Company (location)

Product

Description

Indication

Status

Date

Cancer

Astellas Pharma Inc., of Tokyo, and Pfizer Inc., of New York

Xtandi (enzalutamide)

Androgen receptor inhibitor

Metastatic hormone-sensitive prostate cancer

FDA accepted for filing the supplemental NDA, granting priority review; PDUFA date expected in the fourth quarter of 2019

8/21/19

Astrazeneca plc, of Cambridge, U.K.

Calquence (acalabrutinib)

Bruton tyrosine kinase inhibitor

Chronic lymphocytic leukemia

FDA granted breakthrough therapy designation

8/14/19

Beigene Ltd., of Beijing

Zanubrutinib

BTK inhibitor

Mantle cell lymphoma

FDA accepted NDA for zanubrutinib for the treatment of patients with mantle cell lymphoma who have received at least one prior therapy; Priority review; PDUFA date: Feb. 27, 2020

8/21/19

Blueprint Medicines Corp., of Cambridge, Mass.

Avapritinib

Inhibits KIT and PDGFRA mutant kinases

Gastrointestinal stromal tumors

FDA accepted the NDA seeking approval in adults with PDGFRA exon 18 mutant GIST, regardless of prior therapy, and fourth-line GIST; FDA granted priority review and set PDUFA date of Feb. 14, 2020

8/7/19

Bristol-Myers Squibb Co., of Princeton, N.J. and Abbvie Inc., of North Chicago

Empliciti (elotuzumab)

SLAM family member 7 inhibitor; Lymphocyte function antigen-3 receptor modulator

Multiple myeloma

EC approved as third line therapy when taken with pomalidomide and low-dose dexamethasone for adults with r/r MM

8/27/19

Cstone Pharmaceuticals Co. Ltd., of Suzhou, China

CS-3002

Inhibits cyclin-dependent kinases 4 and 6

Advanced solid tumors

Study received ethics approval from the Human Research Ethics Committee in Australia and Australia's Therapeutic Goods Administration has acknowledged the electronic Clinical Trial Notification; plans to initiate the dose-escalation and dose-expansion study shortly

8/22/19

Daiichi Sankyo Co. Ltd., of Tokyo

Turalio (pexidartinib)

CSF1R inhibitor

Tenosynovial giant cell tumor

FDA approved for use in adults with symptomatic TGCT associated with severe morbidity or functional limitations and not responsive to improvement with surgery

8/2/19

G1 Therapeutics Inc., of Research Triangle Park, N.C.

Trilaciclib

Myelopreservation agent

Small-cell lung cancer

FDA granted breakthrough therapy designation

8/7/19

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

Rozlytrek (entrectinib)

Targets kinase activity of TRK type A/B/C and ROS1 proteins

ROS1-positive metastatic non-small-cell lung cancer

Approved by FDA for treating adults; also received accelerated approval for treating adult and pediatric patients, 12 and older, with solid tumors that have NTRK gene fusion

8/15/19

Genmab A/S, of Copenhagen, Denmark

Darzalex (daratumumab)

CD38-targeting human IgG1k mAb

Multiple myeloma

Approved in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma ineligible for autologous stem cell transplant in Japan

8/22/19

Glenmark Pharmaceuticals Ltd., of Mumbai

Fulvestrant

Generic of the estrogen receptor antagonist Faslodex (Astrazeneca plc)

Breast cancer

FDA granted final approval to the ANDA

8/23/19

Heat Biologics Inc., of Durham, N.C.

