Company |
Product |
Description |
Indication |
Status |
Date |
AUTOIMMUNE | |||||
Allergan plc (Dublin) |
Aczone |
Dapsone gel; topical |
Acne |
FDA approved it in patients 12 years of age and older |
2/29/16 |
Eli Lilly and Co. (Indianapolis) |
Ixekizumab |
Targets IL-17A |
Moderate to severe plaque psoriasis |
EMA's Committee for Medicinal Products for Human Use issued a positive opinion for ixekizumab for the treatment of moderate to severe plaque psoriasis in adults in the European Union who are candidates for systemic therapy |
2/29/16 |
Genentech (unit of Roche AG, of Basel, Switzerland) |
Ocrevus |
Ocrelizumab |
Primary progressive multiple sclerosis |
FDA has designated Ocrevus a breakthrough therapy |
2/18/16 |
Pfizer Inc. (New York) |
Xeljanz XR |
JAK inhibitor; tofacitinib citrate extended-release 11-mg tablets |
Moderate to severe rheumatoid arthritis |
FDA approved it in patients who have had an inadequate response or intolerance to methotrexate |
2/25/16 |
Promius Pharma LLC (Princeton, N.J.; subsidiary of Dr. Reddy's Laboratories Ltd.) |
Sernivo |
Betamethasone dipropionate spray, 0.05% |
Mild to moderate plaque psoriasis |
Was granted FDA approval to treat patients 18 and older |
2/9/16 |
True North Therapeutics Inc. (South San Francisco) |
TNT009 |
A monoclonal antibody designed to target C1s |
Autoimmune hemolytic anemia |
EMA granted orphan designation for TNT009 |
2/26/16 |
CANCER | |||||
Adaptimmune Ltd. (Oxford, U.K.) |
T-cell therapy |
Targets NY-ESO |
Synovial sarcoma |
FDA has granted breakthrough therapy designation |
2/10/16 |
Astellas Pharma Inc. (Tokyo) and Medivation Inc. (South San Francisco) |
Xtandi |
Enzalutamide |
Metastatic castration-resistant prostate cancer |
FDA accepted for review a supplemental NDA seeking approval to market the therapy; the PDUFA date is Oct. 22. |
2/24/16 |
Astrazeneca plc (London) |
Tagrisso |
AZD9291, osimertinib |
Locally advanced or metastatic epidermal growth factor receptor T790M mutation-positive non-small-cell lung cancer |
European Commission has granted conditional marketing authorization |
2/4/16 |
Astrazeneca plc (London) and Acerta Pharma BV (Redwood City, Calif.) |
ACP-196 |
Acalabrutinib |
Chronic lymphocytic leukemia/small lymphocytic lymphoma, mantle cell lymphoma and lymphoplasmacytic lymphoma |
EMA's Committee for Orphan Medicinal Products adopted three positive opinions recommending it for designation as an orphan medicinal product |
2/26/16 |
Aveo Oncology Inc. (Cambridge, Mass.) |
Tivozanib |
VEGF tyrosine kinase inhibitor |
Advanced renal cell carcinoma |
A registration dossier seeking to obtain marketing authorization has been accepted by the Ministry of Health of the Russian Federation |
2/23/16 |
Bristol-Myers Squibb Co. (New York) |
Empliciti |
Elotuzumab; humanized, IgG1 monoclonal antibody that specifically targets the SLAMF7 protein |
Multiple myeloma |
The CHMP recommended approval of Empliciti |
2/1/16 |
Bristol-Myers Squibb Co. (New York) |
Opdivo |
Nivolumab; anti-PD-1 drug |
Locally advanced or metastatic non-small-cell lung cancer, and advanced renal cell carcinoma |
EMA's Committee for Medicinal Products for Human Use recommended the approval of Opdivo for two new indications: adults with locally advanced or metastatic non-small-cell lung cancer after prior chemotherapy, and adults with advanced renal cell carcinoma after prior therapy; Opdivo is already approved in Europe for advanced melanoma and previously treated advanced squamous NSCLC. |
2/29/16 |
Epizyme Inc. (Cambridge, Mass.) |
Tazemetostat |
