Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Janssen Pharmaceutical K.K. (Tokyo; unit Johnson & Johnson)

Sirukumab

A human anti-interleukin-6 monoclonal antibody

Rheumatoid arthritis (RA)

Submitted a new drug application to Japan's Ministry of Health, Labour and Welfare seeking approval for the treatment of adult patients with moderately to severely active RA

10/26/16

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

Sarilumab

Interleukin-6 antibody

Moderate to severe rheumatoid arthritis

FDA issued a complete response letter due to deficiencies identified during a routine good manufacturing practice (GMP) inspection of the Sanofi Le Trait facility in France where the drug is filled and finished; Sanofi submitted a comprehensive corrective action plan to the FDA

10/31/16

Tigenix NV (Leuven, Belgium)

Cx601

Suspension of allogeneic expanded adipose-derived stem cells locally injected

Complex perianal fistulas in Crohn's disease

Was granted orphan drug designation status in Switzerland

10/18/16

CANCER

Abbvie Inc. (North Chicago)

Venclexta

Venetoclax; small-molecule antineoplastic drug acts by inhibiting B-cell lymphoma-2

Chronic lymphocytic leukemia

EMA recommended conditional approval on the basis of a single-arm pivotal phase II study

10/17/16

Actinium Pharmaceuticals Inc. (New York)

Iomab-B

A radioimmunoconjugate consisting of murine monoclonal antibody BC8 and iodine-131 radioisotope

Relapsed or refractory acute myeloid leukemia

EMA awarded orphan designation to Iomab-B for use in preparing patients with relapsed or refractory acute myeloid leukemia, who are older than 55, for a bone marrow transplant

10/19/16

Astellas Pharma Inc. (Tokyo) and Pfizer Inc. (New York)

Xtandi

Enzalutamide capsules; an androgen receptor inhibitor

Metastatic castration-resistant prostate cancer

FDA approved a supplemental NDA to update the labeling for Xtandi to include new clinical data vs. bicalutamide from the TERRAIN study; the data demonstrated improvement in radiographic progression-free survival in patients treated with enzalutamide compared to patients who were treated with bicalutamide

10/24/16

Bristol-Myers Squibb Co. (New York)

Opdivo

PD-1 inhibitor; nivolumab

Relapsed or refractory classical Hodgkin lymphoma

EMA's Committee for Medicinal Products for Human Use recommended approval of Opdivo to treat lymphoma after autologous stem cell transplant and treatment with Adcetris (brentuximab vedotin, Seattle Genetics Inc.)

10/17/16

Bristol-Myers Squibb Co. (New York)

Opdivo

PD-1 inhibitor; nivolumab

Locally advanced unresectable or metastatic urothelial carcinoma

FDA accepted a supplemental BLA seeking expanded use of Opdivo; the PDUFA date is March 2, 2017

10/24/16

Eli Lilly and Co. (Indianapolis)

Lartruvo

Olaratumab (IMC-3G3, LY-3012207)

Soft tissue sarcoma

Gained accelerated approval from the FDA for use with doxorubicin to treat patients whose STS cannot be removed using radiation or surgery and for whom anthracycline is not appropriate

10/20/16

Genentech Inc. (South San Francisco; unit of Roche Holdings AG)

Tecentriq

Atezolizumab

Metastatic non-small-cell lung cancer

FDA approved it for patients who have disease progression during or following platinum-containing chemotherapy, and have progressed on an appropriate FDA-approved targeted therapy if their tumor has EGFR or ALK gene abnormalities

10/20/16

Immune Therapeutics Inc. (Orlando, Fla.)

Lodonal

Naltrexone

HIV/AIDS, cancer and immune boosting

The Ministry of Health and Prevention Division of Pharmacy in the Republic of Senegal accepted for review the company's new drug applications for Lodonal as a stand alone or adjunct therapy non-toxic in the treatment of HIV/AIDS, cancer and as an immune booster

10/27/16

Keystone Nano Inc. (State College, Pa.)

Ceramide

Ceramide Nanoliposome (KN-001)

Liver cancer

Received FDA orphan drug status for the use of Ceramide to treat liver cancer

10/19/16

Merck & Co Inc. (Kenilworth, N.J.)

