Cancer

Abbvie Inc., of North Chicago

Venclexta (venetoclax)

Oral Bcl-2 inhibitor

Newly diagnosed acute myeloid leukemia

Submitted a supplemental NDA for use in combination with a hypomethylating agent or in combination with low-dose cytarabine in patients who are ineligible for intensive chemotherapy

7/12/18

Advaxis Inc., of Princeton, N.J.

Axalimogene filolisbac

Vaccine comprising Listeria monocytogenes

Metastatic cervical cancer

Plans to withdraw its conditional MAA in the EU for use in patients who progress beyond first-line therapy; decision is based on EMA feedback following initial review, indicating the application will likely need additional data

7/10/18

Advaxis Inc., of Princeton, N.J.

ADXS-HOT

Immunotherapy

Non-small-cell lung cancer

FDA cleared the IND for a phase I/II trial; first patient to be dosed by the end of 2018

7/30/18

Agios Pharmaceuticals Inc. (Cambridge, Mass.)

Tibsovo

Ivosidenib; formerly AG-120; oral

Relapsed or refractory acute myeloid leukemia with a susceptible isocitrate dehydrogenase-1 mutation

FDA granted approval for use with the Abbott Realtime IDH1 test; Tibsovo had priority review, as well as fast-track and orphan drug designations

7/23/18

Array Biopharma Inc., of Boulder, Colo.

Braftovi (encorafenib) plus Mektovi (binimetinib)

Oral small-molecule BRAF kinase inhibitor and oral small-molecule MEK inhibitor

Unresectable or metastatic melanoma with a BRAF mutation

EMA's CHMP issued a positive recommendation for use of the drugs in adults; final EC decision expected by the end of September

7/27/18

Astrazeneca plc, of Cambridge, Mass.

Tagrisso (osimertinib)

Third-generation, irreversible EGFR tyrosine kinase inhibitor

First-line locally advanced or metastatic non-small-cell lung cancer

Approved by Health Canada for patients whose tumors have EGFR exon 19 deletions or exon 21 (L858R) substitution mutations (either alone or in combination with other EGFR mutations)

7/12/18

Astrazeneca plc, of Cambridge, U.K.

Imfinzi (durvalumab)

Anti-PD-L1 antibody

Locally advanced, unresectable non-small-cell lung cancer

EMA's CHMP recommended approval for use in adults whose tumors express PD-L1 on ≥1% of tumor cells and whose disease has not progressed following platinum-based chemotherapy and radiation therapy

7/27/18

Astrazeneca plc, of Dublin 

Imfinzi (durvalumab) 

PD-L1-targeting antibody 

Non-small-cell lung cancer

Approved by Japan's Ministry of Health, Labour and Welfare for use as a maintenance therapy after definitive chemoradiation therapy in patients with locally advanced (stage III), unresectable disease

7/2/18

Astrazeneca plc, of Dublin, and Merck & Co. Inc., of Kenilworth, N.J. 

Lynparza (olaparib) 

PARP inhibitor 

Breast cancer 

Approved by Japan's PMDA for use in patients with unresectable or recurrent BRCA-mutated, HER2-negative disease who have received prior chemotherapy; patients are selected for therapy based on an approved companion diagnostic

7/2/18

Beigene Ltd., of Beijing

Zanubrutinib

BTK inhibitor

Waldenström macroglobulinemia

FDA granted fast track designation

7/22/18

Bristol-Myers Squibb Co., of New York

Opdivo (nivolumab)

Anti-PD-1 antibody

Melanoma with involvement of lymph nodes or metastatic disease in patients who have undergone complete resection

European Commission granted marketing authorization

7/31/18

Bristol-Myers Squibb Co., of New York

Opdivo (nivolumab)/Yervoy (ipilimumab)

Anti-PD-1 antibody/anti-CTLA-4 antibody

Intermediate/poor-risk advanced or metastatic renal cell carcinoma

Combination therapy was approved by Health Canada for use in adults

7/9/18

Bristol-Myers Squibb Co., of Princeton

Opdivo (nivolumab)/Yervoy (ipilimumab)

Anti-PD-1 antibody/Anti-CTLA-4 antibody

Microsatellite instability high or mismatch repair deficient metastatic colorectal cancer

Approved by FDA for use in adult and pediatric patients whose disease has progressed following treatment with a fluoropyrimidine, oxaliplatin and irinotecan; approval was granted under the accelerated pathway based on overall response rate and duration of response

7/11/18

Bristol-Myers Squibb Co., of Princeton, N.J.

Sprycel

Dasatinib

Chronic myeloid leukemia

EC expanded the indication to include treatment of children and adolescents ages 1 to 18 with Philadelphia chromosome-positive CML in chronic phase, and to include a powder for oral suspension formulation

7/6/18

Cellectar Biosciences Inc., of Madison, Wis.

