small-molecule immunomodulatory drug

Multiple myeloma

Health Canada approved triplet combination with bortezomib and dexamethasone for use in patients who have received at least 1 prior treatment regimen that included Revlimid (lenalidomide)

7/10/19

Celyad SA, of Mont-Saint-Guibert, Belgium

CYAD-02

Next-generation NKG2D-based CAR T therapy

Relapsed/refractory acute myeloid leukemia and myelodysplastic syndromes

FDA accepted IND, including OptimAb manufacturing process, for phase I trial set to start in 2020

7/1/19

Chia Tai Tianqing Pharmaceutical Holdings Co. Ltd., of China

Focus V (anlotinib hydrochloride capsules)

Small-molecule, multitargeted tyrosine kinase inhibitor

Soft tissue sarcoma

NMPA pproved the drug in China for a second indication to treat soft tissue sarcoma

7/11/19

Cstone Pharmaceuticals Co. Ltd., of Suzhou, China

Tibsovo (ivosidenib)

Targets mutant isocitrate dehydrogenase-1

Relapsed or refractory acute myeloid leukemia with an IDH1 mutation

China National Medical Products Administration approved a phase I bridging registrational study

7/22/19

Cytori Therapeutics Inc., of Austin, Texas

ATI-1123

Liposomal formulation of docetaxel

Platinum-sensitive small-cell lung cancer

FDA feedback indicated a 505(b)(2) application appears acceptable and agreed that completed nonclinical studies are sufficient to support initiation of the phase II trial in patients who have progressed at least 60 days after initiation of first-line therapy

7/1/19

Epizyme Inc., of Cambridge, Mass.

Tazemetostat

EZH2 inhibitor

Metastatic or locally advanced epithelioid sarcoma

NDA accepted for filing; seeking approval for use in patients not eligible for curative surgery

7/25/19

Foundation Medicine Inc., of Cambridge, Mass.

Foundationone CDx

Companion diagnostic for Lynparza (olaparib)

BRCA-mutated advanced ovarian cancer

Approved by FDA as companion diagnostic for first-line maintenance therapy with the PARP inhibitor

7/1/19

Helix Biopharma Corp., of Richmond Hill, Ontario

L-DOS47

Targets tumor microenvironment

Metastatic pancreatic cancer

Submitted IND to FDA for a phase I/II trial in combination with doxorubicin

7/9/19

Hookipa Pharma Inc., of New York and Vienna

HB-201

Therat-based immunotherapy

Human papillomavirus-positive cancers

FDA cleared the IND for a phase I/II trial

7/11/19

I-Mab Biopharma Co. Ltd., of Beijing

TJC-4

Monoclonal antibody targeting CD47

Advanced malignant tumors

China's National Medical Products Administration approved the IND

7/13/19

Immix Biopharma Inc., of Los Angeles

IMX-110

NF-kB/Stat3/pan-kinase inhibitor curcumin combined with a small amount of doxorubicin encased in a nano-sized delivery system

Solid tumors

Authorized by FDA to proceed with expanding its phase Ib/IIa trial to the U.S.

7/24/19

Iovance Biotherapeutics Inc., of San Carlos, Calif.

LN-145

Tumor-infiltrating lymphocyte-based immunotherapy

Advanced cervical cancer

Based on end-of-phase II meeting, FDA acknowledged that ongoing innovaTIL-04 study may be sufficient to support registration; potential BLA submission in second half of 2020

7/2/19

Janssen Pharmaceutical Cos., of Raritan, N.J., part of Johnson & Johnson, and Genmab A/S, of Copenhagen

Darzalex (daratumumab)

CD38-targeted antibody

Multiple myeloma

Submitted BLA seeking approval of new subcutaneous formulation

7/12/19

Janssen Pharmaceuticals Cos., of Beerse, Belgium, part of Johnson & Johnson, and Genmab A/S, of Copenhagen

Darzalex (daratumumab)

Monoclonal antibody targeting CD38

Multiple myeloma

Submitted an extension application to EMA for subcutaneous version co-formulated with recombinant human hyaluronidase PH20 (Halozyme Inc.)

7/19/19

Karyopharm Therapeutics Inc., of Newton, Mass.

Xpovio (selinexor)

Oral selective inhibitor of nuclear transport

Relapsed/refractory multiple myeloma

FDA granted accelerated approval for use in combination with corticosteroid dexamethasone to treat adults who have received at least 4 prior therapies and whose disease is resistant to several other forms of treatment, including at least 2 proteasome inhibitors, at least 2 immunomodulatory agents and an anti-CD38 monoclonal antibody

7/3/19

Kura Oncology Inc., of San Diego

KO-539

Menin-mixed lineage leukemia inhibitor

Acute myeloid leukemia

Given orphan drug designation by FDA

7/24/19

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

Keytruda (pembrolizumab)

Anti-PD-1 antibody

Multiple cancer indications

FDA accepted for review 6 supplemental BLAs to update dosing frequency to include an every-6-weeks schedule option for use in melanoma, classical non-Hodgkin lymphoma, primary mediastinal large B-cell lymphoma, gastric cancer, hepatocellular carcinoma and Merkel cell carcinoma; PDUFA date is Feb. 18, 2020

