Top-Line Results for Investigational Osteoporosis Drug

Amgen and UCB announced top-line results from the STRUCTURE trial evaluating romosozumab in women with postmenopausal osteoporosis at high risk for fracture previously treated with bisphosphonate therapy.

Rapid Reversal Seen With Idarucizumab in Pradaxa-Treated Patients

Boehringer Ingelheim presented positive results of its Phase 3 RE-VERSE AD study for idarucizumab as a reversal agent for the anticoagulant effect of Pradaxa (dabigatran) in patients needing emergency surgery or for life-threatening or uncontrolled bleeding events.

Elotuzumab BLA Given Priority Review for Multiple Myeloma

The Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for Empliciti (elotuzumab, Bristol-Myers Squibb/AbbVie) for the treatment of multiple myeloma as combination therapy in patients who have received one or more prior therapies.

Biologic Tx BLA Accepted for Bladder Cancer

The Food and Drug Administration (FDA) has accepted for filing the Biologics License Application (BLA) for MCNA (Telesta) as a therapeutic alternative to surgery in high risk non-muscle invasive bladder cancer patients who are refractory to or relapsing from front line therapy.

Letairis/Tadalafil Combo Demonstrates Efficacy for Pulmonary HTN

Gilead Sciences announced positive results from the AMBITION study, a Phase 3/4 trial of first-line combination therapy with Letairis (ambrisentan) and tadalafil in patients with WHO/NYHA functional class II and III pulmonary arterial hypertension (PAH).

Novel Antibiotic Fast Tracked for Bacterial Vaginosis

The Food and Drug Administration (FDA) has granted SYM-1219 (secnidazole, Symbiomix) Fast Track designation for the treatment of bacterial vaginosis (BV).

FDA Accepts Eteplirsen NDA for Duchenne Muscular Dystrophy

The Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for eteplirsen (Sarepta) for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.

Novel Drug Granted Orphan Designation for Rare Musculoskeletal Disorder

The Food and Drug Administration (FDA) has granted AT001 (Audentes) Orphan Drug designation for the treatment of X-Linked Myotubular Myopathy (XLMTM).

Positive Results for First Long-Acting Injection-Free GLP-1 Agonist

Intarcia Therapeutics has announced that the Phase 3 trial of ITCA 650 (exenatide) demonstrated positive top-line results in reducing HbA1c in patients with type 2 diabetes following a year of treatment.

Anti-PDL1 Immunotherapy Demonstrates Efficacy in Phase 2 NSCLC Study

Genentech announced that the Phase 2 study of atezolizumab met its primary endpoint and shrank tumors in people with locally advanced or metastatic non-small cell lung cancer (NSCLC) whose disease expressed PD-L1 (Programmed Death Ligand-1).

BromSite NDA Accepted for Post-Cataract Surgery

The Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for BromSite (0.075% bromfenac; InSite Vision) for the treatment of inflammation and prevention of ocular pain post-cataract surgery.

FDA Accepts Keytruda sBLA for First-Line Melanoma Indication

The Food and Drug Administration (FDA) has accepted for review the sBLA for Keytruda (pembrolizumab, Merck) for the first-line treatment of unresectable or metastatic melanoma patients.

New Microbiome Therapy Granted Orphan Drug Designation for Recurrent CDI

New Microbiome Therapy Granted Orphan Drug Designation for Recurrent CDI

The Food and Drug Administration (FDA) has granted Orphan Drug designation to SER-109 (Seres Therapeutics) for the prevention of recurrent Clostridium difficile infection (CDI) in adults.

Adjunctive Fycompa Demonstrates Efficacy in PGTC Seizures

The Phase 3 trial for Fycompa (perampanel, Eisai) demonstrated statistically significant results when used as adjunctive therapy in the treatment of primary generalized tonic-clonic (PGTC) seizures in patients 12 years and older.

Cabozantinib Receives Breakthrough Therapy Designation for Renal Cancer

The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to cabozantinib (Exelixis) for the treatment of patients with advanced renal cell carcinoma (RCC) who have received one prior therapy.