CLR-131

Phospholipid drug conjugate

Ewing's sarcoma

FDA granted orphan designation

7/9/18

Celyad SA, of Mont-Saint-Guibert, Belgium

CYAD-101

Allogeneic CAR T candidate encoding CYAD-01 CAR T and peptide TCR inhibiting molecule

Unresectable colorectal cancer

FDA accepted the IND for the Allo-SHRINK trial testing safety and clinical activity in combination with standard chemotherapy

7/24/18

Clovis Oncology Inc., of Boulder, Colo.

Rubraca (rucaparib)

PARP inhibitor

Epithelial ovarian, fallopian tube or primary peritoneal cancer

EMA validated the application for a type II variation for the marketing authorization to include maintenance treatment of adult patients who are in complete or partial response to platinum-based chemotherapy

7/5/18

Clovis Oncology Inc., of Boulder, Colo.

Rubraca

Rucaparib

Epithelial ovarian, fallopian tube, or primary peritoneal cancer

EMA validated the application for a type II variation to the marketing authorization for Rubraca (rucaparib) to include maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy

7/5/18

Cstone Pharmaceuticals (Suzhou) Co. Ltd., of Suzhou, China

CS-1003

Anti-PD-1 monoclonal antibody

Advanced cancers

China's CNDA approved the first clinical trial application

7/12/18

Cstone Pharmaceuticals Co. Ltd., of Suzhou, China

CS-3006

Small-molecule MEK inhibitor

Cancer

IND cleared by the CNDA in China

7/30/18

Eagle Pharmaceuticals Inc., of Woodcliff Lake, N.J.

Bendeka

Bendamustine hydrochloride injection

Chronic lymphocytic leukemia

FDA granted 7 years of orphan drug exclusivity

7/9/18

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

Lenvima (lenvatinib)

VEGFR1, VEGFR2 and VEGFR3 kinase inhibitor

Second-line or later advanced and/or metastatic non-microsatellite instability high/proficient mismatch repair endometrial carcinoma

FDA granted breakthrough designation for use with Keytruda (pembrolizumab)

7/31/18

Eusa Pharma Ltd., of Hemel Hempstead, U.K.

Qarziba (dinutuximab beta)

Immunotherapy

High-risk neuroblastoma

Recommended by the U.K.'s National Institute for Health and Care Excellence for use in children in England and Wales

7/12/18

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

Tecentriq (atezolizumab)

PD-L1-inhibiting antibody

Urothelial cancer

FDA posted its June 19 action in which it limited the drug's use to patients with locally advanced or metastatic disease who are not eligible for cisplatin-containing therapy; decision was based on data showing decreased survival with Tecentriq as a monotherapy vs. platinum-based chemotherapy in patients who have not received prior therapy and have low expression of PD-L1

7/3/18

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

Darzalex (daratumumab)

Biologic targeting CD38

Multiple myeloma

EMA's CHMP recommended broadening the use for front-line therapy, in combination with bortezomib, melphalan and prednisone, in adults with newly diagnosed disease ineligible for autologous stem cell transplant

7/27/18

Immuno-medics Inc., of Morris Plains, N.J.

Sacituzumab govitecan

Antibody-drug conjugate targeting TROP-2

Metastatic triple-negative breast cancer

FDA accepted the BLA and granted a priority review with a target action date of Jan. 18, 2019

7/18/18

Immutep Ltd., of Sydney

Eftilagimod alpha

LAG-3Ig fusion protein

Non-small-cell lung carcinoma or head and neck carcinoma

FDA approved IND for TACTI-002 study testing the drug in combination with Keytruda (pembrolizumab, Merck & Co. Inc.)

7/30/18

Innovent Biologics Inc., of Suzhou, China

IBI-101

Anti-OX40 agonistic antibody

Cancer and hepatitis B

Received IND approval from Chinese regulators

7/26/18

JHL Biotech Co. Ltd., of Hsinchu, Taiwan

JHL-1101

Rituximab biosimilar

Diffuse large B-cell lymphoma

Received scientific advice from the EMA's CHMP related to the EU approval pathway; the agency confirmed it agrees with JHL's development approach, clinical development proposal, and study design of the global phase III study

7/9/18

JHL Biotech Inc., of Hsinchu, Taiwan

JHL-1101

Rituximab biosimilar

Non-Hodgkin lymphoma

The Center for Drug Evaluation, State Drug Administration of China approved JHL's phase I and phase III clinical trial application; it will begin trials "in the following months"

7/6/18

Medical Prognosis Institute, of Hoersholm, Denmark and Oncology Venture Sweden AB 

Liplacis

Targeted liposomal formulation of cisplatin 

Hard-to-treat metastatic breast cancer 

Company discussed interim phase II data with Danish Medicines Agency and proposed pivotal trial design; regulators indicated that the planned trial will be considered a pivotal phase II study if accepted for the EMA PRIME program

7/2/18

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

Keytruda (pembrolizumab)