7/9/19

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

Keytruda (pembrolizumab)

PD-1 inhibitor

Renal cell carcinoma

EMA's Committee for Medicinal Products for Human Use recommended approval, in combination with tyrosine kinase inhibitor Inlyta (axitinib, Pfizer Inc.) for first-line treatment in individuals with advanced disease

7/29/19

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

Keytruda (pembrolizumab)

Anti-PD-1 monoclonal antibody

Recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus

FDA approved as a monotherapy for use in patients whose tumors express PD-L1 as determined by an FDA-approved test, with disease progression after 1 or more prior lines of systemic therapy

7/31/19

Merck & Co. Inc., of Kenilworth, N.J., and Eisai Inc., of Tokyo

Keytruda (pembrolizumab) plus Lenvima (lenvatinib)

Anti-PD-1 inhibitor and oral kinase inhibitor

First-line advanced unresectable hepatocellular carcinoma

FDA granted breakthrough therapy designation for the combination to treat patients whose disease is not amenable to locoregional treatment

7/23/19

Mereo Biopharma Group plc, of London

Navicixizumab 

Anti-DLL4/VEGF bispecific antibody

Advanced ovarian cancer

In a type B meeting with the FDA, the agency agreed to the outline of a phase II study using overall response rate as the primary endpoint; study could be used for accelerated approval

7/15/19

Mereo Biopharma Group plc, of London

Navicixizumab

Anti-DLL4/VEGF bispecific antibody

Advanced ovarian cancer

Held type B meeting with FDA, in which agency agreed in principle to an outline for phase II design to potentially support accelerated approval in patients, including those with peritoneal or fallopian tube cancers, who have become resistant to prior therapies; primary endpoint will be confirmed overall response rate

7/16/19

Mustang Bio Inc., of New York

MB-102

CD123 CAR T therapy

Acute myeloid leukemia

FDA granted orphan designation

7/24/19

Pfizer Inc., of New York

Ruxience (rituximab-pvvr)

Biosimilar to anti-CD20 antibody rituximab

Non-Hodgkin's lymphoma, chronic lymphocytic leukemia, granulomatosis with polyangiitis, microscopic polyangiitis

Approved by FDA

7/23/19

Propanc Biopharma Inc., of Melbourne, Australia

PRP

Proenzymes (trypsinogen and chymotrypsinogen)

Advanced cancer

Company plans to submit clinical trial application in the second half of 2019 and start the study in the first half of 2020

7/15/19

Puma Biotechnology Inc., of Los Angeles

Neratinib

Irreversible pan-HER inhibitor

HER2-positive metastatic breast cancer

Submitted supplemental NDA to FDA for approval in combination with capecitabine in patients who have failed 2 or more prior lines of HER2-directed treatment

7/1/19

Puma Biotechnology Inc., of Los Angeles, and Knight Therapeutics Inc., of Montreal

Nerlynx (neratinib)

Pan-HER inhibitor

Early stage HR-positive, HER2-overexpressed/amplified breast cancer

Drug received marketing authorization from Health Canada as an adjuvant treatment within 1 year after completion of trastuzumab-based adjuvant therapy

7/16/19

Roche Holding AG, of Basel, Switzerland

Tecentriq (atezolizumab)

Anti-PD-L1 antibody

Metastatic non-squamous non-small cell lung cancer

EMA's Committee for Medicinal Products for Human Use issued a positive opinion for use in combination with chemotherapy (carboplatin and Abraxane [albumin-bound paclitaxel; nab-paclitaxel, Celgene Corp.]) as first-line treatment of adults who do not have EGFR-mutant or ALK-positive NSCLC

7/26/19

Sanofi SA, of Paris

Isatuximab

Monoclonal antibody targeting an epitope on the CD38 receptor of a plasma cell

Relapsed/refractory multiple myeloma

FDA accepted BLA; PDUFA data of April 30, 2020

7/10/19

Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y.

Libtayo (cemiplimab)

Fully human monoclonal antibody targeting PD-1

Metastatic or locally advanced cutaneous squamous cell carcinoma

European Commission granted conditional marketing authorization for treating adults who are not candidates for curative surgery or radiation

7/1/19

Seattle Genetics Inc., of Bothell, Wash., and Astellas Pharma Inc., of Tokyo

Enfortumab vedotin

Antibody-drug conjugate comprising anti-Nectin-4 mAb attached to microtubule-disrupting agent MMAE

Locally advanced or metastatic urothelial cancer

Submitted BLA seeking accelerated approval for use in patients who have received a PD-1/L1 inhibitor and who have received a platinum-containing chemotherapy in the neoadjuvant/adjuvant, locally advanced or metastatic setting

7/16/19

Servier SAS, of Paris

Lonsurf (trifluridine/tipiracil) 

Thymidine-based nucleoside analogue and thymidine phosphorylase inhibitor

Metastatic gastric cancer

EMA's Committee for Medicinal Products for Human Use issued a positive opinion

7/26/19

Spring Bank Pharmaceuticals Inc., of Hopkinton, Mass.