Investigational Agent for Huntington's Disease to Be Reviewed by FDA

The Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for SD-809 (deutetrabenazine, Teva) for the treatment of chorea associated with Huntington's disease (HD).

Venetoclax Demonstrates Efficacy in Phase 2 CLL Study

The Phase 2 trial of venetoclax (AbbVie) has met its primary endpoint in patients with relapsed/refractory or previously untreated chronic lymphocytic leukemia (CLL) with 17p deletion.

Brintellix sNDA Accepted for Labeling Update on Cognitive Effects

The Food and Drug Administration (FDA) has accepted for review the supplemental New Drug Application (sNDA) for Brintellix (vortioxetine, Takeda and Lundbeck) to add to the current labeling its effects on certain aspects of cognitive function in adults with Major Depressive Disorder (MDD).

Recombinant IVIG granted Orphan Drug Designation for CIDP

The Food and Drug Administration (FDA) has granted GL-2045 (recombinant Intravenous Immune Globulin, Gliknik/Pfizer) Orphan Drug designation for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP).

Kynamro Demonstrates Efficacy in Heterozygous Familial Hypercholesterolemia

The Phase 3 study of Kynamro (mipomersen sodium; Isis) met its primary endpoint in patients with severe heterozygous familial hypercholesterolemia (severe HeFH).

Positive Results for Third Naldemedine Phase 3 Study in OIC

The Phase 3 study (COMPOSE II) for oral naldemedine (Shionogi) met its primary endpoints for the treatment of opioid-induced constipation (OIC) in adult patients with chronic non-cancer pain.

Adjunctive Telotristat Demonstrates Efficacy in Carcinoid Syndrome

Lexicon announced top-line results from the Phase 3 study, TELESTAR, which evaluated oral telotristat etiprate in cancer patients with carcinoid syndrome that is not adequately controlled by the current standard of care.

EDO-S101 NDA Accepted for Relapsed or Refractory Malignancies

The New Drug Application (NDA) for EDO-S101 (Mundipharma) has been accepted by the Food and Drug Administration (FDA) for the treatment of relapsed or refractory hematologic malignancies and solid tumors.

Positive Phase 3 Results for Plecanatide in Chronic Idiopathic Constipation

Synergy announced positive top-line results from the second Phase 3 trial of plecanatide 3mg and 6mg doses in adult patients with chronic idiopathic constipation (CIC).

Dactinomycin Nanoparticle Formulation Receives Orphan Drug Designation

The Food and Drug Administration (FDA) has granted Orphan Drug designation to dactinomycin nanoparticle formulation (NanoSmart) for the treatment of Ewing's sarcoma.

Amphetamine XR-ODT NDA Resubmitted for ADHD Treatment

The New Drug Application (NDA) for NT-0202 (amphetamine XR-ODT, Neos) has been resubmitted to the Food and Drug Administration (FDA) for the treatment of attention-deficit/hyperactivity disorder (ADHD).

Rayaldee NDA Accepted for SHPT in Stage 3 or 4 CKD and Vitamin D Deficiency

The Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for Rayaldee (calcifediol modified-release capsules, Opko) for the prevention and treatment of secondary hyperparathyroidism (SHPT) in patients with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency.

Inj Combo Diabetes Drug: Phase 3 Results Announced

Sanofi announced that the Phase 3 trial of the fixed-ratio combination of insulin glargine 100 units/mL and lixisenatide met its primary endpoint in patients with type 2 diabetes treated with metformin.

BLA for Novel Hemophilia A Therapy Accepted for Review

The Food and Drug Administration (FDA) has accepted CSL Behring's Biologics License Application (BLA) for its novel investigational recombinant factor VIII single-chain (rVIII-SingleChain) for the treatment of hemophilia A.

Lenvatinib Granted Breakthrough Therapy for Advanced or Metastatic Renal Cell Carcinoma

The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for lenvatinib for use in patients with advanced or metastatic renal cell carcinoma (RCC) who were previously treated with a vascular endothelial growth factor (VEGF)-targeted therapy.

FDA Accepts Grazoprevir/Elbasvir NDA for Chronic HCV

The Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for grazoprevir/elbasvir (Merck) for the treatment of adult patients infected with chronic hepatitis C virus (HCV) genotypes (GT) 1, 4, or 6.