PD-1-targeting antibody

Urothelial cancer

FDA posted its June 19 action in which it limited the drug's use to patients with locally advanced or metastatic disease who are not eligible for cisplatin-containing therapy; decision was based on data showing decreased survival with Tecentriq as a monotherapy vs. platinum-based chemotherapy in patients who have not received prior therapy and have low expression of PD-L1

7/3/18

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

Keytruda (pembrolizumab)

Anti-PD-1 antibody

Advanced hepatocellular carcinoma

FDA accepted the supplemental BLA seeking accelerated approval and granted priority review; PDUFA date is Nov. 9, 2018

7/11/18

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

Keytruda (pembrolizumab)

Anti-PD-1 antibody

Unresectable or metastatic melanoma

Approved by the China National Drug Administration for use in adults following failure of 1 prior line of therapy

7/26/18

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

Keytruda (pembrolizumab)

Anti-PD-1 antibody

First-line metastatic nonsquamous non-small-cell lung cancer

EMA's CHMP recommended approval in combination with pemetrexed and platinum chemotherapy in adults whose tumors have no EGFR or ALK genomic tumor aberrations, regardless of PD-L1 expression

7/30/18

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

Keytruda (pembrolizumab) 

Anti-PD-1 antibody 

Metastatic squamous non-small-cell lung cancer 

FDA accepted for priority review the supplemental BLA seeking approval in combination with carboplatin-paclitaxel or nab-paclitaxel as a first-line treatment, regardless of PD-L1 expression; PDUFA date is Oct. 30, 2018

7/2/18

Merck KGaA, of Darmstadt, Germany

Euthyrox (levothyroxine)

Synthetically produce hormone corresponding to thyroxin

Hyperpara-thyroidism, goiter and to suppress TSH in post-treatment of differentiated thyroid cancer

German Federal Institute for Drugs and Medical Devices recommended approval of the new formulation in 21 EU countries

7/26/18

Mersana Therapeutics Inc., of Cambridge, Mass.

XMT-1522

Antibody-drug conjugate targeting HER2-expressing tumors

HER2-positive breast and gastric cancers, advanced breast cancer with low HER2 expression and non-small-cell lung cancer

FDA placed a phase I study on partial clinical hold; no new patients will be enrolled, though current study participants will continue to receive drug and otherwise participate in the trial consistent with protocol; hold was initiated following report of a grade 5 serious adverse event (patient death) in dose level 7 that was classified as possibly drug-related

7/19/18

Novartis AG, of Basel, Switzerland

Tafinlar (dabrafenib) plus Mekinist (trametinib)

BRAF kinase inhibitor plus MEK1/2 inhibitor

Adjuvant treatment of stage III melanoma with BRAFV600 mutation

EMA's CHMP recommended approval for use in adults following complete resection

7/27/18

Oasmia Pharmaceutical AB, of Uppsala, Sweden

Apealea

Cremophor- and albumin-free formulation of paclitaxel

Epithelial ovarian cancer

Company decided to withdraw EMA orphan medicine application following new prevalence data; Apealea designated orphan drug by the EC in 2006; company also disclosed that the EMA's CHMP determined that the remaining list of outstanding issues must be addressed for the pending MAA

7/27/18

Pfizer Inc., of New York

Trazimera

Biosimilar to Herceptin (trastuzumab)

HER2 overexpressing breast cancer and HER2 overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma

EC granted marketing authorization

7/31/18

Progenics Pharmaceuticals Inc., of New York

Azedra (iobenguane I 131)

Radiolabeled guanidine analog

Lobenguane scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma

FDA approved the NDA

7/30/18

Puma Biotechnology Inc., of Los Angeles

Nerlynx (neratinib)

Pan-HER kinase inhibitor

Extended adjuvant treatment of early stage HER2-overexpressed/amplified breast cancer following adjuvant trastuzumab-based therapy

Health Canada accepted the new drug submission for review

7/31/18

Roche Holding AG, of Basel, Switzerland

Tecentriq (atezolizumab) plus Avastin (bevacizumab)

Anti-PD-L1 and anti-VEGF antibodies

First-line treatment of advanced or metastatic hepatocellular carcinoma

FDA granted breakthrough therapy designation for the combination

7/18/18

Roche Indonesia, of Jakarta, Indonesia, a unit of Roche Holding AG

Subcutaneous trastuzumab

HER2-targeting antibody

HER2-positive breast cancer

Approved by Indonesia's National Agency of Drug and Food Control

7/11/18

Sellas Life Sciences Group Inc., of New York

Galinpepimut-S

Heteroclitic multivalent, multipeptide cancer immunotherapeutic agent

Multiple myeloma

FDA granted fast track status

7/20/18

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

DSP-1958

Thiotepa

Solid tumors

Submitted NDA in Japan for conditioning treatment prior to autologous hematopoietic stem cell transplantation in pediatric solid tumors

7/5/18

Takeda Pharmaceutical Co. Ltd., of Osaka, Japan

Alunbrig (brigatinib)

ALK inhibitor

ALK-positive metastatic non-small-cell lung cancer

Approved by Health Canada for use as a monotherapy in adults whose disease has progressed on or who were intolerant to an ALK inhibitor

7/30/18

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

Erleada (apalutamide)

Androgen receptor inhibitor

Prostate cancer

Health Canada approved as an oral tablet for patients with nonmetastatic castration-resistant prostate cancer; product was granted priority review

7/5/18

Cardiovascular

United Therapeutics Corp., of Silver Spring, Md.