SB-11285

STING agonist

Advanced solid tumors

Submitted IND to the FDA for a phase I trial

7/1/19

Spring Bank Pharmaceuticals Inc., of Hopkinton, Mass.

SB-11285

Stimulator of interferon gene (STING) agonist

Advanced solid tumors

FDA accepted IND application for phase I trial to evaluate safety, tolerability and initial anti-tumor activity

7/29/19

Springworks Therapeutics Inc., of Stamford, Conn.

Mirdametinib (formerly PD-0325901)

Inhibitor of MEK1 and MEK2

Neurofibromatosis type 1

European Commission granted orphan drug designation

7/30/19

Syndax Pharmaceuticals Inc., of Waltham, Mass.

SNDX-5613

Menin inhibitor

Relapsed/refractory acute leukemias

FDA cleared the IND for a phase I/II trial

7/10/19

Synthorx Inc., of San Diego

THOR-707

Recombinant interleukin-2 cytokine

Advanced or metastatic solid tumors

FDA cleared IND for patient enrollment in U.S. for phase I/II Hammer study

7/24/19

Transgene SA, of Strasbourg, France

TG-4050

Myvac MVA-based immunotherapy

Squamous cell carcinoma of the head and neck

U.K.'s MHRA cleared firm to proceed with phase I testing in newly diagnosed, locoregionally advanced, human papillomavirus-negative disease

7/10/19

Transgene SA, of Strasbourg, France

TG-6002

Next-generation oncolytic virus

Colorectal cancer

UK's MHRA cleared firm to proceed with phase I/IIa trial testing TG-6002 administered by intrahepatic artery infusion in patients with unresectable liver metastases

7/18/19

Trillium Therapeutics Inc., of Toronto

TTI-621

CD47 immune checkpoint inhibitor

Cutaneous T-cell lymphoma

FDA accepted company's request for an in-person meeting, currently scheduled later this quarter, to discuss proposed pivotal pathway for intratumoral administration of TTI-621 in early stage CTCL

7/24/19

Y-mabs Therapeutics Inc., of New York

Naxitamab

Humanized anti-GD2 3F8 monoclonal antibody

Relapsed/refractory high-risk neuroblastoma

Completed type B pre-BLA meeting with FDA and reached alignment on accelerated approval pathway along with rolling BLA submission; company plans to submit clinical/safety portion and nonclinical portion of BLA in November 2019 and expects to complete CMC portion in early 2020

7/8/19

Cardiovascular

Azurity Pharmaceuticals Inc., of Woburn, Mass.

Katerzia (amlodipine) oral suspension

Angioselective calcium channel blocker

Hypertension and coronary artery disease

Approved for hypertension by FDA for use in adults and pediatric patients, 6 and older, and for use in coronary artery disease in adults

7/10/19

Bayer AG, of Leverkusen, Germany

Gadavist (gadobutrol)

Gadolinium-based extracellular contrast agent

Cardiac magnetic resonance imaging agent

FDA approved for use to assess myocardial perfusion and late gadolinium enhancement in adults with known or suspected coronary artery disease

7/15/19

Caladrius Biosciences Inc., of Basking Ridge, N.J.

CLBS-12

CD34+ cell therapy

Critical limb ischemia

EMA granted advanced therapy medicinal product designation

7/15/19

Correvio Pharma Corp., of Vancouver, British Columbia

Brinavess (vernakalant hydrochloride)

Anti-arrhythmic drug

Atrial fibrillation

FDA accepted resubmitted NDA

7/25/19

Scpharmaceuticals Inc., of Burlington, Mass.

Furoscix

Furosemide solution formulated as wearable subcutaneous injector

Congestion in heart failure

Received minutes from type C meeting with FDA, with agency confirming it does not believe additional clinical safety, efficacy or pharmacology studies will be needed to file NDA; firm expects resubmission of NDA by mid-2020

7/16/19

Dermatologic

Exopharm Ltd., of Melbourne, Australia

Plexaris

Autologous exosomes from blood platelets

Wound healing

Melbourne Health Human Research Ethics Committee approved the 42-day phase I study measuring safety, wound closure and scar formation

7/22/19

Ilya Pharma Ab, of Uppsala, Sweden

ILP-100

Lactic acid bacteria expressing CXCL12

Healthy volunteers (eventually wound healing)

Swedish Medical Products Agency approved study

7/17/19

Lifemax Laboratories Inc., of Palo Alto, Calif.

LM-030

Kallikrein-related peptidase inhibitor

Netherton syndrome

FDA granted rare pediatric disease designation

7/18/19

Endocrine/Metabolic

Alnylam Pharmaceuticals Inc., of Cambridge, Mass.

Onpattro (patisiran)

RNAi therapeutic

Hereditary transthyretin-mediated amyloidosis

Received Health Canada approval for use in adults

7/23/19

Alnylam Pharmaceuticals Inc., of Cambridge, Mass.,

Givosiran

RNAi candidate targeting aminolevulinic acid synthase

Acute hepatic porphyria

Submitted MAA to EMA

7/1/19

Arrowhead Pharmaceuticals Inc., of Pasadena, Calif.