Evacetrapib Phase 3 Study to Continue in High-Risk ASCVD

Eli Lilly announced the continuation of the Phase 3 trial of evacetrapib in participants with high-risk atherosclerotic cardiovascular disease (ASCVD).

Intranasal Narcan NDA Submitted for Opioid Overdose

Adapt Pharma announced that it has submitted a New Drug Application (NDA) to the Food and Drug Administration (FDA) for Narcan (naloxone) Nasal Spray for the treatment of opioid overdose.

Olysio sNDA Submitted for Labeling Update

Janssen Therapeutics announced the submission of a supplemental New Drug Application (sNDA) to the Food and Drug Administration (FDA) to update the label for once-daily, all-oral Olysio (simeprevir) in combination with sofosbuvir for adults with genotype 1 chronic hepatitis C (CHC) infection with or without cirrhosis.

OMS721 Granted Fast Track Designation for Atypical Hemolytic Uremic Syndrome

The FDA has granted Fast Track designation to OMS721 (Omeros) for the treatment of patients with atypical hemolytic uremic syndrome (aHUS).

Kyprolis sNDA Submitted for Expanded Relapsed Multiple Myeloma Indication

The sNDA for Kyprolis (carfilzomib, Amgen) was submitted to the FDA for the treatment of patients with relapsed multiple myeloma, who has received at least one prior therapy.

FDA Accepts Ready-to-Use Bivalirudin NDA for Filing

The FDA has accepted for filing the New Drug Application (NDA) for ready-to-use (RTU) bivalirudin (Eagle) for the treatment of patients undergoing percutaneous coronary intervention (PCI) with use of glycoprotein IIb/IIa inhibitor; undergoing PCI with, or at risk of heparin-induced thrombocytopenia and thrombosis syndrome; or with unstable angina undergoing percutaneous transluminal coronary angioplasty (PTCA).

Delayed-Start Method Shows Early Treatment With Solanezumab Beneficial

Eli Lilly and Company announced results from the Phase 3 study evaluating whether the treatment effect of solanezumab was preserved within a pre-specified amount in patients with mild Alzheimer's disease who received solanezumab earlier in the disease compared to patients who began treatment at a later point.

Single Tablet HIV-1 Infection Regimen Non-Inferior to TDF-Based Regimens

Gilead Sciences announced results from Study 109, a Phase 3 study evaluating once daily single tablet regimen (STR) of elvitegravir 150mg, cobicistat 150mg, emtricitabine 200mg, and tenofovir alafenamide 10mg (E/C/F/TAF) in adults switching from tenofovir disoproxil fumarate (TDF) containing regimens.

First Study Tests New Combo Tx for HCV/HIV Co-Infection

Researchers from the University of California, San Diego School of Medicine reported a new combination therapy that effectively treated hepatitis C virus (HCV) in patients co-infected with HIV.

Osmolex ER Awarded Orphan Drug Status for Levodopa-Induced Dyskinesia in Parkinson's Disease

The Food and Drug Administration (FDA) has granted Orphan Drug designation to Osmolex ER (amantadine HCl extended release tablets; Osmotica) for the treatment of levodopa induced dyskinesia (LID) for patients with Parkinson's disease.

BMS-663068 Receives Breakthrough Therapy Designation for HIV-Infection

The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to BMS-663068 (Bristol-Myers Squibb), when used in combination with other antiretroviral (ARV) agents for the treatment of HIV-1 infection in heavily treatment-experienced adult patients.

Melphalan Designated Orphan Drug for Bile Duct Cancer

The Food and Drug Administration (FDA) has granted Orphan Drug designation to melphalan (Delcath Systems) for the treatment of cholangiocarcinoma.

Cometriq Demonstrates Efficacy in Renal Cell Carcinoma Study

Exelixis announced positive top-line results from the primary analysis of METEOR, a Phase 3 trial comparing Cometriq (cabozantinib) to everolomus in patients with metastatic renal cell carcinoma (RCC) who have experienced disease progression following treatment with a VEGF receptor tyrosine kinase inhibitor (TKI).