Remodulin (treprostinil)

Prostacyclin vasodilator

Pulmonary arterial hypertension

FDA approved he NDA

7/31/18

Dermatologic

Aclaris Therapeutics Inc., of Wayne, Pa.

ATI-502

Topical JAK1/3 inhibitor

Alopecia areata

FDA granted fast track designation for alopecia areata, including patchy alopecia areata and more severe variants alopecia totalis and universalis

7/9/18

Almirall SA, of Barcelona, Spain

Tildrakizumab

Anti-interleukin-23 antibody

Moderate to severe chronic plaque psoriasis

Received positive opinion from the EMA's CHMP

7/27/18

Aslan Pharmaceuticals Pte Ltd., of Singapore

ASLAN-004

Fully human monoclonal antibody targeting IL-13 receptor alpha1 subunit

Atopic dermatitis

Submitted a clinical trial authorization application with the Singapore Health Sciences Authority for a phase I trial

7/3/18

Biopharmax Corp., of Menlo Park, Calif.

BPX-01

Topical gel formulation of minocycline

Inflammatory acne

FDA waived the requirement for a dermal carcinogenicity study, eliminating several years of nonclinical research normally required for review

7/19/18

Endocrine/Metabolic

Aegerion Pharma-ceuticals Inc., of Windsor, U.K., a unit of Novelion Therapeutics Inc., of Vancouver, British Columbia

Myalepta (metreleptin)

Leptin replacement therapy

Lipodystrophy

EC granted marketing authorization

7/31/18

Alnylam Pharmaceuticals Inc., of Cambridge, Mass.

Patisiran

RNAi therapeutic targeting transthyretin

Hereditary transthyretin-mediated amyloidosis

EMA's CHMP recommended approval in adults with stage 1 or stage 2 polyneuropathy; if approved, drug will be sold under the brand Onpattro

7/27/18

Amarin Corp. plc, of Dublin

Vascepa (icosapent ethyl)

Highly-pure, omega-3 fatty acid product

Adjunct to diet to reduce triglyceride levels in adults with severe hypertriglyceridemia

Approved by the United Arab Emirates Ministry of Health and Prevention

7/26/18

Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis

Jardiance (empagliflozin), Synjardy (empagliflozin/metformin) and Glyxambi (empagliflozin/linagliptin) 

SGLT2 inhibitor-based therapies 

Type 2 diabetes

EMA's CHMP recommended label updates to include additional data from the EMPA-REG OUTCOME trial on heart failure and kidney endpoints, specifically a relative risk reduction in hospitalization for heart failure by 35% and a relative risk reduction for new-onset or worsening of kidney disease by 39%

7/2/18

Dicerna Pharmaceuticals Inc., of Cambridge, Mass.

DCR-PHXC

Next-generation RNAI product developed using Galxc technology

Primary hyperoxaluria

EMA's CHMP recommended orphan designation

7/11/18

Eli Lilly and Co., of Indianapolis

Trulicity (dulaglutide)

GLP-1 receptor agonist

Type 2 diabetes

U.S. label updated to include safety and efficacy in people with type 2 diabetes who have moderate to severe chronic kidney disease, based on data from the AWARD-7 trial

7/19/18

Ferring Pharmaceuticals Inc., of Parsippany, N.J.

Zomacton (somatropin)

Recombinant human growth hormone

Growth failure in pediatric patients due to inadequate secretion of endogenous growth hormone and replacement of endogenous GH in adults with GH deficiency

4 pediatric indications approved by FDA: idiopathic short stature; short stature associated with Turner syndrome; short stature born small for gestational age with no catch-up growth by 2 to 4 years; and short stature or growth failure in short stature homeobox-containing gene deficiency

7/20/18

Fortress Biotech Inc., of New York 

Copper histidinate (CUTX-101) 

Subcutaneous injectable formulation intended to improve tolerability due to physiological pH and bypass the oral absorption of copper 

Classic Menkes disease 

FDA granted fast track designation to Fortress subsidiary Cyprium Therapeutics for use in patients who have not demonstrated significant clinical progression

7/2/18

Janssen Pharmaceutical Cos., of Raritan, N.J., part of Johnson & Johnson

Invokana (canagliflozin)

SGLT2 inhibitor

Reducing the risk of major adverse cardiovascular events in adults with type 2 diabetes

FDA extended the review timeline for the sNDA, seeking approval in patients who are at risk for CV disease; action date is now in October 2018

7/13/18

Opko Health Inc., of Miami, and Vifor Fresenius Medical Care Renal Pharma Ltd., of Paris

Rayaldee

Extended-release prohormone of calcitriol

Secondary hyperparathyroidism

Approved by Health Canada for use in adults with stage 3 or 4 chronic kidney disease and vitamin D deficiency

7/16/18

Ultragenyx Pharmaceutical Inc., of Novato, Calif.