HS-130 and HS-110

Allogeneic cell line engineered to express extracellular domain of OX40 ligand fusion protein and gp96-Fc engineered to deliver 78 cancer antigens

Advanced solid tumors

FDA cleared the IND for a phase I trial of the combination in patients whose tumors are refractory to standard of care

8/12/19

Helix Biopharma Corp., of Richmond Hill, Ontario

LDOS-47

Single domain antibody conjugated therapeutic

Metastatic pancreatic cancer

FDA approved phase Ib/II study with doxorubicin

8/8/19

Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson

Imbruvica (ibrutinib)

BTK inhibitor

First-line chronic lymphocytic leukemia and Waldenström's macroglobulinemia

European Commission approved the expanded indications to include Imbruvica plus Gazyva (obinutuzumab, Roche Holding AG) in adult patients with previously untreated chronic lymphocytic leukemia and Imbruvica plus Rituxan (rituximab, Roche Holding AG and Biogen Inc.) in adults with Waldenström's macroglobulinemia

8/13/19

Morphosys AG, of Planegg, Germany

Tafasitamab (MOR-208)

CD19-targeting monoclonal antibody

Relapsed or refractory diffuse large B-cell lymphoma

Intends to submit MAA to EMA based on phase II L-Mind study in combination with lenalidomide; letter of intent submitted to EMA in July 2019 and MAA submission expected to complete by mid-2020

8/6/19

Mustang Bio Inc., of New York

MB-102

CD123 CAR T-cell therapy

Acute myeloid leukemia, Blastic plasmacytoid dendritic cell neoplasm and high-risk myelodysplastic syndrome

FDA cleared the IND for a phase I/II trial

8/5/19

Nektar Therapeutics Inc., of San Francisco, and Bristol-Myers Squibb Co., of New York

Bempegaldesleukin (NKTR-214) and Opdivo (nivolumab)

CD122-preferential IL-2 pathway agonist and anti-PD-1 antibody

Metastatic melanoma

FDA granted breakthrough therapy designation for the combination in previously untreated unresectable or metastatic disease

8/1/19

Oncoceutics Inc., of Philadelphia

ONC-206

Targets a G protein-coupled receptor

Primary central nervous system neoplasms

FDA accepted the IND application for a first-in-human trial in adults with primary CNS neoplasms.

8/26/19

Oncology Venture A/S, of Hoersholm, Denmark

Liplacis

Liposomal formulation of cisplatin

Metastatic breast cancer

FDA approved an investigational device exemption for use of drug response predictor Liplacis DRP in planned pivotal phase III study; evaluation of IND is ongoing

8/15/19

Pharmamar SA, of Madrid, Spain

Lurbinectedin

RNA polymerase II inhibitor and TAT protein inhibitor

Small-cell lung cancer

Company plans to file NDA with FDA in fourth quarter, using accelerated approval pathway following discussion with agency, seeking approval for second-line treatment

8/19/19

Roche Holding AG, of Basel, Switzerland

Tecentriq (atezolizumab)

Monoclonal antibody targeting PD-L1

First-line extensive-stage small-cell lung cancer

Health Canada approved use of the drug in combination with carboplatin and etoposide

8/13/19

Roche Holding AG, of Basel, Switzerland

Tecentriq (atezolizumab)

Monoclonal antibody targeting PD-L1

Unresectable locally advanced or metastatic triple-negative breast cancer

EC approved the drug in combination with Abraxane (nab-paclitaxel, Celgene Corp.)

8/29/19

SFA Therapeutics Inc., of Philadelphia

SFA-001

Microbiome-based therapy

Hepatocellular carcinoma

Filed application seeking FDA orphan designation

8/5/19

Springworks Therapeutics Inc., of Stamford, Conn.

Nirogacestat

Gamma-secretase inhibitor

Progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis

FDA granted breakthrough designation

8/29/19

Telix Pharmaceuticals Ltd., of Melbourne, Australia

TLX-250

89Zr-radiolabeled version of girentuximab

Renal tumor

Completed a clinical trial notification submission to Japan's PMDA, allowing start of phase I/II trial designed to bridge its international ZIRCON phase III study

8/29/19

Ziopharm Oncology Inc., of Boston

Ad-RTS-hIL-12 plus veledimex

Gene therapy to induce and control interleukin-12

Glioma

EMA Committee for Orphan Medicinal Products adopted a positive opinion recommending orphan designation