EZH2 inhibitor |
Malignant rhabdoid tumors |
FDA designated it an orphan drug |
2/9/16 |
Exelixis Inc. (South San Francisco) |
Cabozantinib |
Multi-tyrosine kinase inhibitor |
Advanced renal cell carcinoma |
EMA accepted for review the MAA |
2/1/16 |
Genentech (unit of Roche AG; Basel, Switzerland) |
Gazyva |
Obinutuzumab |
Follicular lymphoma |
FDA approved Gazyva plus bendamustine chemotherapy followed by Gazyva alone as a new treatment for people with follicular lymphoma who did not respond to a Rituxan (rituximab)-containing regimen, or whose follicular lymphoma returned after such treatment |
2/29/16 |
Immunomedics Inc. (Morris Plains, N.J.) |
IMMU-132 |
Antibody that targets the TROP-2 receptor |
Triple-negative breast cancer |
Received breakthrough therapy designation from the FDA |
2/8/16 |
Kremers Urban Pharmaceuticals Inc. (subsidiary of Lannett Co. Inc.; Philadelphia) |
Temozolomide |
Capsules 5 mg, 20 mg, 100 mg, 140 mg, 180 mg and 250 mg; therapeutic equivalent to Temodar (Merck & Co. Inc.) |
Cancer |
Received FDA approval of the aNDA |
2/12/16 |
Medimmune and Astrazeneca plc (London) |
MEDI4736 |
Durvalumab; human monoclonal antibody directed against programmed death ligand-1 |
PD-L1 positive inoperable or metastatic urothelial bladder cancer |
FDA granted breakthrough therapy |
2/18/16 |
Merck & Co. Inc. (Kenilworth, N.J.) |
Emend |
Fosaprepitant dimeglumine; substance P/neurokinin-1 receptor antagonist |
To prevent delayed nausea and vomiting |
FDA approved an sNDA for single-dose Emend in combination with other anti-emetic medicines for adults receiving initial and repeat courses of moderately emetogenic chemotherapy |
2/5/16 |
Novartis AG (Basel, Switzerland) |
PKC412 |
Midostaurin |
Acute myeloid leukemia |
Gained FDA breakthrough therapy status |
2/22/16 |
Novartis AG (Basel, Switzerland) |
Afinitor |
Everolimus tablets |
Progressive, well-differentiated, nonfunctional neuroendocrine tumors of gastrointestinal or lung origin that are unresectable, locally advanced or metastatic |
FDA approved it |
2/29/16 |
Oasmia Pharmaceutical AB (Uppsala, Sweden) |
Apealea |
Paclical; a new version of paclitaxel based on the excipient platform XR-17, which forms Cremophor-free micellar nanoparticles with paclitaxel |
Epithelial ovarian cancer |
Submitted a marketing authorization application (MAA) to the EMA for Apealea combined with carboplatin |
2/9/16 |
Pfizer Inc. (New York) |
Ibrance |
Palbociclib |
Metastatic breast cancer |
Received expanded approval from the FDA for hormone receptor-positive, human epidermal growth factor receptor 2-negative advanced or metastatic breast cancer to be combined with fulvestrant in women with disease progression following endocrine therapy |
2/23/16 |
Roche AG (Basel, Switzerland) |
Cotellic |
Cobimetinib |
Unresectable or metastatic melanoma |
Health Canada approved Cotellic in combination with Zelboraf (vemurafenib) |
2/26/16 |
Sandoz Inc. (Holzkirchen, Germany) |
Biosimilar |
To Amgen's Neulasta (pegfilgrastim, a long-acting recombinant human granulocyte colony-stimulating factor |
Chemo-induced neutropenia |
EMA accepted the MAA |
2/12/16 |
Spectrum Pharmaceuticals Inc. (Henderson, Nev.) |
Eoquin |
Apaziquone for intravesical instillation |
Cancer |
FDA accepted the NDA for review and set a target action date of Dec. 11 |
2/22/16 |
Takeda Pharmaceutical Co. Ltd. (Osaka, Japan) |
Ixazomib |
Oral proteasome inhibitor |
Relapsed and/or refractory multiple myeloma |
Health Canada accepted the new drug submission for ixazomib for priority review |
2/17/16 |