DTX-401

AAV-based gene therapy

Glycogen storage disease type 1a

FDA granted fast track designation

7/26/18

Gastrointestinal

Aequus Pharmaceuticals Inc., of Vancouver, British Columbia

AQS-1303

Anti-nausea transdermal patch

Nausea and vomiting during pregnancy

Received marketing authorization in the U.K. under the brand Xonvea

7/19/18

Emmaus Life Sciences Inc., of Torrance, Calif.

L-glutamine oral powder

Increases synthesis of glutathione in the intestine

Diverticulosis

FDA accepted the IND for a pilot study set to start by the end of 2018

7/9/18

Merz North America Inc., of Raleigh, N.C.

Xeomin

Incobotulinum-toxinA

Sialorrhea (excessive drooling)

Approved by FDA for use in adults; sBLA was cleared under priority review

7/3/18

Genitourinary/Sexual Function

Abbvie Inc., of North Chicago, and Neurocrine Biosciences Inc., of San Diego

Orilissa (elagolix)

Oral gonadotropin-releasing hormone antagonist

Moderate to severe endometriosis pain

Approved by FDA under priority review

7/24/18

Hansa Medical AB, of Lund, Sweden

Imlifidase

Immunoglobulin G-degrading enzyme of Streptococcus pyogenes

Anti-GBM antibody disease (Goodpasture disease)

FDA granted orphan designation

7/9/18

Hematologic

Accord Healthcare Inc., of Ahmadabad, India

Pelgraz

Pegylated G-CSF biosimilar

Neutropenia

EMA's CHMP issued a positive opinion for use of the drug to reduce duration of neutropenia and the incidence of febrile neutropenia in adults undergoing cytotoxic chemotherapy

7/27/18

Bluebird Bio Inc., of Cambridge, Mass.

Lentiglobin

Gene therapy

Transfusion-dependent beta-thalassemia and a non-β0/β0 genotype

EMA's CHMP granted accelerated assessment; company intends to file MAA in 2018

7/27/18

Coherus Biosciences Inc., of Redwood City, Calif.

Udenyca (CHS-1701)

Pegfilgrastim biosimilar

Neutropenia

EMA's CHMP issued a positive opinion

7/27/18

CTI Biopharma Corp., of Seattle

Pacritinib

Inhibitor of JAK2, FLT3, IRAK1 and CSF1R

Myelofibrosis

After getting advice in a type B meeting with the FDA, company plans to run a phase III study starting in 2019 

7/18/18

Pfizer Inc., of New York

Nivestym (filgrastim-aafi)

Biosimilar of Neupogen (filgrastim)

Neutropenia

Approved by FDA for all eligible indications of the reference product

7/20/18

Portola Pharmaceuticals Inc., of South San Francisco

Betrixaban

Once-daily factor Xa inhibitor

Prevention of venous thromboembolism

EMA's CHMP maintained its negative opinion following a re-examination procedure requested by the company; MAA seeks approval for use in adults hospitalized with acute medical illness with risk factors for VTE

7/27/18

Shire plc, of Dublin 

Veyvondi (vonicog alfa) 

Recombinant von Willebrand factor 

Bleeding events and treatment/prevention of surgical bleeding in von Willebrand disease patients 

EMA's CHMP recommended approval for treating adults when desmopressin treatment alone is ineffective or not indicated

7/2/18

Immune

Cellix Biosciences Inc., of Newark, N.J.

CLX-106

Molecular conjugate designed to deliver monomethyl fumarate

Relapsing-remitting multiple sclerosis

FDA confirmed the 505(b)(2) pathway is appropriate

7/11/18

Enzyvant Sciences GmbH, of Basel, Switzerland

RVT-802

Tissue-based regenerative therapy

Primary immune deficiency resulting from congenital athymia associated with complete DiGeorge anomaly

Initiated a rolling BLA to the FDA; company expects to complete submission by the end of 2018

7/9/18

Gamida Cell Ltd., of Tel Aviv, Israel

Nicord

Ex vivo expanded cell graft derived from umbilical cord stem cells

Hematopoietic stem cell transplantation

FDA granted orphan designation

7/17/18

Merck KGaA, of Darmstadt, Germany

Cladribine tablets

Activated by lymphocytes

Relapsing multiple sclerosis

Resubmission of NDA accepted for filing by FDA

7/30/18

Mylan NV, of Hertfordshire, U.K., and Fujifilm Kyowa Kirin Biologics Co. Ltd., of Tokyo

Hulio (FKB-327)

Biosimilar to TNF-alpha inhibitor Humira (adalimumab)

Rheumatoid arthritis, psoriasis, ankylosing spondylitis, psoriatic arthritis, et al.