8/8/19

Cardiovascular

Akari Therapeutics plc, of London

Nomacopan

C5 complement inhibitor and leukotriene BLT receptor antagonist

Pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy

FDA granted fast track designation

8/14/19

Akari Therapeutics plc, of London

Nomacopan

Leukotriene BLT receptor antagonist; Complement C5 factor inhibitor

Thrombotic microangiopathy

FDA granted orphan status for treatment of hematopoietic stem cell transplantation-associated thrombotic microangiopathy

8/30/19

La Jolla Pharmaceutical Co., of San Diego

Giapreza (angiotensin II)

Vasoconstrictor

Refractory hypotension

EC approved the drug

8/29/19

Mesoblast Ltd., of New York

Revascor

Allogeneic mesenchymal precursor cells

End-stage heart failure patients implanted with a left ventricular assist device

FDA agreed to a confirmatory phase III study with a primary endpoint of reduction in major mucosal bleeding events and key secondary endpoints demonstrating improvement in various parameters of cardiovascular function

8/27/19

Milestone Pharmaceuticals Inc., of Montreal

Etripamil

Short-acting calcium channel blocker

Paroxysmal supraventricular tachycardia

Following interactions with the FDA, the company plans to start enrollment of its Node-303 open-label study before the end of the year; the study will enroll older patients and patients taking concomitant beta-blockers and calcium channel blockers and won't require an in-office safety test dose

8/13/19

Phasebio Pharmaceuticals Inc., of Malvern, Pa.

PB-2452

Recombinant human monoclonal antibody antigen-binding fragment

Bleeding

Minutes from end-of-phase I meeting with FDA said to align with company on design and execution of single, non-randomized phase III trial to support BLA submission

8/14/19

Windtree Therapeutics Inc., of Warrington, Pa.

Istaroxime

Na+ K+ ATPase inhibitor

Acute heart failure

FDA granted fast track designation

8/13/19

Zensun USA Inc., of San Diego

Neucardin

Recombinant human neuregulin-1 fragment

Chronic heart failure

FDA granted fast tract designation; National Medical Products Administration in China granted a priority review for the conditional approval application

8/28/19

Dermatologic

Cassiopea SpA, of Lainate, Italy

Clascoterone cream 1%

Topical androgen receptor inhibitor

Acne

Submitted NDA to FDA

8/20/19

Foamix Pharmaceuticals Ltd., of Rehovot, Israel

FMX-103

Minocycline foam, 1.5%

Moderate to severe papulopustular rosacea

Submitted NDA to FDA for use in patients 18 and older

8/5/19

Glenmark Pharmaceuticals Ltd., of Mumbai, India

Pimecrolimus

Inflammatory cytokine release inhibitor

Skin conditions such as eczema

FDA approved its ANDA for generic of Bausch Health U.S. LLC's Elidel cream

8/30/19

Leo Pharma A/S, of Ballerup, Denmark, and JW Pharmaceutical Corp., of Seoul, South Korea

JW-1601/LP-0190

Histamine H4 receptor inverse agonist

Atopic dermatitis

Filed an IND with the FDA for a pharmacokinetics study scheduled to start in 2020

8/27/19

Leo Pharma Inc., of Madison, N.J.

Enstilar (calcipotriene and betamethasone dipropionate) foam

Combination of vitamin D analogue and corticosteroid

Plaque psoriasis

FDA expanded approved indication to include patients 12 and older; agency granted pediatric exclusivity, extending U.S. market exclusivity by an additional 6 months to Dec. 10, 2031

8/1/19

Ortho Dermatologics, part of Bausch Health Cos. Inc., of Laval, Quebec

IDP-123 (tazarotene 0.045%) lotion

Retinoic acid receptor-specific retinoid

Acne vulgaris

FDA accepted the NDA and set PDUFA date of Dec. 22, 2019

8/7/19

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

Dupixent (dupilumab)

IL-4/IL-13-targeting monoclonal antibody

Moderate to severe atopic dermatitis

European Commission extended marketing authorization to include adolescents, ages 12 to 17, who are candidates for systemic therapy

8/6/19

Endocrine/Metabolic

Alnylam Pharmaceuticals Inc., of Cambridge, Mass.