Keytruda

Pembrolizumab; anti-PD-1 therapy

Non-small-cell lung cancer (NSCLC)

Gained FDA approval to market it for the first-line treatment of patients with metastatic NSCLC whose tumors have high PD-L1 expression with no EGFR or ALK genomic tumor aberrations

10/26/16

Opsona Therapeutics Ltd. (Dublin)

OPN-305

Humanized IgG4 monoclonal antibody against Toll-like receptor 2

Myelo-dysplastic syndromes

Received FDA orphan drug designation

10/26/16

Pharmamar SA (Madrid)

Aplidin

Plitidepsin

Relapsed/refractory multiple myeloma

EMA accepted the marketing authorization application for Aplidin in combination with dexamethasone

10/31/16

Philogen SpA (Siena, Italy)

L19TNF

Noncovalent trimer of tumor necrosis factor

Soft-tissue sarcoma

European Commission granted orphan drug designation

10/31/16

Roche Holding AG (Basel, Switzerland)

Ventana

PD-L1 assay

Metastatic non-small-cell lung cancer

FDA approved the Ventana PD-L1 assay as a complementary diagnostic to identify PD-L1 expression levels in patients considering treatment with immunotherapy drug Tecentriq (atezolizumab)

10/28/16

Shire plc (Dublin)

Onivyde

Pegylated liposomal irinotecan hydrochloride trihydrate; MM-398

Metastatic adenocarcinoma of the pancreas

European Commission granted marketing authorization of Onivyde for use in combination with 5-FU and leucovorin, in adults whose disease has progressed following gemcitabine-based therapy

10/19/16

Sunesis Pharmaceuticals Inc. (South San Francisco)

Qinprezo

Vosaroxin; anticancer quinolone derivative

Relapsed/refractory acute myeloid leukemia

Submitted its responses to the EMA's day 120 list of questions issued by the Committee for Medicinal Products for Human Use as part of the centralized review process of the marketing authorization application for vosaroxin in relapsed/refractory acute myeloid leukemia patients, ages 60 and older

10/17/16

Taiwan Liposome Co. Ltd. (Taipei, Taiwan)

TLC178

A liposomal-encapsulated formulation of vinorelbine

Cutaneous T-cell lymphoma

FDA granted orphan designation for TLC178

10/20/16

Tesaro Inc. (Waltham, Mass.)

Niraparib

Niraparib

Platinum-sensitive, recurrent ovarian cancer

EMA accepted the marketing authorization application for niraparib for the maintenance treatment of patients with platinum-sensitive, recurrent ovarian cancer who are responders to platinum-based chemotherapy

10/31/16

Teva Pharmaceutical Industries Ltd. (Jerusalem)

Trisenox

Arsenic trioxide

Low to intermediate risk acute promyelocytic leukemia

CHMP of the EMA has adopted a positive opinion recommending an indication extension of Trisenox for use in newly diagnosed disease in combination with retinoic acid

10/18/16

Yisheng Biopharma Co. Ltd. (Beijing)

YS-ON-001

Designed to work via immunomodulating effects

Hepato-cellular carcinoma

FDA granted orphan designation

10/25/16

CARDIOVASCULAR

Janssen Pharmaceuticals Inc. (Raritan, N.J.)

Xarelto

Rivaroxaban

Atrial fibrillation

FDA gave the all-clear to Xarelto as a safe and effective alternative to warfarin in patients with atrial fibrillation

10/12/16

La Jolla Pharmaceutical Co. (San Diego)

LJPC-401

Synthetic human hepcidin

Sickle cell disease

The EMA's Committee for Orphan Medicinal Products issued a positive opinion recommending LJPC-401 for designation as an orphan medicinal product for the treatment of sickle cell disease

10/25/16

Portola Pharmaceuticals Inc. (South San Francisco)

Betrixaban

Factor Xa inhibitor anticoagulant

Venous thromboembolism (VTE)

Submitted an NDA to the FDA seeking approval to market betrixaban for extended-duration prophylaxis of VTE in acute medically ill patients with risk factors for VTE

10/26/16

Swedish Orphan Biovitrum AB (Stockholm) and Biogen Inc. (Cambridge, Mass.)

Alprolix

Recombinant factor IX Fc fusion protein therapy; eftrenonacog alfa

Hemophilia B

The Swiss Agency for Therapeutic Products, Swissmedic, approved it to treat hemophilia B

10/31/16

CENTRAL NERVOUS SYSTEM

Adamas Pharmaceuticals Inc. (Emeryville, Calif.)

ADS-5102

Amantadine hydrochloride extended-release capsules

Levodopa-induced dyskinesia in Parkinson's disease

Submitted an NDA to the FDA for ADS-5102

10/28/16

Egalet Corp. (Malvern, Pa.)