EMA's CHMP adopted a positive opinion for the biosimilar for all reference drug indications

7/27/18

Nohla Therapeutics Inc., of Seattle

Dilanubicel (NLA-101)

Off-the-shelf, ex vivo expanded hematopoietic stem and progenitor cell therapy

Reduction of morbidity and mortality associated with hematopoietic stem cell transplant

FDA granted orphan designation

7/16/18

Sandoz, a unit of Basel, Switzerland-based Novartis AG

Hyrimoz (adalimumab)

Biosimilar of anti-TNF-alpha antibody

Rheumatoid arthritis, plaque psoriasis, Crohn's disease, uveitis, ulcerative colitis, et al.

EC granted marketing approval for use in all indications of the reference drug

7/27/18

Infection

60 Degrees Pharmaceuticals LLC, of Washington

Arakoda (tafenoquine)

8-aminoquinoline chemically derived from Primaquine

Prevention of malaria

FDA's Antimicrobial Drugs Advisory Committee voted 11-2 to support the drug's efficacy and 9-4 in favor of its safety

7/26/18

Adma Biologics Inc., of Ramsey, N.J.

Bivigam

Intravenous immune globulin (human)

Prevention and treatment of certain infectious diseases

FDA acknowledged receipt of the prior approval supplement filing for review, seeking approval to amend use for Bivigam; if approved, Adma plans to relaunch the product in the U.S.; target action date is Oct. 25, 2018; the company also reported that the FDA closed its manufacturing inspection for the Boca Raton, Fla.-based facility

7/26/18

Ascletis Bioscience Co. Ltd., of Hangzhou, China

Ravidasvir 

NS5A inhibitor

Hepatitis C

Filed a new drug application with the China FDA for use in combination with Ganovo

7/31/18

Cellerant Therapeutics Inc., of San Carlos, Calif. 

Romyelocel-L 

Human myeloid progenitor cells 

Prevention of serious bacterial and fungal infections in de novo acute myeloid leukemia patients undergoing induction chemotherapy 

FDA granted regenerative medicine advanced therapy designation

7/2/18

Gilead Sciences Inc., of Foster City, Calif.

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

Once-daily, single-tablet regimen comprising an integrase strand transfer inhibitor with dual nucleoside reverse transcriptase inhibitor backbone

HIV-1 infection

Approved by Health Canada

7/12/18

Glaxosmithkline plc, of London

Krintafel (tafenoquine)

8-aminoquinoline derivative

Prevention of relapse of Plasmodium vivax malaria

Approved by FDA under priority review for use in patients, 16 and older, who are receiving appropriate antimalarial therapy for acute P. vivax infection

7/20/18

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

Symtuza (darunavir, cobicistat, emtricitabine and tenofovir alafenamide)

Single-tablet regimen

HIV-1 in treatment-naïve and certain virologically suppressed adults

FDA approved the drug

7/17/18

Siga Technologies Inc., of New York

Tpoxx (tecovirimat)

Antiviral

Smallpox

Approved by FDA; awarded a material threat medical countermeasure priority review voucher

7/13/18

Tetraphase Pharmaceuticals Inc., of Watertown, Mass.

Xerava (eravacycline)

Fully synthetic fluorocycline antibiotic

Complicated intra-abdominal infections

EMA's CHMP recommended approval in adults; EC expected to make final decision within 3 months

7/28/18

Theratechnologies Inc., of Montreal

Egrifta (tesamorelin)

Stimulates growth hormone secretion

HIV-associated lipodystrophy

Filed an sNDA with FDA for a single-vial formulation that is 4 times more concentrated than the currently commercialized formulation

7/5/18

Theratechnologies Inc., of Montreal

Trogarzo

Ibalizumab-uiyk

HIV

EMA will review the application for marketing authorization of Trogarzo injection under the accelerated assessment procedure

7/31/18

Utility Therapeutics Ltd., of London

Pivmecillinam

Beta-lactam antibiotic

Uncomplicated urinary tract infection

Designated a qualified infectious disease product by the FDA

7/16/18

Inflammatory

Corbus Pharmaceuticals Holdings Inc., of Norwood, Mass.

Lenabasum

Synthetic oral endocannabinoid-mimetic drug

Dermatomyositis

FDA granted orphan designation

7/20/18

Kolon Tissuegene Inc., of Rockville, Md.

Invossa

Allogeneic cell and gene therapy

Knee osteoarthritis

FDA lifted the clinical hold so firm can move forward with phase III trials

7/6/18

Taiwan Liposome Co. Ltd., of Taipei, Taiwan

TLC-599

Bioseizer formulation of dexamethasone sodium phosphate

Mild to moderate knee osteoarthritis

Cleared by FDA to begin a phase II, open-label, pharmacokinetic trial; enrollment set to start in the fourth quarter of 2018

7/9/18

Musculoskeletal

Acceleron Pharma Inc., of Cambridge, Mass.