ARO-ANG3

RNAi targeting angiopoietin like protein 3

Homozygous familial hypercholesterolemia

FDA granted orphan drug designation

7/16/19

Astrazeneca plc, of Cambridge, U.K.

Farxiga (dapagliflozin)

SGLT2 inhibitor

Type 1 diabetes

FDA issued a complete response letter

7/15/19

Baxter International Inc., of Deerfield, Ill.

Myxredlin

Insulin human in 0.9% sodium chloride injection

Diabetes mellitus

FDA approved the ready-to-use I.V. infusion that has an extended shelf life of 30 days at room temperature or 24 months if refrigerated

7/22/19

Canbridge Pharmaceuticals Inc., a unit of Canbridge Life Sciences Ltd., of Beijing

Hunterase (idursulfase beta)

Recombinant human iduronate-2-sulfatase

Hunter syndrome

Filed NDA with the NMPA to treat the indication, also known as mucopolysaccharidosis type II, in China

7/29/19

Cerecor Inc., of Rockville, Md.

CERC-802

D-mannose

Mannose phosphate isomerase deficiency

FDA accepted NDA filing

7/29/19

Cytrx Corp., of Los Angeles, and Orphazyme A/S, of Copenhagen, Denmark

Arimoclomol 

Amplifies production of heat-shock proteins

Niemann-Pick disease type C

After a type C meeting with the FDA, Orphazyme remains on track to submit an NDA in the first half of 2020

7/30/19

Dicerna Pharmaceuticals Inc., of Cambridge, Mass.

DCR-PHXC

RNAi targeting lactate dehydrogenase A

Primary hyperoxaluria type 1

FDA granted breakthrough therapy designation; FDA said primary hyperoxaluria type 2 and 3 meet criteria for a serious or life-threatening disease or condition

7/15/19

Diurnal Group plc, of Cardiff, U.K.

Alkindi (hydrocortisone granules in capsules for opening)

Corticosteroid

Adrenal insufficiency

Submitted marketing application to Australian Therapeutics Goods Administration for use as replacement therapy in infants, children and adolescents, from birth to under 18 years

7/3/19

Eli Lilly and Co., of Indianapolis

Baqsimi (glucagon) nasal powder

Glucagon receptor agonist

Hypoglycemia

FDA approval for the treatment of severe hypoglycemia in patients with diabetes aged four years and older.

7/25/19

Horizon Therapeutics plc, of Dublin

Procysbi (cysteamine bitartrate) delayed-release oral granules in packets

Cystine-depleting agent

Nephropathic cystinosis

FDA accepted NDA for new dosage form; decision expected in 2020

7/18/19

Khondrion BV, of Nijmegen, the Netherlands

KH-176

Small-molecule antioxidant and redox modulator

Maternally inherited diabetes and deafness

European Commission granted orphan designation for the subtype of diabetes often accompanied by hearing loss caused by mutation within mitochondrial DNA

7/2/19

Logicbio Therapeutics Inc., of Cambridge, Mass.

LB-001

Recombinant adeno-associated vector with human methylmalonyl-COA mutase gene

Methylmalonic acidemia

FDA granted rare pediatric disease designation

7/16/19

Orphazyme A/S, of Copenhagen

Arimoclomol

Amplifies the production of heat-shock proteins

Niemann-Pick disease type C

Following a meeting with the FDA, the company remains on track to submit an NDA in the first half of 2020

7/21/19

Rhythm Pharmaceuticals Inc., of Boston

Setmelanotide

Melanocortin MC4 receptor agonist

Bardet-Biedl syndrome

EMA's Committee for Orphan Medicinal Products issued positive opinion on application for orphan designation in the indication

7/29/19

Seelos Therapeutics Inc., of New York

SLS-005 (trehalose)

Low molecular weight disaccharide

Sanfilippo syndrome

Company submitted an IND to the FDA for a phase IIb/III study

7/17/19

Zafgen Inc., of Boston

ZGN-1061

MetAP2 inhibitor 

Metabolic disorders

Reached agreement with FDA on an in vivo animal study design and protocol to establish relevant safety margins

7/25/19

Gastrointestinal

Genfit SA, of Lille, France

Elafibranor

PPAR alpha/delta agonist

Primary biliary cholangitis

FDA and EMA granted orphan drug designation in the indication

7/29/19

Genkyotex SA, of Archamps, France

Setanaxib (GKT-831)

NADPH oxidase (NOX) inhibitor

Primary biliary cholangitis

World Health Organization recommended setanaxib as the international nonproprietary name and adopted naxib as the stem for NOX inhibitors

7/22/19

Hepion Pharmaceuticals Inc., of Edison, N.J.

CRV-431

Inhibits cyclophilin enzymes

Non-alcoholic steatohepatitis

FDA authorized IND for study testing the safety, tolerability and pharmacokinetics of the drug; study will enroll 24 patients and up to an additional 24 healthy subjects

7/29/19

Janssen Pharmaceutical Cos., unit of Johnson & Johnson, of New Brunswick, N.J.