Tapimmune Inc. (Jacksonville, Fla.) |
TPIV200 |
Multiple-epitope folate receptor alpha peptide vaccine |
Ovarian cancer |
FDA granted fast track designation |
2/4/16 |
CARDIOVASCULAR | |||||
Actelion Ltd. (Allschwil, Switzerland) |
Uptravi |
Selexipag |
Pulmonary arterial hypertension |
Added a positive EMA opinion to the FDA and Health Canada approvals |
2/1/16 |
Astrazeneca plc (London) |
Brilique |
Ticagrelor |
Heart attack |
European Commission granted marketing authorization |
2/22/16 |
Baxalta Inc. (Bannockburn, Ill.) |
Adynovate |
Antihemophilic factor (recombinant), pegylated |
Hemophilia A |
Submitted supplemental BLAs to the FDA for the use of Adynovate to treat children under the age of 12 with hemophilia A and for use in surgical settings |
2/29/16 |
Eagle Pharmaceuticals Inc. (Woodcliff Lake, N.J.) |
Ryanodex |
Dantrolene sodium |
Exertional heat stroke |
FDA has granted fast track designation |
2/2/16 |
Les Laboratoires Servier SAS (Neuilly-sur-Seine, France) |
Viacoram |
Perindopril arginine/amlodipine besylate |
Mild to moderate hypertension |
Its Canadian unit reported Health Canada approval as a first-line treatment |
2/1/16 |
Merck & Co. Inc. (Kenilworth, N.J.) |
Zetia and Vytorin |
Ezetimibe; and ezetimibe and simvastatin |
To lower cholesterol |
FDA issued a complete response letter to the supplemental NDA |
2/17/16 |
Nuvox Pharma LLC (Tucson, Ariz.) |
NVX-508 |
Designed to deliver oxygen to the diseased tissues |
Sickle cell disease |
Secured orphan designation from the FDA for vaso-occlusive crisis and acute chest syndrome |
2/17/16 |
Pharming Group NV (Leiden, the Netherlands) |
Ruconest |
Conestat alpha |
Hereditary angioedema |
EMA's Committee for Medicinal Products for Human Use issued a positive opinion on the company's request to add the treatment of hereditary angioedema (HAE) attacks in adolescents with HAE to the Ruconest (conestat alpha) label and to remove the requirements for rabbit IgE testing that formed part of the EU label; the FDA's 2014 approval of the recombinant human C1 esterase inhibitor to treat acute HAE attacks did not require rabbit IgE testing |
2/29/16 |
Portola Pharmaceuticals Inc. (South San Francisco) |
Andexanet alfa |
To reverse anticoagulant activity of direct and indirect factor Xa inhibitors |
To reverse anticoagulation |
FDA accepted for priority review the BLA |
2/19/16 |
Remedy Pharmaceuticals Inc. (New York) |
Cirara |
A high-affinity inhibitor of Sur1-Trpm4 channels |
Acute subarachnoid hemorrhage |
FDA granted orphan status |
2/17/16 |
Swedish Orphan Biovitrum AB (Stockholm) and Biogen Inc. (Cambridge, Mass.) |
Alprolix |
A recombinant factor IX Fc fusion protein therapy |
Hemophilia B |
Received a positive opinion from the EMA's Committee for Medicinal Products for Human Use, recommending that marketing authorization be granted |
2/29/16 |
CENTRAL NERVOUS SYSTEM | |||||
Acadia Pharmaceuticals Inc. (San Diego) |
Nuplazid |
Pimavanserin |
Psychosis associated with Parkinson's disease |
A March 29 meeting of the FDA's Psychopharmacologic Drugs Advisory Committee will review data included in the company's new drug application (NDA) |
2/2/16 |
Adapta Pharma Ltd. (Dublin) |
Narcan |
Naloxone hydrochloride nasal spray |
Opioid overdose |
Now available in the U.S. for the emergency treatment of known or suspected opioid overdose, as manifested by respiratory and/or central nervous system depression |
2/29/16 |
Allergan plc (Dublin) |
GLYX-13 |
Rapastinel; intravenous NMDA receptor partial agonist |
Major depressive disorder |