Study on Opdivo for Renal Cell Carcinoma Halted Early

Bristol Myers Squibb Company announced that CheckMate-025, a Phase 3 study investigating Opdivo (nivolumab) vs. everolimus in previously treated patients with advanced or metastatic renal cell carcinoma (RCC) was stopped early due to the primary endpoint being met.

Sustol NDA Resubmitted for Chemotherapy-Induced Nausea and Vomiting

Heron Therapeutics announced that it has resubmitted its New Drug Application (NDA) for Sustol (granisetron) injection, extended release to the Food and Drug Administration (FDA) for the prevention of acute and delayed chemotherapy-induced nausea and vomiting (CINV) associated with moderately emetogenic chemotherapy (MEC) or highly emetogenic chemotherapy HEC) regimens.

Positive Phase 2 Study Results for Blincyto in Leukemia Study

Amgen announced positive results from the Phase 2 trial of Blincyto (blinatumomab) for the treatment of relapsed Philadelphia chromosome-positive B-cell precursor acute lymphoblastic leukemia (ALL).

Progressive Supranuclear Palsy Tx Granted Orphan Drug Status

C2N Diagnostics and AbbVie announced that the Food and Drug Administration (FDA) has granted orphan drug designation for C2N-8E12 (ABBV-8E12) for the treatment of progressive supranuclear palsy (PSP). C2N-8E12 is a recombinant humanized antibody targeting the tau protein found in neurofibrillary tangles in the brain of patients with tauopathies such as PSP and Alzheimer's disease (AD).

Investigational Malaria Drug Could Be One-Dose Tx

Investigational Malaria Drug Could Be One-Dose Tx

An investigational enzyme inhibitor may be able to cure malaria in a single dose when used in conjunction with another drug, and also be utilized as a preventative treatment.

Ixazomib NDA Submitted for Relapsed/Refractory Multiple Myeloma

Takeda has submitted a New Drug Application (NDA) to the Food and Drug Administration (FDA) for ixazomib for the treatment of patients with relapsed and/or refractory multiple myeloma.

Sarizotan Designated Orphan Drug for Neurologic Disorder

The Food and Drug Administration (FDA) has granted Orphan Drug designation to sarizotan (Newron) for the treatment of Rett syndrome.

Novel Oral Susp Found Effective in Treating Hyperkalemia

Patiromer oral suspension, an investigational drug, was found to reduce elevated potassium levels in patients with chronic diabetic kidney disease in a study published in JAMA.

Toca 511 and Toca FC Fast Tracked for Recurrent High Grade Glioma

The Food and Drug Administration (FDA) has granted Fast Track status to Toca 511 and Toca FC for the treatment of recurrent high grade glioma, which includes glioblastoma and anaplastic astrocytoma.

Guselkumab May Clear Plaque Psoriasis, Shows Study

Janssen Research & Development announced results from the Phase 2b X-PLORE trial with guselkumab in patients with moderate to severe plaque psoriasis.

Hereditary Angioedema Tx Now a Breakthrough Therapy

The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for the investigation of DX-2930 (Dyax) for hereditary angioedema (HAE).

FDA to Review New Xeljanz Formulation for Rheumatoid Arthritis

Pfizer announced that the Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for Xeljanz (tofacitinib citrate) 11mg once daily modified release tablets for the treatment of moderate to severe rheumatoid arthritis in patients with inadequate response or intolerance to methotrexate.

Volanesorsen Designated Orphan Drug Status for Familial Chylomicronemia Syndrome

The Food and Drug Administration (FDA) has granted Orphan Drug designation to volanesorsen (ISIS-APOCIIIRx; Akcea Therapeutics) for the treatment of patients with Familial Chylomicronemia Syndrome (FCS).

Gene Therapy Found to Improve Lung Function in CF

Gene therapy showed a significant benefit in lung function in patients with cystic fibrosis, data from a Phase 2 trial has shown.

Aerucin Awarded Fast Track for HAP/VAP

The Food and Drug Administration (FDA) has granted Fast Track designation to Aerucin (Aridis Pharmaceuticals) for the treatment of hospital-acquired and ventilator-associated pneumonia caused by Pseudomonas aeruginosa.

