Company |
Product |
Description |
Indication |
Status |
Date |
AUTOIMMUNE | |||||
Active Biotech AB (Lund, Sweden) |
Nerventra |
Laquinimod |
Relapsing-remitting multiple sclerosis |
CHMP issued a negative opinion, concluding that the risk-benefit profile was not favorable at this time |
1/27/14 |
CANCER | |||||
Arqule Inc. (Woburn, Mass.) |
Tivantinib |
Tyrosine kinase inhibitor |
Hepatocellular carcinoma |
A data monitoring committee recommended the phase III trial continue with a lower dose of 120 mg twice daily, which showed a reduced incidence of neutropenia; top-line phase III data in Asia of tivantinib and Tarceva in patients with advanced or metastatic nonsquamous non-small-cell lung cancer with wild-type epidermal growth factor receptor indicated that overall survival favored the treatment arm, compared to the erlotinib-only control arm; the result, however, was not statistically significant |
1/21/14 |
Biovest International Inc. (Minneapolis) |
BiovaxID |
Personalized cancer vaccine |
Non-Hodgkin's lymphoma |
EMA accepted the MAA |
1/15/14 |
Celgene International Sarl (Boudry, Switzerland; a unit of Celgene Corp.) |
Abraxane |
Nab-paclitaxel |
First-line metastatic adenocarcinoma of the pancreas or metastatic pancreatic cancer |
European Commission approved it for use in combination with gemcitabine |
1/8/14 |
Cell Therapeutics Inc. (Seattle) |
Pactrinib |
Oral JAK2/FLT3 inhibitor |
Relapsed acute myeloid leukemia |
Started an international cooperative group phase II trial of pacritinib in adult patients with mutations of the FLT3 gene; approximately 80 patients at sites in England and Wales will be enrolled and, if an encouraging response rate is observed, a pacritinib arm may be adopted in the first-line therapy study |
1/31/14 |
Celltrion Inc. (Incheon, South Korea) |
Herzuma |
Biosimilar monoclonal antibody trastuzumab, a biosimilar of Herceptin |
Advanced HER2-positive breast cancer and stomach cancer |
Received approval in Korea |
1/16/14 |
Cel-Sci Corp. (Vienna, Va.) |
Multikine |
Leukocyte interleukin, injection |
Head and neck cancer |
Received approval from Serbian regulators allowing it to start enrolling patients in its ongoing phase III head and neck cancer study |
1/24/14 |
Halozyme Therapeutics Inc. (San Diego) |
Mabthera |
Rituximab |
Non-Hodgkin's lymphoma |
CHMP recommended that the European Commission approve the subcutaneous formulation |
1/27/14 |
Helix Biopharma Corp. (Aurora, Ont.) |
L-DOS47 |
An immunoconjugate candidate |
Inoperable, locally advanced, recurrent or metastatic, nonsquamous stage IIIB/IV non-small-cell lung cancer |
Opened patient screening for the sixth dose level cohort of its ongoing phase I/II trial in Poland, following completion of the first treatment cycle of the three patients enrolled in the fifth cohort, in which therapy was well tolerated |
1/24/14 |
Novocure (Haifa, Israel) |
NovoTTF |
Anti-mitotic treatment |
Locally advanced and metastatic pancreatic adenocarcinoma |
The first patient was enrolled in the open-label, pilot EF-20 Study, designed to examine NovoTTF therapy plus gemcitabine as first-line therapy; the study plans to recruit 20 patients from centers in Spain, Germany and Switzerland |
1/27/14 |
Prima Biomed Ltd. (Sydney) |
Cvax |
An autologous dendritic cell-based product |
Epithelial ovarian cancer |
The amended CAN-004 phase II protocol was approved by Belgian regulators, and it will enroll a new cohort of 210 patients |
1/21/14 |
Seattle Genetics Inc. (Bothell, Wash.) and Takeda Pharmaceutical Co. Ltd. (Osaka, Japan) |
Adcetris |
Brentuximab vedotin |
CD30-positive relapsed or refractory Hodgkin’s lymphoma and anaplastic large cell lymphoma |
Received approval in Japan |
1/21/14 |
Synta Pharmaceuticals Corp. (Lexington, Mass.) |
Ganetespib |
Inhibits the heat shock protein 90 chaperone protein |
Metastatic ovarian cancer |
Launched GANNET53, a pan-European randomized trial evaluating its combination with paclitaxel vs. paclitaxel alone in more than 200 patients |