Stelara (ustekinumab)

IL-12/23 receptor antagonist

Ulcerative colitis

EMA's Committee for Medicinal Products for Human Use recommended marketing authorization in EU to treat adults with moderately to severely active disease and inadequate or lost response or intolerance to other therapy

7/26/19

Lipocine Inc., of Salt Lake City

LPCN-1144

Oral prodrug of bioidentical testosterone

Nonalcoholic steatohepatitis

FDA cleared the firm to investigate drug in an expanded target population of adult male NASH patients, specifically waiving the limitation of only testing NASH subjects with total testosterone levels below 300 ng/dL

7/23/19

Genitourinary/Sexual Health

Akebia Therapeutics Inc., of Cambridge, Mass.

Vadadustat

Oral hypoxia-inducible factor prolyl hydroxylase inhibitor

Anemia due to chronic kidney disease

Partner Mitsubishi Tanabe Pharma Corp. submitted a Japanese NDA

7/23/19

Regulus Therapeutics Inc., of La Jolla, Calif.

RGLS-4326

Oligonucleotide designed to inhibit miR-17

Autosomal dominant polycystic kidney disease

FDA placed IND on partial clinical hold following review of interim analyses from a new mouse chronic toxicity study and nonhuman primate (NHP) chronic toxicity study; in order to resume multiple ascending-dose study, FDA requested final reports from chronic toxicity studies in both mice and NHP and satisfactory related analyses to ensure subjects can be safely dosed

7/23/19

Hematologic

Alexion Pharmaceuticals Inc., of Boston

Ultomiris (ravulizumab)

Long-acting C5 complement inhibitor

Paroxysmal nocturnal hemoglobinuria

European Commission approved for use in adults with hemolysis with clinical symptoms indicative of high disease activity and also for adults who are clinically stable after treating with Soliris for at least the past 6 months

7/3/19

Biomarin Pharmaceutical Inc., of San Rafael, Calif.

Valoctocogene roxaparvovec

Gene therapy

Severe hemophilia A

Plans to submit marketing applications to both FDA and EMA in the fourth quarter 2019

7/8/19

CTI Biopharma Corp., of Seattle

Pacritinib

JAK2 inhibitor

Myelofibrosis

Following type B, end-of-phase IIa meeting with FDA, CTI plans phase III study to test 200 mg administered twice daily in 180 patients with myelofibrosis and severe thrombocytopenia; Pacifica study set to start in third quarter of 2019

7/18/19

Momenta Pharmaceuticals Inc., of Cambridge, Mass.

Nipocalimab (M-281)

Monoclonal antibody targeting FcRn

Hemolytic disease of the fetus and newborn

FDA granted fast track designation

7/30/19

Novartis AG, of Basel, Switzerland

Crizanlizumab (SEG-101)

MAb targeting P-selectin-mediated multicellular adhesion

Sickle cell disease

FDA accepted BLA and granted priority review

7/16/19

Novo Nordisk A/S, of Bagsvaerd, Denmark

Esperoct (antihemophilic factor VIII [recombinant, B-domain truncated] pegylated)

Extended half-life factor VIII molecule

Hemophilia A

Approved by Health Canada for use in adults and children for routine prophylaxis, on-demand treatment and control of bleeding episodes and perioperative management of bleeding

7/12/19

Omeros Corp., of Seattle

Narsoplimab (OMS-721)

Monoclonal antibody targeting mannan-binding lectin-associated serine protease-2

Hematopoietic stem cell transplant-associated thrombotic microangiopathy

Company reached agreement with the FDA finalizing the endpoint for the pivotal study that includes laboratory measures and markers of organ function as well as platelet and red blood cell transfusion burden

7/11/19

Vivacelle Bio Inc., of Chicago

VBI-S

Injectable fluid composed of phospholipid nanoparticles

Hypovolemic shock

FDA cleared IND for a phase IIa trial

7/8/19

Immune

Atara Biotherapeutics Inc., of South San Francisco

Tab-cel (tabelecleucel)

Allogeneic T-cell immunotherapy

Epstein-Barr virus-associated post-transplant lymphoproliferative disease

Based on recent FDA discussions, company said it plans to initiate BLA submission in second half of 2020

7/16/19

Celgene Corp., of Summit, N.J.

Otezla (apremilast)

Oral PDE4 inhibitor

Oral ulcers associated with Behçet's disease

Approved by FDA for use at 30 mg twice daily to treat adults

7/19/19

Cyxone AB, of Stockholm

Rabeximod

Immuno-modulator

Rheumatoid arthritis

Submitted application to Polish regulators for permission to start phase IIb trial

7/5/19

Gilead Sciences Inc., of Foster City, Calif., and Galapagos NV, of Mechelen, Belgium

Filgotinib

Oral, selective JAK1 inhibitor

Rheumatoid arthritis

During recent pre-NDA meeting with FDA, a path forward was established to submit for approval in 2019

7/2/19

Grifols SA, of Barcelona, Spain

Xembify (immune globulin subcutaneous, human- klhw)

20% subcutaneous immunoglobulin

Primary immunodeficiencies

Approved by FDA

7/4/19

Medicinova Inc., of La Jolla, Calif.

