Company |
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. |