Givosiran

RNAi therapeutic targeting aminolevulinic acid synthase 1

Acute hepatic porphyria

FDA accepted the NDA and granted priority review; PDUFA date set for Feb. 4, 2020

8/6/19

Amarin Corp. plc, of Dublin

Vascepa (icosapent ethyl)

Omega-3 fatty acid

Severe hypertriglyceridemia

Received notice from FDA that an advisory committee is tentatively set for Nov. 14, 2019, in connection to review of pending supplemental NDA for expanding labeling based on Reduce-IT cardiovascular outcomes study; company now does not expect agency to take action on the sNDA by previously announced PDUFA date of Sept. 28, 2019

8/8/19

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

Galafold (migalastat)

Oral pharmaceutical chaperone

Fabry disease

Agency of Medicines, Food and Medical Devices in Argentina granted approval

8/2/19

Astrazeneca plc, of Cambridge, U.K.

Forxiga (dapagliflozin)

Oral SGLT2 inhibitor

Type 2 diabetes

European Commission approved update to marketing authorization to include positive cardiovascular outcomes and renal data from phase III Declare-TIMI 58 trial in adults

8/5/19

Cerecor Inc., of Rockville, Md.

CERC-802

Ultra-pure formulation of D-mannose

Mannose-phosphate isomerase deficiency

FDA granted fast track designation

8/21/19

Clarus Therapeutics Inc., of Northbrook, Ill.

Jatenzo (testosterone undecanoate)

Androgen receptor agonist

Hypogonadism

FDA granted 3-year market exclusivity

8/19/19

Evoke Pharma Inc., of Solana Beach, Calif.

Gimoti

Metoclopramide nasal spray

Diabetic gastroparesis

NDA to be resubmitted in the fourth quarter of 2019

8/8/19

Glenmark Pharmaceuticals Ltd., of Mumbai

Remo-M/Remozen-M (remogliflozin etabonate + metformin hydrochloride)

Sodium glucose co-transporter-2 + AMP activated protein kinase stimulator

Type 2 diabetes

Combination therapy approved in India to treat adults

8/19/19

Mundipharma International Corp. Ltd.

Invokana (canagliflozin)

SGLT-2 inhibitor

Non-insulin dependent diabetes

EMA has accepted license extension submission to treat type 2 diabetes patients with chronic kidney disease

8/21/19

Mundipharma International Corp. Ltd.

Vokanamet (canagliflozin and metformin)

Fixed dose SGLT-2 inhibitor + insulin sensitizer

Non-insulin dependent diabetes

EMA has accepted license extension submission to treat type 2 diabetes patients with chronic kidney disease

8/21/19

Oxthera AB, of Stockholm

Oxabact 

Oxalobacter formigenes oxalate-degrading bacterium

Primary hyperoxaluria

EMA approved the pediatric investigational plan

8/23/19

Provention Bio Inc., of Oldwick, N.J.

Teplizumab (PRV-031)

Anti-CD3 monoclonal antibody

Prevention of delay of clinical type 1 diabetes

FDA granted breakthrough therapy designation for use in individuals at risk of developing the disease

8/5/19

Seelos Therapeutics Inc., of New York

SLS-005 (trehalose)

Low molecular weight disaccharide

Sanfilippo syndrome

Received FDA clearance to start phase IIb/III study to assess safety, tolerability and efficacy of trehalose IV in Sanfilippo A and B

8/22/19

Ultragenyx Pharmaceuticals Inc., of Novato, Calif.