MN-166 (ibudilast)

Small-molecule macrophage migration inhibitory factor inhibitor and PDE410 inhibitor

Progressive multiple sclerosis

Received FDA feedback on plans for phase III to enroll subjects with secondary progressive MS without relapses; agency also agreed that primary endpoint should be time to 3-month confirmed disability progression, as measured by Expanded Disability Status Scale; FDA said single trial can be basis for marketing approval, depending on results

7/11/19

Orchard Therapeutics Ltd., of London

OTL-103

Ex vivo autologous hematopoietic stem cell-based gene therapy

Wiskott-Aldrich Syndrome

FDA issued Regenerative Medicine Advanced Therapy designation

7/29/19

Samsung Bioepis Co. Ltd., of Incheon, South Korea

Hadlima (adalimumab-bwwd)

Biosimilar of Humira, TNF-alpha antibody

Rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, adult Crohn's disease, ulcerative colitis, plaque psoriasis.

Approved by FDA

7/24/19

X4 Pharmaceuticals Inc., of Cambridge, Mass.

Mavorixafor (X4P-001)

Antagonist of CXCR4

Warts, hypogammaglobulinemia, infections and myelokathexis (WHIM) syndrome

European Commission granted orphan drug designation

7/30/19

Infection

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

Neutrolin

Catheter lock solution

Prevention of catheter-related bloodstream infection in hemodialysis patients

Received FDA feedback regarding statistical analyses requested for primary and secondary endpoints for Lock-It-100 study; based on feedback, company believes it has completed phase III development and does not need to conduct another trial at this time

7/9/19

Janseen Pharmaceutical Cos., unit of Johnson & Johnson, of New Brunswick, N.J., and Viiv Healthcare Ltd., of London

Rilpivirine long-acting (LA, Janssen) + cabotegravir LA (Viiv)

HIV-1 reverse transcriptase inhibitor; HIV integrase inhibitor

HIV-1 infection

Companies submitted marketing authorization application to EMA for once-monthly injectable 2-drug regimen

7/29/19

La Jolla Pharmaceutical Co., of San Diego

LJPC-0118 (artesunate)

Inhibits malaria proteins EXP1

Malaria

FDA granted orphan designation

7/23/19

Matinas Biopharma Holdings Inc., of Bedminster, N.J.

MAT-2203

Oral amphotericin B 

Cryptococcal meningitis

Received fast track and qualified infectious disease product status from the FDA

7/25/19

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

Recarbrio (imipenem, cilastatin and relebactam)

Antibiotic cocktail

Complicated urinary tract infections and complicated intra-abdominal infections

FDA approved drug

7/17/19

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

Zerbaxa (ceftolozane and tazobactam)

Cephalosporin antibiotic and inhibitor of beta-lactamases

Hospital-acquired pneumonia, including ventilator-associated pneumonia

CHMP adopted a positive opinion

7/29/19

Motif Bio plc, of New York

Iclaprim

Antibiotic

Acute bacterial skin and skin structure infections

Company has requested a type B meeting with the FDA to review the proposed study required for the resubmission of the NDA following a February 2019 complete response letter

7/15/19

Motif Bio plc, of New York

Iclaprim

Antibiotic

Acute bacterial skin and skin structure infection

Type B meeting with FDA slated for Sept. 19, 2019

7/26/19

Redhill Biopharma Ltd., of Tel Aviv, Israel

Talicia (RHB-105)

Fixed-dose, oral capsule combination of antibiotics rifabutin and amoxicillin plus proton pump inhibitor omeprazole

H. pylori infection

FDA accepted the NDA and granted priority review; PDUFA date set for Nov. 2, 2019

7/3/19

Scynexis Inc., of Jersey City, N.J.

Ibrexafungerp

Triterpenoid antifungal

Recurrent vulvovaginal candidiasis

Reached agreement with FDA under a special protocol assessment on design, trial population, endpoints and statistical analysis of the Candle pivotal study for the prevention of recurrent VVC

7/24/19

Theratechnologies Inc., of Montreal

Trogarzo (ibalizumab)

Recombinant humanized monoclonal antibody of immunoglobulin G isotype 4

Multidrug-resistant HIV-1

EMA's Committee for Medicinal Products for Human Use issued a positive opinion

7/26/19

Viiv Healthcare Ltd., of London

Dovato (dolutegravir/lamivudine)

Once-daily, single pill combining integrase strand transferase inhibitor and nucleoside analogue reverse transcriptase inhibitor

HIV-1 infection

European Commission granted marketing approval for use in adults and adolescents over 12 weighing at least 40 kg, with no known or suspected resistance to integrase inhibitor class or lamivudine

7/3/19

Inflammatory

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

Stelara (ustekinumab)

Blocks interleukin-12 and 23

Moderate to severe ulcerative colitis

EMA's Committee for Medicinal Products for Human Use issued a positive opinion

7/26/19

Musculoskeletal

Apic Bio Inc., of Cambridge, Mass.