Received breakthrough therapy designation from the FDA |
2/1/16 |
Amarantus Bioscience Holdings Inc. (San Francisco) |
Eltoprazine |
A 5HT1a/1b partial agonist small-molecule drug |
Parkinson's disease levodopa-induced dyskinesia |
Received orphan drug designation from the FDA |
2/11/16 |
Asterias Biotherapeutics Inc. (Fremont, Calif.) |
AST-OPC1 |
An oligodendrocyte progenitor population derived from human embryonic stem cells |
Acute spinal cord injury |
FDA granted orphan designation |
2/5/16 |
Dr. Reddy's Laboratories Ltd. (Hyderabad, India) |
Zembrace Symtouch |
Sumatriptan succinate injection; a drug-device combination product |
Acute migraine episodes, with or without aura |
FDA approved it in adults who are inadequately managed with existing treatment regimens |
2/1/16 |
H. Lundbeck A/S (Valby, Denmark) and Otsuka Pharmaceutical Co. Ltd. (Princeton, N.J.) |
Rexulti |
Brexipiprazole |
Schizophrenia |
FDA accepted for review a supplemental new drug application (sNDA) for the proposed labeling update for the maintenance treatment of adults |
2/9/16 |
Kempharm Inc. (Coralville, Iowa) |
KP201/APAP |
Abuse-deterrent immediate-release hydrocodone combination candidate |
Acute pain |
FDA accepted and granted priority review to the NDA |
2/11/16 |
Lannett Co. Inc. (Philadelphia) |
Sumatriptan |
Nasal spray USP; therapeutic equivalent to Imitrex nasal spray (Glaxosmithkline plc; London) |
Migraine |
FDA approved the abbreviated NDA |
2/23/16 |
Optinose A/S (Oslo, Norway) and Avanir Pharmaceuticals Inc. (Aliso Viejo, Calif.) |
Onzetra Xsail |
Sumatriptan nasal powder; formerly AVP-825 |
Migraine with or without aura |
FDA approved it for acute treatment in adults |
2/1/16 |
Otsuka Pharmaceuticals Co. Ltd. (Tokyo) and H. Lundbeck A/S (Valby, Denmark) |
Abilify Maintena |
Aripiprazole |
Schizophrenia |
CHMP released a positive opinion on updating the product's label for schizophrenia who have acutely relapsed and is intended as maintenance treatment of patients who have been stabilized with oral aripiprazole |
2/4/16 |
Retrophin Inc. (San Diego) |
RE-024 |
Phosphopanto-thenate replacement therapy |
Pantothenate kinase-associated neurodegeneration |
European Commission granted orphan drug designation |
2/25/16 |
Shire plc (Dublin) and Shionogi & Co. Ltd. (Osaka, Japan) |
S-877503 |
Guanfacine hydrochloride prolonged release tablets |
Attention deficit hyperactivity disorder |
Submitted an NDA in Japan |
2/12/16 |
Takeda Pharmaceutical Co. Ltd. (Osaka, Japan) |
Brintellix |
Vortioxetine |
Major depressive disorder |
FDA's Psychopharmacologic Drugs Advisory Committee voted 8-2 Wednesday in support of an expanded label for Takeda's Brintellix that would recognize cognitive dysfunction in adults with major depressive disorder |
2/4/16 |
Titan Pharmaceuticals Inc. and Braeburn Pharmaceuticals Inc. |
Probuphine |
Subdermal buprenorphine implant |
Opioid dependence |
FDA extended by three months the PDUFA date, which is now set for May 27; the agency requested additional changes to the risk evaluation and mitigation strategy portion of the NDA |
2/23/16 |
Trevena Inc. (King of Prussia, Pa.) |
TRV130 |
Oliceridine |
Moderate-to-severe acute pain |
FDA designated intravenous oliceridine a breakthrough therapy |
2/23/16 |
UCB SA (Brussels, Belgium) |
Briviact |
Brivaracetam; high-affinity synaptic vesicle protein 2A (SV2A) ligand |
Partial-onset seizures in epilepsy |
FDA approved it as adjunctive therapy |
2/19/16 |
Zynerba Pharmaceuticals Inc. (Devon, Pa.) |
ZYN002 |
Cannabidiol gel |
Fragile X syndrome |