UX-007 (triheptanoin)

Highly purified, pharmaceutical-grade, medium-chain triglyceride consisting of 3 7-carbon fatty acids on a glycerol backbone

Long-chain fatty acid oxidation disorders

Submitted NDA to FDA

8/1/19

Gastrointestinal

Actobio Therapeutics Inc., of Ghent, Belgium, wholly owned subsidiary of Intrexon Corp., of Germantown, Md.

AG-017

Oral Actobiotic consisting of genetically engineered Lactococcus lactis

Celiac disease

FDA approved IND application for phase Ib/IIa study in the U.S. and Europe, expected to begin enrollment this year

8/19/19

Ascletis Pharma Inc., of Hangzhou, China and Sagimet Biosciences, of San Mateo, Calif.

ASC-40 / TVB-2640

Fatty acid synthase inhibitor

Non-alcoholic steatohepatitis

Received IND approval from the NMPA following April start of U.S. phase II NASH trial

8/27/19

Astrazeneca plc, of Cambridge, U.K.

Fasenra (benralizumab)

IL-5 receptor antagonist

Eosinophilic esophagitis

FDA granted orphan status

8/28/19

Galectin Therapeutics Inc., of Norcross, Ga.

Belapectin (GR-MD-02)

Complex carbohydrate drug targeting galectin-3

Compensated nonalcoholic steatohepatitis cirrhosis without esophageal varices

Submitted for assessment phase III protocol via type C written response only submission to FDA, with goal of finalizing protocol and starting trial in fourth quarter of 2019

8/5/19

US Worldmeds LLC, of Louisville, Ky.

Myobloc

Rimabotu-linumtoxin B injection

Chronic sialorrhea in adults

FDA approved the sBLA

8/26/19

Genitourinary/Sexual Health

Abbvie Inc., of North Chicago

Elagolix

Oral gonadotropin-releasing hormone antagonist

Uterine fibroids

Submitted NDA to FDA seeking approval for management of heavy menstrual bleeding associated with uterine fibroids

8/5/19

Astrazeneca plc, of Cambridge, U.K.

Farxiga (dapagliflozin)

SGLT2 inhibitor

Chronic kidney disease

FDA granted fast track designation

8/27/19

Rockwell Medical Inc., of Wixom, Mich.

Triferic

Intravenous iron product

End-stage renal disease and chronic kidney disease

FDA accepted the NDA for the I.V. formulation to replace iron and maintain hemoglobin in hemodialysis patients via dialysate during each dialysis treatment; PDUFA date is March 28, 2020

8/6/19

Hematologic

Astrazeneca plc, of Cambridge, U.K. and Fibrogen Medical Technology Development Co. Ltd., of Beijing

Roxadustat

Hypoxia-inducible factor prolyl hydroxylase inhibitor

Anemia

NMPA granted marketing authorization for roxadustat in China for the treatment of anemia caused by chronic kidney disease in non-dialysis-dependent patients

8/22/19

Celgene Corp., of Summit, N.J.

Inrebic (fedratinib)

JAK2 inhibitor

Myelofibrosis

Approved by FDA for use in adults with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) disease

8/16/19

Rocket Pharmaceuticals Inc., of New York

RP-L102

FANCA gene stimulator

Fanconi anemia

Spanish Agency for Medicines and Health Products cleared it to start enrollment in FANCOLEN-II phase II registration-enabling study; primary endpoint is the emergence of mitomycin-C resistance in bone marrow colony forming cells

8/22/19

Sigilon Therapeutics Inc., of Cambridge, Mass.

SIG-001

Implantable cells producing factor VIII

Hemophilia A

FDA granted orphan drug designation

8/27/19

Immune

Abbvie Inc., of North Chicago

Rinvoq (upadacitinib)

Oral JAK inhibitor

Moderate to severe rheumatoid arthritis

Approved by FDA for use in adults who have inadequate response or intolerance to methotrexate

8/16/19

Mustang Bio Inc., of New York and St. Jude Children's Research Hospital, of Memphis, Tenn.