Investigational MS Tx Reduces Relapse Rate in Trials

Genentech announce positive results from two Phase 3 studies evaluating ocrelizumab compared with Rebif (interferon beta-1a) in patients with relapsing multiple sclerosis (MS).

Eteplirsen NDA Submission Completed for Duchenne Muscular Dystrophy

Sarepta Therapeutics announced that it has completed a rolling submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA) for eteplirsen.

Emend Efficacious in Preventing Chemotherapy-Induced Nausea and Vomiting

Merck announced results from a Phase 3 study of single-dose Emend (fosaprepitant dimeglumine) for injection in combination with other anti-vomiting medications for the prevention of chemotherapy-induced nausea and vomiting (CINV) in adult cancer patients receiving moderately emetogenic chemotherapy (MEC).

Egg-Free Flu Vaccine Beats Traditional Vaccine, Finds Study

Egg-Free Flu Vaccine Beats Traditional Vaccine, Finds Study

Protein Sciences announced that Flublok Quadrivalent was better at preventing influenza than the traditional influenza vaccine.

FDA to Review NDA for Metastatic Pancreatic Adenocarcinoma Therapy

The Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for irinotecan liposome injection (MM-398; Merrimack Pharmaceuticals and Baxalta) for the treatment of patients with metastatic adenocarcinoma of the pancreas who have been previously treated with gemcitabine-based therapy.

Peanut Allergy Patch Gets Closer to FDA Submission

Peanut Allergy Patch Gets Closer to FDA Submission

DBV Technologies announced that based its meeting with the Food and Drug Administration (FDA), it plans to initiate a global Phase 3 study of its investigational Viaskin Peanut epicutaneous immunotherapy (EPIT) patch for the treatment of peanut allergies.

No Virologic Failures With Combo HCV Tx in Study

AbbVie announced results from the Phase 3b TURQUOISE-III study with Viekirax (ombitasvir/paritaprevir/ritonavir) plus Exviera (dasabuvir) without ribavirin (RBV) in genotype 1b (GT1b) chronic hepatitis C virus (HCV) infected adult patients with compensated liver cirrhosis.

NDA Submitted for Oral Formulation of OIC Drug Relistor

Valeant Pharmaceuticals and Progenics Pharmaceuticals announced the submission of a New Drug Application (NDA) to the FDA for Oral Relistor (methylnaltrexone bromide) Tablets for the treatment of opioid-induced constipation (OIC) in adult patients with chronic non-cancer pain.

Eliquis Reversal Agent Demonstrates Efficacy in Phase 3 Study

Portola Pharmaceuticals, Bristol-Myers Squibb Company, and Pfizer announced results from the Phase 3 ANNEXA-A (Andexanet Alfa a Novel Antidote to the Anticoagulant Effects of FXa Inhibitors - Apixaban) study with andexanet alfa as a reversal agent for Eliquis (apixaban).

Pradaxa Reversal Agent Works Within Minutes in Phase 3 Study

Pradaxa Reversal Agent Works Within Minutes in Phase 3 Study

Boehringer Ingelheim announced results from an interim analysis of the Phase 3 RE-VERSE AS patient study with idarucizumab for urgent reversal of the anticoagulant effect of Pradaxa (dabigatran etexilate mesylate).

ImMucin Designated Orphan Drug for Multiple Myeloma

The Food and Drug Administration (FDA) has granted Orphan Drug designation to ImMucin (Vaxil Bio) for the treatment of multiple myeloma.

Encenicline Granted Fast Track Status for Cognitive Impairment in Schizophrenia

The Food and Drug Administration (FDA) has granted Fast Track designation to encenicline (Forum Pharmaceuticals) for the treatment of cognitive impairment in schizophrenia.

Peanut Allergy Drug Designated Breakthrough Therapy

Peanut Allergy Drug Designated Breakthrough Therapy

The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to AR101, an oral immunotherapy for children and adolescents 4 to 17 years of age who are allergic to peanuts.