Arymo ER

Morphine sulfate extended-release tablets

Pain

The FDA will not meet its Oct. 14 PDUFA date for Arymo ER, indicating that it needs more time, as it has with other applications for abuse-deterrent opioids; the company said the FDA identified no particular issue with its application and that no additional scientific data are needed

10/17/16

Ligand Pharmaceuticals Inc. (San Diego) and Lundbeck A/S (Copenhagen)

Carnexiv

Intravenous formulation of the antiepileptic drug carbamazepine

Seizures

FDA approved it as a short-term replacement therapy in adults with certain seizure types when oral administration is temporarily not feasible

10/11/16

Orexo AB (Uppsala, Sweden)

Zubsolv

Buprenorphine and naloxone sublingual tablets

Opioid dependence

Won U.S. approval of Zubsolv 0.7 mg/0.18 mg for the treatment of patients with opioid dependence; the drug was originally approved by the FDA on July 3, 2013, in two dosages of 5.7-mg/1.4-mg and 1.4-mg/0.36-mg sublingual tablet strengths

10/7/16

Shire plc (Dublin)

Vyvanse

Lisdexamfetamine dimesylate

Moderate to severe binge eating disorder

An update to Vyvanse labeling will include information regarding the approval of an sNDA by the FDA, the maintenance of efficacy data over 38 weeks

10/18/16

Shire plc (Dublin)

Vyvanse

Lisdexamfetamine dimesylate

Moderate to severe binge eating disorder

Health Canada approved Vyvanse for adults

10/26/16

Teva Pharmaceutical Industries Ltd. (Jerusalem)

SD-809

Deutetrabenazine; small-molecule inhibitor of vesicular monoamine 2 transporter

Chorea associated with Huntington disease

FDA accepted the resubmission of an NDA; the FDA has assigned a PDUFA date of April 3, 2017

10/21/16

DIABETES

Novo Nordisk A/S (Bagsvaerd, Denmark)

Victoza

Liraglutide

Type 2 diabetes

Submitted a supplemental NDA to the FDA and a type II variation to the EMA for including data from the LEADER cardiovascular outcomes trial in the product information of Victoza

10/26/16

INFECTION

Atara Biotherapeutics Inc. (South San Francisco)

EBV-CTL

Healthy donor's T cells are stimulated to recognize EBV antigens

Epstein-Barr virus

EMA's Committee for Medicinal Products for Human Use and Committee for Advanced Therapies granted access to the Priority Medicines (PRIME) regulatory initiative for EBV-CTL to treat rituximab-refractory Epstein-Barr virus-post-transplant lymphoproliferative disorder following hematopoietic cell transplant

10/19/16

Glaxosmithkline plc (London)

Shingrix

Non-live, recombinant vaccine

Shingles

Submitted a biologics license application for its candidate shingles vaccine, Shingrix, to the FDA, seeking approval for the prevention of herpes zoster (shingles) in people ages 50 or older

10/25/16

Melinta Therapeutics Inc. (New Haven, Conn.)

Baxdela

Intravenous and oral delafloxacin

Acute bacterial skin and skin structure infections

Submitted NDAs to the FDA

10/25/16

Merck & Co. Inc. (Kenilworth, N.J.)

Zinplava

Bezlotoxumab; a monoclonal antitoxin antibody targeting Clostridium difficile infection

Clostridium difficile infection

Won FDA approval for adults at high risk for recurrence of the infection who are also on antibacterial therapy

10/25/16

Protein Sciences Corp. (Meriden, Conn.)

Flublok

Influenza vaccine, quadrivalent formulation

Influenza

FDA approved its quadrivalent formulation of Flublok influenza vaccine, which is designed to protect against four strains of influenza, three of the same strains found in trivalent Flublok plus an additional B strain

10/12/16

INFLAMMATORY

Celgene Corp. (Summit, N.J.)

Otezla

Oral apremilast

Chronic plaque psoriasis

Received a positive determination from the National Institute for Health and Care Excellence (NICE)

10/21/16

MISCELLANEOUS

Abeona Therapeutics Inc. (New York)

ABO-102

Gene therapy

Sanfilippo syndrome type A

EMA's Committee for Orphan Medicinal Products granted orphan drug designation

10/19/16

Aerie Pharmaceuticals Inc. (Irvine, Calif.)

Rhopressa

A once-daily eye drop

Glaucoma or ocular hypertension

The company withdrew its Rhopressa NDA, submitted to the FDA in the third quarter of 2016, as the result of a third-party manufacturing facility in Tampa, Fla., not being ready for pre-approval inspection by the FDA

10/31/16

Alexion Pharmaceuticals Inc. (New Haven, Conn.)