MB-107

Lentiviral gene therapy

Severe combined immunodeficiency syndrome

Granted RMAT designation by the FDA

8/22/19

Infection

Abbvie Inc., of North Chicago

Maviret (glecaprevir/pibrentasvir)

Combination of 2 direct-acting antivirals

Hepatitis C virus

European Commission granted marketing authorization to shorten once-daily treatment duration from 12 to 8 weeks in treatment-naïve, compensated cirrhotic, chronic HCV patients with genotype 1, 2, 4, 5 and 6 infection

8/2/19

Ascletis Pharma Inc., of Hangzhou, China

ASC-18

Single-pill, once-daily fixed-dose antiviral combination

Hepatitis C virus

Received IND approval from China's NMPA

8/4/19

Dare Bioscience Inc., of San Diego

DARE-BV1

Thermosetting hydrogel containing clindamycin phosphate 2%

Bacterial vaginosis

FDA granted qualified infectious disease product designation

8/12/19

Eiger Biopharmaceuticals Inc., of Palo Alto, Calif.

Lambda (peginterferon lambda) 

Type III interferon

Hepatitis delta virus

FDA granted breakthrough therapy status

8/20/19

Gilead Sciences Inc., of Foster City, Calif.

Descovy

Emtricitabine 200-mg and tenofovir alafenamide 25-mg tablets

Reduction of risk of sexually acquired HIV infection in men and transgender women who have sex with men

FDA Antimicrobial Drugs Advisory Committee recommended approval 16-2

8/8/19

Gilead Sciences Inc., of Foster City, Calif.

Biktarvy (bictegravir 50mg/emtricitabine 200mg/tenofovir alafenamide 25mg)

Single-tablet combination antiviral

HIV-1

China's National Medical Products Administration approved the once-daily regimen

8/9/19

Inotrem SA, of Paris

Nangibotide (LR-12)

Targets TREM-1 pathway

Septic shock

FDA cleared IND for phase IIb Astonish trial

8/6/19

Nabriva Therapeutics plc, of Dublin

Contepo (fosfomycin)

Antibiotic

Complicated urinary tract infections, including acute pyelonephritis 

After reviewing notes from FDA meeting, company plans to submit for approval early in the fourth quarter of this year

8/16/19

Nabriva Therapeutics plc, of Dublin

Xenleta (lefamulin)

Pleuromutilin antibacterial

Community-acquired bacterial pneumonia

FDA granted marketing approval

8/19/19

Novavax Inc., of Gaithersburg, Md.

Nanoflu

Adjuvanted recombination quadrivalent seasonal influenza vaccine

Influenza

Received input from FDA on end-of-phase II questions and reached agreement on phase III design for testing in adults, 65 and older; study set to start in fall 2019, with top-line data in first quarter of 2020

8/5/19

Recce Pharmaceuticals Ltd., of Sydney

RECCE-327

Antibiotic

Blood infections and sepsis from E. Coli and S. aureus

FDA gave positive feedback on the chemistry, manufacturing and controls data pack

8/26/19

TB Alliance, of New York

Pretomanid

Nitroimidazole antibacterial

Extensively drug-resistant tuberculosis and treatment intolerant/non-responsive multidrug-resistant TB

Approved by FDA for use in regimen containing bedaquiline and linezolid

8/14/19

Inflammatory

Gilead Sciences Inc., of Foster City, Calif.

Filgotinib

Oral, selective JAK1 inhibitor

Rheumatoid arthritis

Marketing application has been validated and is under review by the EMA

8/16/19

Musculoskeletal

Discgenics Inc., of Salt Lake City

IDCT

Homologous, allogeneic discogenic cells

Pain and disability associated with degenerative disc disease

FDA granted fast track designation

8/26/19

Eli Lilly and Co., of Indianapolis

Taltz (ixekizumab)

Monoclonal antibody targeting interleukin 17A

Active ankylosing spondylitis

FDA approved the additional indication

8/26/19

Sarepta Therapeutics Inc., of Cambridge, Mass.