New Drug Class for Migraine Prevention Could Be Coming

New Drug Class for Migraine Prevention Could Be Coming

Clinical trials of a new drug class for preventing high-frequency episodic migraine and chronic migraine have shown promising results, according to several studies presented at the American Headache Society's annual Scientific Meeting.

Top-Line Results for Plecanatide in Chronic Idiopathic Constipation Trial

Synergy Pharmaceuticals announced positive results from the first of two Phase 3 clinical trials evaluating the efficacy and safety of plecanatide 3mg and 6mg treatment doses in patients with chronic idiopathic constipation (CIC).

Gedeptin Granted Orphan Drug Status for Oral and Pharyngeal Cancers

The Food and Drug Administration (FDA) has granted Orphan Drug designation to Gedeptin (adenoviral vector expressing E. coli purine nucleoside phosphorylase gene; PNP Therapeutics) for the intratumoral treatment of anatomically accessible oral and pharyngeal cancers.

Chiasma Submits Octreotide NDA for Acromegaly

Chiasma announced that it has submitted a New Drug Application (NDA) to the Food and Drug Administration (FDA) for octreotide capsules for the maintenance treatment of adults with acromegaly.

Ischemic Chronic Heart Failure Drug Fast Tracked by FDA

The Food and Drug Administration (FDA) has granted Fast Track designation to JVS-100 (Juventas Therapeutics) for the treatment of advanced ischemic chronic heart failure.

Orphan Drug Status Granted to Immune Thrombocytopenia Therapy

The Food and Drug Administration (FDA) has granted Orphan Drug designation to PRTX-100 (Protalex) for the treatment of immune thrombocytopenia (ITP).

Investigational Device Could Deliver Up to a Full Year of GLP-1 Therapy

Investigational Device Could Deliver Up to a Full Year of GLP-1 Therapy

Intarcia Therapeutics announced results from two Phase 3 trials of its investigational drug candidate ITCA 650 (continuous exenatide delivery via subcutaneous osmotic min-pump) at the 75th American Diabetes Association (ADA) Scientific Sessions in Boston, MA.

Short Bowel Syndrome Therapy Designated Orphan Drug Status

The Food and Drug Administration (FDA) has granted Orphan Drug designation to NTRA-9620 (Nutrinia), an orally-administered drug for treating short bowel syndrome (SBS).

Investigational Peanut Allergy Tx Improves Tolerance in Younger Patients

Investigational Peanut Allergy Tx Improves Tolerance in Younger Patients

Aimmune Therapeutics announced that the Phase 2 study of a novel investigational oral immunotherapy (AR101) for the treatment of peanut allergy met its primary endpoint of tolerating a cumulative amount of peanut protein of at least 443mg.

Study: No Increased CV Risk With Lixisenatide for Diabetes

Study: No Increased CV Risk With Lixisenatide for Diabetes

Sanofi announced the full results of the first event-driven cardiovascular (CV) outcomes study on the safety of lixisenatide in patients with type 2 diabetes and high CV risk at the 75th American Diabetes Association (ADA) Scientific Sessions in Boston, MA.

Canagliflozin + Metformin XR Superior to Either Alone in Study

Janssen R&D announced results from a Phase 3 study on the use of initial combination therapy of canagliflozin and metformin extended-release (XR) in type 2 diabetes patients with higher A1C levels and who treat their type 2 diabetes with diet and exercise only.

Vaccine for Type 1 Diabetes Reversal to Be Studied in Clinical Trial

Vaccine for Type 1 Diabetes Reversal to Be Studied in Clinical Trial

The Food and Drug Administration (FDA) has granted approval to the initiation of a Phase 2 clinical trial evaluating the use of generic vaccine bacillus Calmette-Guerin (BCG) to reverse advanced type 1 diabetes .

Novel Enzyme Designated Breakthrough Therapy for Niemann-Pick Disease

Genzyme announced that the Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for olipudase alfa, an enzyme replacement therapy being studied for the treatment of patients with non-neurological manifestations of acid sphingomyelinase deficiency (ASMD).