FDA granted orphan drug designation to ZYN002 |
2/26/16 |
DIABETES | |||||
Sanofi SA (Paris) and Zealand Pharma AS (Copenhagen) |
Basal insulin glargine and lixisenatide |
Fixed-ratio combination of basal insulin glargine 100 U/mL and lixisenatide |
Type 2 diabetes |
FDA accepted its NDA for a fixed-ratio combination of basal insulin glargine 100 U/mL and lixisenatide, a GLP-1 receptor agonist |
2/23/16 |
GASTROINTESTINAL | |||||
Synergy Pharmaceuticals Inc. (New York) |
Plecanatide |
A uroguanylin analogue |
Chronic idiopathic constipation |
Filed an NDA with the FDA seeking approval in CIC |
2/1/16 |
INFECTION | |||||
Abbvie Inc. (North Chicago) |
Viekirax + Exviera |
Viekirax (ombitasvir/paritaprevir/ritonavir tablets) + Exviera (dasabuvir) without ribavirin |
Chronic hepatitis C virus genotype 1b with compensated cirrhosis |
EMA's Committee for Medicinal Products for Human Use issued a positive opinion |
2/29/16 |
Allergan plc (Dublin) |
Avycaz |
Ceftazidime and avibacatam |
Complicated intra-abdominal infections |
FDA accepted for filing the company's sNDA, which will add data to the current label from two phase III trials evaluating the safety and efficacy in combination with metronidazole for the treatment of cIAI, including patients with infections due to ceftazidime-nonsusceptible pathogens |
2/25/16 |
Bristol-Myers Squibb Co. (New York) |
Daklinza |
Daclatasvir; an NS5A replication complex inhibitor |
Chronic hepatitis C virus with HIV-1 co-infection |
Approved by the FDA in combination with Sovaldi (sofosbuvir, Gilead Sciences Inc.), with or without ribavirin, in genotypes 1 and 3 |
2/8/16 |
Cidara Therapeutics Inc. (San Diego) |
CD101 IV |
Novel echinocandin |
Candidemia and invasive candidiasis |
FDA granted orphan drug designation |
2/11/16 |
Cipher Pharmaceuticals Inc. (Mississauga, Ontario) |
Sitavig |
Acyclovir mucoadhesive buccal tablets |
Recurrent herpes labialis |
Its new drug submission was accepted for review by Health Canada |
2/17/16 |
Conatus Pharmaceuticals Inc. (San Diego) |
Emricasan |
First-in-class, orally-active pan-caspase inhibitor |
Liver cirrhosis caused by nonalcoholic steatohepatitis |
FDA designated emricasan a fast track drug |
2/4/16 |
Gilead Sciences Inc. (Foster City, Calif.) |
Truvada |
Emtricitabine 200 mg/tenofovir disoproxil fumarate 300 mg |
HIV-1 infection |
Type II variation application of Truvada, in combination with safer sex practices to reduce the risk of sexually acquired HIV in uninfected adults at high risk, has been fully validated and is now under evaluation by the EMA |
2/2/16 |
Gilead Sciences Inc. (Foster City, Calif.) |
Harvoni |
Ledipasvir/sofosbuvir |
Chronic hepatitis C virus |
FDA approved additional indications for Harvoni: in combination with ribavirin for 12 weeks in HCV genotype 1- or 4-infected liver transplant recipients without cirrhosis or with compensated cirrhosis, and for HCV genotype 1-infected patients with decompensated cirrhosis, including those who have undergone liver transplantation |
2/17/16 |
Gilead Sciences Inc. (Foster City, Calif.) |
TAF |
Tenofovir alafenamide; once-daily treatment |
Chronic hepatitis B virus infection |
EMA validated and is now assessing the MAA for TAF |
2/26/16 |
Gilead Sciences Inc. (Foster City, Calif.) |
Descovy |
Emtricitabine and tenofovir alafenamide 200/10 mg and 200/25 mg; F/TAF |
HIV-1 infection |
EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on the MAA for the two doses |
2/29/16 |
Immune Response Biopharma Inc. (New York) |
Remune HIV-1 Immunogen |
HIV vaccine |
HIV-1 infection |