1/13/14 |
CARDIOVASCULAR | |||||
Aegerion Pharmaceuticals Inc. (Cambridge, Mass.) |
Juxtapid |
Lomitapide |
To reduce cholesterol in homozygous familial hypercholes-terolemia |
Mexican authorities approved it as an adjunct treatment to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce low-density lipoprotein cholesterol, total cholesterol, apolipoprotein B and non-high-density lipoprotein cholesterol |
1/22/14 |
INFECTION | |||||
Actelion Ltd. (Allschwil, Switzerland) |
Zavesca |
Miglustat |
Niemann-Pick Type C |
Company filed an application for approval in China |
1/8/14 |
Cell Medica Ltd. (London) |
Cytovir ADV |
T-cell immunotherapy |
Adenovirus infections following allogeneic hematopoietic stem cell transplant |
European Commission for Orphan Medicinal Products issued a positive opinion on the application |
1/8/14 |
Cytos Biotechnology AG (Zurich, Switzerland) |
gH1-Qbeta |
Vaccine |
Influenza |
Met the primary endpoint for immunogenicity (seroconversion based on hemaglutination inhibition titers) in a phase I trial in healthy Asian volunteers |
1/30/14 |
Gilead Sciences Inc. (Foster City, Calif.) |
Sovaldi; nucleotide analogue polyermase inhibitor |
Sofosbuvir 400 mg tablets; once-daily oral treatment |
Chronic hepatitis C virus |
European Commission granted marketing authorization |
1/21/14 |
Viiv Healthcare Ltd. (Middlesex, UK) |
Tivicay |
Dolutegravir; an integrase inhibitor |
HIV |
European Commission approved Tivicay for use in combination with other antiretroviral medicinal products to treat HIV-infected adults and adolescents older than 12 years |
1/22/14 |
MISCELLANEOUS | |||||
Biolinerx Ltd. (Jerusalem) |
BL-5010P |
A disposable, pen-like applicator containing BL-5010, an aqueous solution |
For nonsurgical removal of benign skin lesions |
Received approval in Germany |
1/22/14 |
Cytonet GmbH & Co. KG (Weinheim, Germany) |
N/A |
Liver cell therapy |
Urea cycle disorders |
EMA validated the MAA |
1/17/14 |
Finox Biotech AG (Burgdorf, Switzerland) |
Bemfola |
Follitropin alfa solution for injection in pre-filled pens |
Infertility |
CHMP adopted a positive opinion |
1/24/14 |
Keryx Biopharma-ceuticals Inc. (New York) and Japan Tobacco Inc. (Tokyo) |
Zerenex |
Ferric citrate coordination complex |
Hyperphos-phatemia in patients with chronic kidney disease |
Received approval in Japan |
1/21/14 |
NPS Pharmaceuticals Inc. (Bedminster, N.J.) |
Natpara |
Recombinant human parathyroid hormone |
Hypopara-thyroidism |
European Commission granted orphan drug designation |
1/6/14 |
Proteon Therapeutics Inc. (Waltham, Mass.) |
PRT-201 |
A recombinant elastase |
To prevent arterovenous access dysfunction in hemodialysis patients |
Received orphan drug designation in the European Union |
1/9/14 |
PTC Therapeutics Inc. (South Plainfield, N.J.) |
Ataluren |
A protein restoration therapy |
Duchenne muscular dystrophy |
CHMP adopted a negative opinion on the MAA for conditional approval |
1/27/14 |
Scioderm Inc. (Durham, N.C.) |
SD-101 |
Topical cream |
Epidermolysis bullosa |
European Commission granted orphan drug designation |
1/23/14 |
Tengion Inc. (Winston-Salem, N.C.) |
Neo-Kidney Augment |
Uses a patient's own cells to augment or replace renal function |
To prevent or delay dialysis and transplantation in patients with advanced chronic kidney disease (CKD) |
Neo-Kidney Augment was implanted in five CKD patients in an ongoing phase I trial in Sweden; the product is safe and well tolerated, and the company plans to expand enrollment in the trial to 15 patients |
1/14/14 |
Notes BLA = Biologics license application; CHMP = European Committee for Medicinal Products for Human Use; CMA = Continuous marketing application; EMA = European Medicines Agency; FDA = Food and Drug Administration; IND = Investigational new drug application; MAA = Marketing authorization application; NDA = New drug application; PDUFA = Prescription Drug User Fee Act; SPA = Special protocol assessment. 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. |