ACE-083

Locally acting drug based on follistatin

Facioscapulo-humeral muscular dystrophy

FDA granted orphan designation

7/12/18

Innovent Biologics Inc., of Suzhou, China

IBI-307

Anti-RANKL antibody

Osteoporosis and lytic bone lesions associated with cancer metastasis

Received IND approval from Chinese regulators

7/26/18

Inozyme Pharma Inc., of Boston

INZ-701

Enzyme replacement therapy

ENPP1 deficiency

FDA granted orphan designation

7/17/18

Nektar Therapeutics Inc., of San Francisco

NKTR-181

Long-acting, selective full mu-opioid receptor agonist

Chronic low back pain

FDA accepted for review the NDA seeking approval for use in adults new to opioid therapy; PDUFA date expected to be May 28, 2019

7/30/18

Wave Life Sciences Ltd., of Cambridge, Mass. 

WVE-210201 

Stereopure oligonucleotide designed to induce skipping of exon 51 of dystrophin

Duchenne muscular dystrophy 

EMA's CHMP issued positive opinion for orphan designation 

7/2/18

Neurology/Psychiatric

Alkermes plc, of Dublin 

Aristada Initio (aripiprazole lauroxil) 

Long-acting injectable atypical antipsychotic 

Schizophrenia

Approved by FDA for use in adults

7/2/18

Allergan plc, of Dublin

AGN-241751

Oral, small-molecule N-methyl-D-aspartate receptor modulator

Major depressive disorder

FDA granted fast track designation

7/23/18

Asceneuron SA, of Asceneuron, Switzerland

ASN-120290

O-GlcNAcase inhibitor

Progressive supranuclear pals

FDA granted orphan drug designation

7/18/18

Durect Corp., of Cupertino, Calif., and Indivior, of Slough, U.K.

Perseris (risperidone, RBP-700)

Long-acting antipsychotic

Schizophrenia

Approved by FDA for use in adults

7/30/18

Insys Therapeutics Inc., of Phoenix

Buprenorphine sublingual spray

Binds to opioid receptors

Moderate to severe acute pain

Received a complete response letter from the FDA; company is assessing next steps

7/27/18

Ironshore Pharmaceuticals & Development Inc., of George Town, Grand Cayman

HLD-200

Delayed-release and extended-release methylphenidate

Attention deficit hyperactivity disorder

FDA accepted for review the NDA and set a PDUFA date of Aug. 8, 2018

7/13/18

Luye Pharma Group, of Shanghai

Seroquel XR (quetiapine fumarate)

Atypical antipsychotic

Depression arising from bipolar disorder

Received approval in China for the new indication

7/23/18

Neurim Pharmaceuticals Ltd., of Tel Aviv, Israel

Slenyto

Pediatric prolonged-release melatonin coated tablets

Insomnia

EMA's CHMP recommended approval for use in children and adolescents with autism spectrum disorder and/or Smith-Magenis syndrome

7/30/18

Novartis AG, of Basel, Switzerland

Aimovig (erenumab)

CGRP-R inhibitor

Prevention of migraine in adults experiencing 4 or more migraine days per month

EC granted marketing authorization

7/30/18

Painreform Ltd., of Herzliya, Israel

PRF-110

Extended-release ropivacaine

Postoperative pain

FDA gave approval to run 2 pivotal studies

7/18/18

Pharnext SA, of Paris

PXT-3003

Low-dose fixed combination of baclofen, naltrexone and sorbitol

Charcot-Marie-Tooth disease type 1A

EMA agreed with the firm's pediatric investigation plan

7/10/18

Reata Pharmaceuticals Inc., of Irving, Texas

Omaveloxolone

Oral, once-daily activator of Nrf2

Friedreich's ataxia

EC granted orphan drug designation

7/11/18

Sedor Pharmaceuticals LLC, of Paoli, Pa.

I.M./I.V. Captisol-enabled fosphenytoin

Fosphenytoin, sodium and sulfobutylether beta cyclodextrin sodium for injection

Generalized tonic-clonic status epilepticus/prevention and treatment of seizures during neurosurgery/short-term use for oral phenytoin

FDA accepted the 505(b)(2) NDA

7/30/18

Sumitomo Dainippon Pharma Co. Ltd. and Nitto Denko Corp., both of Osaka, Japan

Lonasen (blonanserin)

Atypical antipsychotic

Schizophrenia

Submitted NDA in Japan for a transdermal patch formulation of the drug

7/31/18

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

Trerief (zonisamide) 

Antiepileptic

Parkinsonism in dementia with Lewy bodies 

Approved in Japan

7/2/18

Tonix Pharmaceuticals Holding Corp., of New York

TNX-102 SL

Cyclobenzaprine HCl sublingual tablets

Agitation in Alzheimer's disease

FDA granted fast track designation

7/16/18

Voyager Therapeutics Inc., of Cambridge, Mass.