ALXN1007

Monoclonal antibody targeting complement protein C5a

Acute graft-vs.-host disease

FDA granted orphan drug designation

10/21/16

Allergan plc (Dublin) and Serenity Pharmaceuticals LLC (Milford, Pa.)

SER120

Desmopressin nasal spray

Nocturia

FDA's Bone, Reproductive and Urologic Drugs Advisory Committee voted 14-4 in favor of the benefit-risk profile of the NDA; the committee also voted 17-1 that there is substantial evidence to conclude that at least one of the SER120 doses is effective

10/21/16

Apricus Biosciences Inc. (San Diego) and Recordati SpA (Milan, Italy)

Vitaros

A topical cream

Erectile dysfunction

Greece has granted marketing approval for Vitaros

10/27/16

Astrazeneca plc (London)

ZS-9

Sodium zirconium cyclosilicate

Hyperkalemia

FDA accepted a complete resubmission of the new drug application (NDA) for ZS-9

10/19/16

Atyr Pharma Inc. (San Diego)

Resolaris

Derived from a naturally occurring protein released in vitro by human skeletal muscle cells

Facioscapu-lohumeral muscular dystrophy

Was granted fast track designation by the FDA

10/25/16

Biogen Inc. (Cambridge, Mass.)

Nusinersen

Antisense oligonucleotide (ASO) that is designed to alter the splicing of pre-mRNA from the SMN2 gene

Spinal muscular atrophy

The new drug application for nusinersen was accepted by the FDA for priority review and that the marketing authorization application was validated by the EMA

10/31/16

Cymabay Therapeutics Inc. (Newark, Calif.)

MBX-8025

Peroxisome proliferator-activated receptor delta agonist

Primary biliary cholangitis (PBC)

Granted priority medicine designation by the EMA for PBC in patients who do not tolerate or respond to ursodeoxycholic acid (UDCA) or do not respond to combination UDCA/obeticholic acid treatment

10/21/16

Debiopharm International SA (Lausanne, Switzerland)

Debio 1450

Antibiotic

Osteomyelitis

Received European orphan designation

10/26/16

Eiger Biopharma-ceuticals Inc. (Palo Alto, Calif.)

Exendin 9-39

To be administered subcutaneously

Non-insulinoma pancreatogeneous hypoglycemia syndrome

EMA designated it an orphan drug

10/18/16

Ferring International Center SA (Saint Prex, Switzerland)

Rekovelle

Follitropin delta

For assisted reproduction

Received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) for use in controlled ovarian stimulation to develop multiple follicles in women undergoing assisted reproductive technologies

10/18/16

Genentech Inc. (South San Francisco)

Lucentis

Ranibizumab injection

Wet Age-related macular degeneration

FDA approved the Lucentis 0.5-mg prefilled syringe as a new method of administering the anti-VEGF treatment for wet age-related macular degeneration and macular edema after retinal vein occlusion

10/17/16

Kuros Biosciences AG (Zurich, Switzerland)

KUR-112

Parathyroid hormone-fibrin-based product

Solitary bone cysts

FDA granted rare pediatric disease designation

10/21/16

Lipocine Inc. (Salt Lake City)

LPCN 1021

Oral testosterone product

Hypogo-nadism

Has submitted a new dosing validation clinical study protocol that the FDA will review through a special protocol assessment (SPA) as a result of the complete response letter

10/18/16

PTC Therapeutics Inc. (South Plainfield, N.J.)

Translarna

Ataluren

Nonsense mutation Duchenne muscular dystrophy

FDA denied the company's first appeal of the refusal-to-file letter issued in February regarding the NDA; the CHMP of the EMA says it wants more information regarding the ongoing annual renewal of its MAA for Translarna

10/18/16

Solid Biosciences LLC (Cambridge, Mass.)

SGT-001

Gene therapy candidate

Duchenne muscular dystrophy

FDA and European Commission granted orphan designations

10/25/16

Swedish Orphan Biovitrum AB (Stockholm)

SOB1003

A chemically modified human recombinant sulfamidase

Mucopoly-saccharidosis type IIIA (Sanfilippo A syndrome)

European Commission granted orphan designation

10/20/16

RESPIRATORY

Corbus Pharmaceuticals Holdings Inc. (Norwood, Mass.)

Resunab

Synthetic, oral endocannabinoid-mimetic

Cystic fibrosis

European Commission granted orphan designation to Resunab; a phase II trial in CF is ongoing, with top-line data expected early in 2017

10/19/16


Notes

Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue.

The date indicated refers to the BioWorld Today issue in which the news item can be found.

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

 

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