Received a complete response letter |
2/17/16 |
Merck & Co. Inc. (Kenilworth, N.J.) |
Zepatier |
Elbasvir and grazoprevir |
Hepatitis C virus |
FDA approved it for genotype 1 and 4 infections |
2/1/16 |
Merck & Co. Inc. (Kenilworth, N.J.) |
Zepatier |
Elbasvir and grazoprevir |
Chronic hepatitis C virus genotypes 1, 3 or 4 |
Received accelerated approval in Canada |
2/4/16 |
Nimbus Therapeutics Inc. (Cambridge, Mass.) |
NDI-010976 |
Liver-targeted allosteric inhibitor of acetyl-CoA carboxylase |
Non-alcoholic steatohepatitis |
FDA granted fast track status |
2/3/16 |
INFLAMMATORY | |||||
Dr. Reddy's Laboratories Ltd. (Hyderabad, India) |
Zenavod |
Doxycycline capsules; a tetracycline-class drug |
Inflammatory lesions of rosacea |
FDA has given tentative approval for Zenavod, 40 mg, with a stay of final approval for up to 30 months pending a patent infringement process under the Drug Price Competition and Patent Term Restoration Act |
2/2/16 |
MISCELLANEOUS | |||||
AB Science SA (Paris) |
Masitinib |
Tyrosine-kinase inhibitor |
Severe systemic mastocytosis |
Following a pre-submission meeting with the EMA, the MAA has been accepted for review |
2/2/16 |
Amag Pharmaceuticals Inc. (Waltham, Mass.) |
Makena |
Single-dose, preservative-free formulation; hydroxyprogesterone caproate injection |
To reduce the risk of preterm birth |
FDA approved it as an expansion of the Makena product line, indicated to reduce the risk of preterm birth in women who are pregnant with one baby and who have spontaneously delivered a preterm baby in the past |
2/24/16 |
Amarantus Bioscience Holdings Inc. (San Francisco) |
MANF |
Mesencephalic-astrocyte-derived neurotrophic factor |
Retinitis pigmentosa |
Amarantus requested rare pediatric disease designation from the FDA; it was previously granted orphan drug designation |
2/8/16 |
Atara Biotherapeutics Inc. (South San Francisco) |
T-lymphocyte |
Off-the-shelf allogeneic cellular therapy, activated against Epstein-Barr Virus |
Post-transplant lymphoproliferative disorder following hematopoietic cell transplant or solid organ transplant |
FDA granted orphan drug designation |
2/9/16 |
Bellicum Pharmaceuticals Inc. (Houston) |
BPX-501 |
Genetically modified T cells |
Immunodeficiency and graft-vs.-host disease |
FDA has granted orphan drug designation for the combination of BPX-501 and activator agent rimiducid |
2/23/16 |
Catalyst Pharmaceuticals Inc. (Coral Gables, Fla.) |
Firdapse |
Amifampridine phosphate |
Lambert-Eaton myasthenic syndrome and congenital myasthenic syndromes |
FDA issued a refuse to file letter for the NDA |
2/18/16 |
CTI Biopharma Corp. (Seattle) |
Pacritinib |
An oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R |
Primary myelofibrosis and post-polycythemia vera myelofibrosis (MF) and post-essential thrombocythemia MF |
The FDA placed a partial clinical hold, citing fatal and life-threatening safety issues in patients treated with the drug vs. those provided best available therapy other than JAK inhibitors in the control arm of PERSIST-1, a phase III study of primary myelofibrosis; the hold also impacts PERSIST-2, an ongoing study extending pacritinib treatment to patients with post-polycythemia vera MF and post-essential thrombocythemia MF; CTI withdrew its NDA after the FDA expanded a partial clinical hold on trials of the drug to a full hold |
2/9/16; 2/11/16 |
Esteve SA (Barcelona, Spain) |
EGT-201 and EGT-301 |
Gene therapies; EGT-301 consists of an adeno-associated viral vector of serotype 9 containing the human iduronate-2-sulfatase transgene |