APB-102

Gene therapy

Genetic SOD1 amyotrophic lateral sclerosis

FDA granted orphan designation

7/22/19

Ascendis Pharma A/S, of Copenhagen

Transcon CNP

Long-acting prodrug of C-type natriuretic peptide

Achondroplasia

Filed an IND with the FDA to initiate the Accomplish phase II trial in children

7/18/19

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

Nurtec (riluzole) (BHV-0223)

Sublingual tablet formulation

Amyotrophic lateral sclerosis

FDA issued a complete response letter for the 505(b)2 application, citing concerns about the active pharmaceutical ingredient that was manufactured by Apotex Pharmachem India Pvt Ltd. between 2014 to 2016 and used in the bioequivalence study in 2017

7/19/19

Neurology/Psychiatric

Acorda Therapeutics Inc., of Ardsley, N.Y.

Inbrija (levodopa inhalation powder)

Converted to dopamine in the body

"Off" episodes in Parkinson's disease

EMA's Committee for Medicinal Products for Human Use issued a positive opinion

7/26/19

Alkermes plc, of Dublin

ALKS-3831

Olanzapine and samidorphan in bilayer tablet

Bipolar I disorder and schizophrenia

Following a pre-NDA meeting with the FDA, company decided to file NDA for bipolar I disorder in addition to the treatment of schizophrenia in the fourth quarter of 2019 

7/15/19

Anavex Life Sciences Corp., of New York

ANAVEX-2-73

Sigma-1 receptor (S1R) agonist

Rett syndrome

EMA's Committee for Orphan Medicinal Products issued positive opinion on application for orphan designation in the indication

7/29/19

Cytokinetics Inc., of South San Francisco

Reldesemtiv

Fast skeletal muscle troponin activator

Spinal muscular atrophy

EMA granted orphan designation

7/23/19

Durect Corp., of Cupertino, Calif.

Posimir (bupivacaine extended-release)

Long-acting anesthetic

Postoperative pain

FDA accepted submission to the complete response letter as a class 2 response; PDUFA date of Dec. 27, 2019 

7/17/19

GW Pharmaceuticals plc, of London

Epidyolex (cannabidiol oral solution) 

Highly purified cannabidiol

Lennox-Gastaut syndrome or Dravet syndrome

EMA's Committee for Medicinal Products for Human Use issued a positive opinion for use as adjunctive therapy of seizures associated with LGS or Dravet syndrome, for patients 2 and older

7/26/19

Intra-Cellular Therapeutics Inc., of New York

Lumateperone tosylate capsules

Modulates serotonin, dopamine and glutamate

Schizophrenia

FDA disclosed on its website that the July 31, 2019, Psychopharmacologic Drugs Advisory Committee meeting is canceled; agency cites new information regarding the NDA and said it intends to continue evaluating application

7/23/19

Invagen Pharmaceuticals Inc., of New York, a subsidiary of Cipla Ltd., of Mumbai, India

Pregabalin 

Generic of Lyrica (Pfizer Inc.)

Neuropathic pain, postherpetic neuralgia, partial onset seizures and fibromyalgia

FDA gave final approval for the abbreviated NDA

7/22/19

Nektar Therapeutics Inc., of San Francisco

NKTR-181

Mu-opioid agonist

Chronic low back pain

FDA postponed Aug. 21m 2019, advisory committee meeting

7/26/19

Neuren Pharmaceuticals Ltd., of Melbourne, Australia

NNZ-2591

Diketopiperazine compound

Phelan-McDermid, Angelman and Pitt Hopkins syndromes

Submitted applications for orphan drug designation in the indications to the FDA

7/29/19

Neurocrine Biosciences Inc., of San Diego

Opicapone

Catechol-O-methyltransferase inhibitor

Parkinson's disease

FDA accepted NDA; PDUFA date of April 26, 2020

7/10/19

Neurovive Pharmaceutical AB, of Lund, Sweden

Neurostat

Cyclosporine A lipid emulsion

Traumatic brain injury

Candidate received FDA fast track designation

7/27/19

Ovid Therapeutics Inc., of New York

OV-101 (gaboxadol)

Delta-selective GABAA receptor agonist

Angelman syndrome

European Commission granted orphan designation

7/2/19

Prevail Therapeutics Inc., of New York

PR-001

Single-dose AAV9-based gene therapy

Parkinson's disease

FDA granted fast track for treating Parkinson's patients with GBA1 mutation

7/8/19

Sumitomo Dainippon Pharma Co. Ltd., of Osaka, Japan

Lurasidone hydrochloride

Atypical antipsychotic

Schizophrenia and bipolar depression

Submitted NDA for approval in Japan

7/31/19

Sunovion Pharmaceuticals Inc., Marlborough, Mass.