Novel Agent AMG 416 Effective in Secondary Hyperparathyroidism Trials

Novel Agent AMG 416 Effective in Secondary Hyperparathyroidism Trials

Amgen announced pooled data from two Phase 3 trials evaluating AMG 416 for the treatment of secondary hyperparathyroidism (SHPT) in patients with chronic kidney disease (CKD) receiving hemodialysis.

NDA Submitted for Combination Chronic Hepatitis C Therapy

Merck has submitted a New Drug Application (NDA) to the Food and Drug Administration (FDA) for grazoprevir/elbasvir 100mg/50mg for the treatment of adults with chronic hepatitis C genotypes 1, 4, or 6 infection

Coagadex Amended BLA Under Review for Rare Bleeding Disorder

The Food and Drug Administration (FDA) has accepted for review the amended Biologics License Application (BLA) for Coagadex (coagulation factor X, human) for hereditary X deficiency.

Results Announced From Phase 3 Trial of Combo COPD Therapy

Novartis announced results from its Phase 3 EXPEDITION trial for indacaterol/glycopyrronium (QVA149) in patients with moderate-to-severe chronic obstructive pulmonary disease (COPD).

Orphan Drug Status Granted to Ovarian Cancer Therapy

The Food and Drug Administration (FDA) has granted Orphan Drug designation to CRLX101 (Cerulean Pharma) for the treatment of ovarian cancer.

Combination COPD Therapy Improves Lung Function in Trials

Boehringer Ingelheim announced new data analyses from the Phase 3 TONADO 1&2 studies of tiotropium/olodaterol delivered via Respimat inhaler for chronic obstructive pulmonary disease (COPD).

Sarilumab Improves Physical Function in Rheumatoid Arthritis Trial

Regeneron and Sanofi announced results from their Phase 3 SARIL-RA-TARGET trial of sarilumab in patients with rheumatoid arthritis (RA).

Optina Efficacious in Diabetic Macular Edema Study

Ampio Pharmaceuticals announced positive results from the OptimEyes Trial for Optina in patients with diabetic macular edema (DME).

Dyanavel XR NDA Accepted for FDA Review

Tris Pharma announced that the Food and Drug Administration (FDA) has accepted for review its New Drug Application (NDA) for Dyanavel XR (amphetamine) CII, an Extended-Release Oral Suspension.

Rare Neurologic Disorder Therapy Designated Orphan Drug

Rare Neurologic Disorder Therapy Designated Orphan Drug

The Food and Drug Administration (FDA) has granted Orphan Drug designation to revusiran (Alnylam) for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis).

Investigational HCV Tx Receives Amended Breakthrough Designation

Investigational HCV Tx Receives Amended Breakthrough Designation

Bristol-Myers Squibb announced that the Food and Drug Administration (FDA) has amended a previously granted Breakthrough Therapy designation for the investigational daclatasvir and sofosbuvir combination for use in hepatitis C (HCV) patients.

Progression-Free Survival Seen With Enzalutamide for Prostate Cancer in Phase 2 Trial

Astellas Pharma and Medivation announced data from the Phase 2 STRIVE trial comparing Xtandi (enzalutamide) and bicalutamide in non-metastatic (M0) and metastatic (M1) prostate cancer patients whose disease progressed despite treatment with a luteinizing hormone-releasing hormone (LHRH) analogue therapy of following surgical castration.

Solifenacin + Mirabegron Shows Superiority in Incontinent Overactive Bladder Trial

Astellas Pharma announced results from the Phase 3b BESIDE trial with solifenacin (SOLI) with mirabegron (MIRA) as an add-on therapy in incontinent overactive bladder (OAB) patients.

Luspatercept Fast Tracked for Two Beta-Thalassemia Indications

The Food and Drug Administration (FDA) has granted Fast Track designations to luspatercept (Celgene and Acceleron) for use in two separate indications: for the treatment of patients with transfusion dependent beta-thalassemia and for the treatment of patients with non-transfusion dependent beta-thalassemia.

Humira Designated Orphan Drug for Painful, Inflammatory Skin Disease

AbbVie announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation for Humira (adalimumab) as an investigational treatment for moderate-to-severe hidradenitis suppurativa (HS; Hurley Stage II and III disease).

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