Sanfilippo B syndrome and Hunter syndrome |
Added two new investigational gene therapies, both developed in collaboration with a group at the Universitat Autònoma de Barcelona; the FDA has granted orphan drug designation to EGT-301 for the treatment of Hunter syndrome or mucopolysaccharidosis II |
2/26/16 |
LFB SA (Les Ulis, France) |
Fibclot |
Human fibrinogen |
Congenital fibrinogen deficiency |
Received the first three European marketing authorizations in Germany, Denmark and Hungary |
2/23/16 |
Onl Therapeutics Inc. (Ann Arbor, Mich.) |
ONL1204 |
A first-in-class small molecule peptide |
Retinal diseases and conditions |
FDA granted orphan drug designation |
2/12/16 |
PTC Therapeutics Inc. (South Plainfield, N.J.) |
Translarna |
Ataluren |
Duchenne muscular dystrophy |
FDA refused to accept the NDA with a refuse to file (RTF) letter, viewing both the phase IIb and phase III ACT DMD trials as negative and not providing substantial evidence of effectiveness |
2/24/16 |
Sarepta Therapeutics Inc. (Cambridge, Mass.) |
Exondys 51 |
Eteplirsen |
Duchenne muscular dystrophy |
The new PDUFA date for eteplirsen has been set for May 26, a month after the originally scheduled action date; a meeting of the FDA's Peripheral and Central Nervous System advisory committee, which is slated to consider the company's applications, was postponed from Jan. 22 for inclement weather; that meeting is rescheduled for April 25 |
2/29/16 |
Tarix Orphan LLC (Cambridge, Mass.) |
TXA127 |
A pharmaceutical grade formulation of the naturally occurring peptide angiotensin (1-7) |
Laminin-deficient congenital muscular dystrophy |
Gained FDA orphan status |
2/18/16 |
RESPIRATORY | |||||
Actavis Laboratories FL Inc. |
Generic Orenitram |
Treprostinil extended-release tablets (United Therapeutics Corp.) |
Pulmonary arterial hypertension |
Actavis submitted an abbreviated NDA (ANDA) to the FDA |
2/22/16 |
Alk-Abello A/S (Copenhagen) and Merck Sharp & Dohme Corp. (subsidiary of Merck & Co.; Kenilworth, N.J.) |
SLIT-tablet |
House dust mite sublingual allergy immunotherapy tablet |
House dust mite allergy |
Submitted a BLA |
2/11/16 |
Arch Biopartners Inc. (Toronto) |
AB569 |
A potential new treatment for respiratory Pseudomonas aeruginosa infections |
Pseudomonas aeruginosa pulmonary infections in cystic fibrosis |
Submitted an application for orphan drug designation to the EMA |
2/4/16 |
Biota Pharmaceuticals Inc. (Atlanta) |
BTA585 |
Antiviral compound |
Respiratory syncytial virus infection |
FDA granted fast track designation |
2/17/16 |
Mylan NV (Hertfordshire, U.K.) |
Fluticasone propionate 100 mcg, 250 mcg, 500 mcg and salmeterol 50 mcg inhalation powder |
Generic version of Glaxosmithkline plc's Advair Diskus |
Asthma and maintenance treatment for chronic obstructive pulmonary disease |
FDA accepted for filing the abbreviated new drug application (ANDA) |
2/22/16 |
Nivalis Therapeutics Inc. (Boulder, Colo.) |
N91115 |
A stabilizer of the cystic fibrosis transmembrane conductance regulator protein; S-nitrosoglutathione reductase inhibitor |
Cystic fibrosis |
FDA granted fast track designation |
2/22/16 |
Proteostasis Therapeutics Inc. (Cambridge, Mass.) |
PTI-428 |
An orally bioavailable CF transmembrane conductance regulator modulator belonging to the amplifier class |
Cystic fibrosis |
Received fast track designation from the FDA |
2/2/16 |
Vertex Pharmaceuticals Inc. (Boston) |
Kalydeco |
Ivacaftor |
Cystic fibrosis |
Received a complete response letter for its sNDA |
2/8/16 |
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. |