VY-AADC

Gene therapy designed to deliver AADC gene

Parkinson's disease patients with motor fluctuations refractory to medical management

Reported feedback from a type C meeting with the FDA; agency indicated in a written response that the phase II study in about 42 patients, if it meets its primary endpoint of demonstrating a statistically significant difference of diary on-time without dyskinesia vs. the placebo surgery group and in the absence of major safety concerns, likely may be considered sufficient for BLA submission; if phase II data are supportive only, the randomized, placebo-controlled phase III trial in about 120 patients, if it achieves its primary endpoint, also likely may be considered sufficient for a BLA

7/17/18

Ocular

Aerie Pharmaceuticals Inc., of Durham, N.C.

Roclatan (netarsudil/latanoprost ophthalmic solution 0.02%/0.005%)

Rho kinase inhibitor/prostaglandin analogue

Reducing intraocular pressure in glaucoma or ocular hypertension

Received the day 74 notification from the FDA, which completed its 60-day review of the NDA and determined the application is sufficiently complete to permit a substantive review; the PDUFA date is March 14, 2019

7/23/18

Apellis Pharmaceuticals Inc., of Crestwood, Ky.

APL-2

Inhibitor of complement factor C3

Geographic atrophy

FDA granted fast track status in GA, an advanced form of age-related macular degeneration

7/24/18

Ocular Therapeutix Inc., of Bedford, Mass.

Dextenza

Dexamethasone insert 0.4 mg

Ocular pain following ophthalmic surgery

FDA acknowledged acceptance of the resubmitted NDA; considered a class 2 response to the July 2017 complete response letter; PDUFA date is Dec. 28, 2018

7/19/18

Other/Miscellaneous

Akcea Therapeutics Inc., of Cambridge, Mass., and Ionis Pharmaceuticals Inc., of Carlsbad, Calif.

Tegsedi (inotersen)

Antisense oligonucleotide inhibitor of human TTR production

Stage 1/2 polyneuropathy in adults with hereditary transthyretin amyloidosis

Received marketing authorization from the EC

7/11/18

Clinuvel Pharmaceuticals Ltd., of Melbourne, Australia

Scenesse (afamelanotide 16 mg)

Alpha-melanocyte-stimulating hormone analogue

Erythropoietic protoporphyria

Completed NDA submission to the FDA

7/5/18

Rhythm Pharmaceuticals Inc., of Boston

Setmelanotide

Melanocortin-4 receptor agonist

Rare genetic disorders of obesity

EMA granted priority medicines, or PRIME, designation for treatment of obesity and the control of hunger associated with deficiency disorders of the MC4R pathway

7/23/18

Soleno Therapeutics Inc., of Redwood City, Calif.

Diazoxide choline controlled-release

Extended-release, crystalline salt formulation of diazoxide

Prader-Willi syndrome

FDA granted fast track designation

7/30/18

Respiratory

Glaxosmithkline plc, of London

Nucala (mepolizumab)

Monoclonal antibody targeting interleukin-5

Chronic obstructive pulmonary disease

FDA's Pulmonary Allergy Drugs Advisory Committee voted 16-3 that the risk-benefit profile was not adequate to support approval for use as an add-on treatment to reduce exacerbations in COPD

7/25/18

Vertex Pharmaceuticals Inc., of Cambridge, Mass.

Symkevi (tezacaftor/ivacaftor)

Cystic fibrosis transmembrane conductance regulator corrector/CFTR potentiator

Cystic fibrosis

EMA's CHMP recommended approval for people with CF, ages 12 and older, who either have 2 copies of the F508del mutation in the CFTR gene, or 1 copy of the F508del mutation and a copy of 1 of the following 14 mutations: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T

7/28/17

Toxicity and Intoxication

Braeburn Pharmaceuticals Inc., of Princeton, N.J., and Camurus AB, of Lund, Sweden

CAM-2038

Buprenorphine flexible-dose weekly and monthly subcutaneous depot injection

Opioid use disorder

FDA assigned a PDUFA date of Dec. 26, 2018, for the NDA, which has fast track and priority review designations

7/16/18

Helsinn Healthcare SA, of Lugano, Switzerland

Akynzeo (netupitant/palonosetron)

NK1 receptor antagonist and 5-HT3 receptor antagonist

Nausea and vomiting associated with highly or moderately emetogenic cancer chemotherapy

Korean Ministry of Food and Drug Safety approved the drug for use

7/31/18

Tetra Bio-Pharma Inc., of Orleans, Ontario

PPP-002

Dronabinol Adversa mucoadhesive product

Chemotherapy-induced nausea and vomiting, anorexia and weight loss in AIDS

Received two letters from the FDA for type B and type C meetings to discuss requirements for obtaining approval under the 505(b)(2) pathway

7/26/18

Notes

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