Golodirsen

Phosphoro-diamidate morpholino oligomer that binds to dystrophin mRNA and targets and excludes exon 53 during mRNA processing

Duchenne muscular dystrophy in patients with a confirmed mutation amenable to exon 53 skipping

FDA issued complete response letter

8/19/19

Neurology/Psychiatric

Alexion Pharmaceuticals Inc., of Boston

Soliris (eculizumab)

Complement inhibitor

Neuromyelitis optica spectrum disorder

European Commission approved the extension of the marketing authorization to include the new indication

8/27/19

Dynacure SAS, of Strasbourg, France

DYN-101

Antisense drug that modulates expression of dynamin 2

Centronuclear myopathies

FDA granted orphan designation

8/8/19

Emalex Biosciences Inc., of Chicago

Ecopipam

Dopamine D1 and receptor antagonist

Tourette syndrome

FDA granted fast track designation

8/28/19

Eton Pharmaceuticals Inc., of Deer Park, Ill.

ET-105 (lamotrigine)

Anti-epileptic

Lennox-Gastaut syndrome

FDA accepts NDA seeking approval as adjunct therapy for partial seizures, primary generalized tonic-clonic seizures and generalized seizures in patients, 2 and older; agency set PDUFA date of March 17, 2020

8/1/19

Harmony Biosciences LLC, of Plymouth Meeting, Pa.

Wakix (pitolisant)

Selective histamine 3 receptor antagonist/inverse agonist

Excessive daytime sleepiness in narcolepsy

Approved by FDA for use in adults

8/15/19

Intra-Cellular Therapies Inc., of New York

Lumateperone

Modulates serotonin, dopamine and glutamate

Schizophrenia

Agreed with FDA to submit additional nonclinical information in connection with the agency's ongoing review of the NDA; FDA said submission constitutes a major amendment to the NDA, resulting in 3-month extension of PDUFA date to Dec. 27, 2019

8/5/19

Kyowa Kirin Co. Ltd., of Tokyo

Nourianz (istradefylline)

Adenosine A2a receptor antagonist

Parkinson's disease

FDA approved as adjunctive treatment to levodopa/carbidopa

8/27/19

Luye Pharma Group Ltd., of Tokyo

LY-03005 (ansofaxine hydrochloride)

Extended release formulation of serotonin-norepinephrine-dopamine triple reuptake inhibitor

Depression

CTA filed in Japan; phase III trials in China and U.S. pivotal study both underway

8/22/19

Medincell SA, of Montpellier, France

mdc-TJK

Long-acting injectable

Antipsychotic

Received FDA IND clearance to initiate clinical activities; a first-in-human study is expected to start 2H19

8/26/19

Neuropore Therapies Inc., of San Diego

NPT-52034

PI3K-AKT-mTor inhibitor

Amyotrophic lateral sclerosis

FDA granted orphan drug designation

8/14/19

Shinkei Therapeutics LLC, of Princeton, N.J.

MR-201

NMDA-antagonist

Emotional lability

Filed IND to test novel formulation for treatment of pseudobulbar affect

8/21/19

Shinkei Therapeutics LLC, of Princeton, N.J.

MR-301

NMDA-antagonist

Traumatic brain injury

FDA accepted filing of IND; healthy volunteer dosing expected to start in next 30 days

8/28/19

Stalicla SA, of Geneva

STP1

2 undisclosed repurposed drugs

Autism spectrum disorder

Completed pre-IND meeting with the FDA; plans to start a phase Ib study in early 2020

8/22/19

Stoke Therapeutics Inc., of Bedford, Mass.

STK-001

Antisense oligonucleotide

Dravet syndrome

FDA granted orphan designation

8/6/19

Trevena Inc., of Chesterbrook, Pa.

