Drugs in the Pipeline

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).

FDA Panel Backs Approval of CF Drug Orkambi

Vertex announced that the Food and Drug Administration's (FDA) Pulmonary-Allergy Drugs Advisory Committee (PADAC) has voted 12 to 1 to recommend that the FDA approve Orkambi (lumacaftor/ivacaftor) for people with cystic fibrosis (CF) aged 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Evofosfamide Fast Tracked for Advanced Pancreatic Cancer

The Food and Drug Administration (FDA) has granted Fast Track status to evofosfamide (Merck KGaA) in combination with gemcitabine, for the treatment of previously untreated patients with metastatic or locally advanced unresectable pancreatic cancer.

Antifungal Therapy Fast Tracked, Granted QDIP Status by FDA

The Food and Drug Administration (FDA) has granted Qualified Infectious Disease Product (QIDP) with Fast Track status to CD101 IV (Cidara Therapeutics) for the treatment of candidemia and invasive candidiasis.

Humira for Panuveitis Reduces Vision Loss in Latest Phase 3 Trial

AbbVie announced results from its Phase 3 study VISUAL-1 investigating the efficacy and safety of Humira (adalimumab) in adults with active non-infectious intermediate, posterior, or panuveitis who still experienced intraocular inflammation while on systemic corticosteroid therapy.

Rare Neurologic Disorder Therapy Granted Orphan Drug Status

The Food and Drug Administration (FDA) has granted Orphan Drug designation to its investigational therapy RE-024 (phosphopantothenate replacement therapy; Retrophin), for the treatment of pantothenate kinase-associated neurodegeneration (PKAN).