Dasotraline

Dopamine and norepinephrine reuptake inhibitor

Moderate-to-severe binge eating disorder

FDA accepted NDA; PDUFA date is May 14, 2020

7/30/19

Takeda Pharmaceuticals Co. Ltd., of Osaka, Japan

Vyvanse (lisdexamfetamine dimesylate)

CNS stimulant

Attention deficit hyperactivity disorder

Approved by Health Canada as chewable tablet for use in patients 6 and older

7/10/19

Vanda Pharmaceuticals Inc., of Washington

Hetlioz (tasimelteon)

Agonist of melatonin MT1 and MT2 receptors

Jet lag disorder

FDA indicated that the supplemental NDA has deficiencies that preclude discussion of labeling and postmarketing requirements/commitments at this time; PDUFA date is Aug. 16, 2019

7/22/19

Ocular

Alexion Pharmaceuticals Inc., of Boston

Soliris (eculizumab)

Inhibitor of C5 protein

Neuromyelitis optica spectrum disorder

EMA's Committee for Medicinal Products for Human Use issued a positive opinion for use in adult patients who are anti-aquaporin-4 antibody positive with a relapsing course of the disease

7/26/19

Allergan plc, of Dublin

Bimatoprost sustained-release

Biodegradable implant releasing bimatoprost

Primary open-angle glaucoma or ocular hypertension

FDA accepted NDA; agency expected to take action by the end of the first half of 2020

7/17/19

Eton Pharmaceuticals Inc., of Deer Park, Ill., and Bausch Health Companies Inc., of Laval, Quebec

EM-100

Topical ophthalmic solution

Ocular itching associated with allergic conjunctivitis

FDA issued a complete response letter that isn't related to the clinical data

7/12/19

Eyegate Pharmaceuticals Inc., of Waltham, Mass.

Ocular bandage gel

Eye drop comprising modified form of hyaluronic acid

Punctate epitheliopathies

Filed a protocol with FDA for a follow-on pilot study

7/9/19

Horizon Therapeutics plc, of Dublin

Teprotumumab

Monoclonal antibody targeting insulin-like growth factor 1 receptor

Active thyroid eye disease

Submitted BLA to FDA

7/10/19

Proqr Therapeutics NV, of Leiden, the Netherlands

Sepofarsen (QR-110)

CEP290 gene stimulator

Leber's congenital amaurosis 10

EMA granted Priority Medicines designation

7/29/19

Sosei Group Corp., of Tokyo, and Formosa Pharmaceuticals Inc., of Taipei, Taiwan

APP-13007

Nanoparticle formulation of corticosteroid clobetasol

Postoperative inflammation of the eye

FDA cleared the IND for first-in-human trial

7/18/19

Other/Miscellaneous

Aeglea Biotherapeutics Inc., of Austin, Texas

Pegzilarginase

Enhanced human arginase

Arginase-1 deficiency

Received FDA breakthrough therapy designation

7/25/19

Dicerna Pharmaceuticals Inc., of Cambridge, Mass.

DCR-A1AT

GalXC-based RNAi therapeutic

Alpha-1 antitrypsin deficiency-associated liver disease

Submitted clinical trial authorization application to Swedish Medical Products Agency for phase I/II study

7/2/19

Novartis AG, of Basel, Switzerland

Lucentis (ranibizumab)

Anti-VEGF therapy

Preterm infants with retinopathy of prematurity 

EMA's Committee for Medicinal Products for Human Use issued a positive opinion

7/26/19

Sensorion SA, of Montpellier, France

SENS-401

Arazasetron besylate

Sudden sensorineural hearing loss

FDA cleared IND for clinical testing

7/12/19

Respiratory

Aridis Pharmaceuticals Inc., of San Jose, Calif.

AR-501

Inhaled formulation of gallium citrate

Cystic fibrosis

EMA granted orphan designation for treating lung infection in CF patients

7/19/19

Astrazeneca plc, of Cambridge, U.K.

Fasenra (benralizumab)

Monoclonal antibody targeting IL-5 receptor alpha

Severe eosinophilic asthma

EMA's Committee for Medicinal Products for Human Use issued positive opinion to add self-administration option and new delivery method as prefilled, single-use, autoinjector to product information

7/1/19

Boehringer Ingelheim GmbH, of Ingelheim, Germany

Ofev (nintedanib)

Tyrosine kinase inhibitor

Systemic sclerosis associated interstitial lung disease

FDA's Arthritis Advisory Committee recommended (10-7 vote) to approve

7/25/19

Genkyotex SA, of Archamps, France

GKT-831

NOX1 and NOX4 inhibitor

Idiopathic pulmonary fibrosis

FDA approved the IND for the phase II investigator-initiated study

7/17/19

Teva Pharmaceuticals Industries Ltd., of Jerusalem

Airduo Digihaler

Fluticasone propionate and salmeterol in a digital inhaler

Asthma

FDA approved sNDA for the drug-device

7/15/19

Vertex Pharmaceuticals Inc., of Boston

VX-445 (elexacaftor), tezacaftor and ivacaftor

CFTR modulator/stimulator

Cystic fibrosis

Company submitted the NDA to the FDA with a request for a priority review

7/22/19

Toxicity and Intoxication

Acacia Pharma Group plc, of Cambridge, U.K.

Barhemsys (amisulpride injection)

Binds to dopamine D2/D3 receptors

Postoperative nausea and vomiting

Received official type A meeting minutes from FDA, during which company agreed with agency on a plan to resubmit the NDA, designating a new supplier of amisulpride, the active pharmaceutical ingredient

7/8/19

Insys Therapeutics Inc., of Phoenix

Naloxone nasal spray

Single-unit dose spray device delivering 8-mg naloxone dose

Opioid overdose

FDA accepted NDA seeking approval for use in emergency treatment of known or suspected opioid overdose in adult and pediatric patients

7/3/19


Notes

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

 

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