Oliceridine

Opioid

Moderate to severe acute pain

Company completed nonclinical work to characterize the 9662 inactive metabolite and finished the drug product validation reports that are required for an NDA resubmission in the first quarter of 2020

8/28/19

Vanda Pharmaceuticals Inc., of Washington

Hetlioz (tasimelteon)

Dual melatonin MT1/MT2 receptor agonist

Jet lag disorder

FDA issued complete response letter to sNDA, citing unclear clinical significance of findings that patients reported sleeping nearly 3 hours longer over 3 nights following transatlantic trip when treated than they did over 3 nights following untreated trip

8/19/19

Viela Bio Inc., of Gaithersburg, Md.

Inebilizumab

Anti-CD19 monoclonal antibody

Neuromyelitis optica spectrum disorder

FDA accepted the BLA

8/27/19

Zosano Pharma Corp., of Fremont, Calif.

C-213 (zolmitriptan)

Dual 5-HT 1b/1d receptor agonist, delivered via microneedle patch

Cluster headache

Company filed IND with FDA to initiate phase II/III study

8/14/19

Ocular

Alimera Sciences Inc., of Atlanta

Iluvien (fluocinolone acetonide intravitreal implant)

Corticosteroid

Diabetic macular edema

Approved by Australian Therapeutic Good Administration for use in patients previously treated with a course of corticosteroids who did not have clinically significant rise in intraocular pressure

8/5/19

Clearside Biomedical Inc., of Alpharetta, Ga.

Xipere (triamcinolone acetonide)

Suprachoroidal suspension of corticosteroid

Macular edema

FDA requested stability data for company's manufacturing process; Expecting CRL on or before Oct. 19 PDUFA date; Plans to re-submit NDA in 2020 Q1 with requested stability data

8/22/19

Kala Pharmaceuticals Inc., of Watertown, Mass.

KPI-121 0.25%

Corticosteroid agonist

Dry eye disease

Received FDA complete response letter for NDA seeking approval for use in temporary relief in signs and symptoms of dry eye; FDA indicated efficacy data from additional trial will be needed to support resubmission; Kala is targeting resubmission during first half of 2020

8/8/19

Proqr Therapeutics NV, of Leiden, the Netherlands

QR-1123

Antisense targeting rhodopsin mRNA

Autosomal dominant retinitis pigmentosa

FDA cleared IND for study of up to 12 patients measuring the restoration or improvement of visual function and retinal structure by visual acuity, visual field and optical coherence tomography

8/12/19

Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y.

Eylea (aflibercept)

VEGFR inhibitor

Neovascular age-related macular degeneration, macular edema following retinal vein occlusion, diabetic macular edema and diabetic retinopathy

FDA approved a chemistry, manufacturing and controls prior-approval supplement to include single-use prefilled syringes

8/13/19

Other/Miscellaneous

DBV Technologies SA, of Montrouge, France

Viaskin Peanut

Allergen desensitizer

Peanut allergy

BLA submitted

8/8/19

Vertex Pharmaceuticals Inc., of Boston

VX-445 (elexacaftor), tezacaftor and ivacaftor (Kalydeco, Vertex Pharmaceuticals Inc.) triple-combination regimen

CFTR correctors

Cystic fibrosis

FDA accepted NDA

8/20/19

Viralgen Vector Core S.L., of San Sebastián, Spain

Various adeno-associated virus gene therapies

Various

Various

New facility received current good manufacturing practices compliance accreditation and pharmaceutical laboratory authorization by the AEMPS (Spanish Agency for Medicines and Health Products) and certification from the EMA

8/27/19

Vivus Inc., of Campbell, Calif.

Qsymia (phentermine and topiramate extended release)

Anti-obesity compound

Weight loss

Approved by South Korea Ministry of Food and Drug Safety; marketed in South Korea by partner Alvogen Korea Ltd.

8/5/19

Toxicity and Intoxication

Chrysalis Biotherapeutics Inc., of Galveston, Texas

TP-508 (rusalatide acetate)

Regenerative peptide

Radiation sickness

FDA and EMA granted orphan status for the treatment of acute radiation syndrome

8/21/19


Notes

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

 

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