Drugs in the Pipeline

FDA Grants Lorlatinib Breakthrough Therapy Designation for NSCLC

FDA Grants Lorlatinib Breakthrough Therapy Designation for NSCLC

Pfizer has begun enrolling patients in a Phase 3 study of lorlatinib, called 'CROWN'. The trial will be an open-label randomized, two-arm study comparing lorlatinib to crizotinib in the first-line treatment of patients with metastatic ALK-positive NSCLC.

FDA to Review BLA Resubmission of Rheumatoid Arthritis Biologic

FDA to Review BLA Resubmission of Rheumatoid Arthritis Biologic

The FDA has accepted for review the resubmission of the Biologics License Application (BLA) for Kevzara (sarilumab; Regeneron and Sanofi) for the treatment of adults with moderately to severely active rheumatoid arthritis (RA) who have had an inadequate response or intolerance to one or more disease modifying antirheumatic drugs.

Novel DNRI Agent Improves ADHD Symptoms in Study

Novel DNRI Agent Improves ADHD Symptoms in Study

Study data revealed children taking dasotraline 4mg daily had statistically significant and clinically meaningful improvement vs. placebo on the primary endpoint.

New Findings for Novel Pan-Genotypic HCV Regimen in Phase 3 Study

New Findings for Novel Pan-Genotypic HCV Regimen in Phase 3 Study

New findings from EXPEDITION-1 showed that 99% of HCV infected patients with GT 1, 2, 4, 5 or 6 and compensated cirrhosis achieved SVR12 after 12 weeks of G/P treatment.

Tissue-Based Tx Granted Breakthrough Status for Complete DiGeorge Syndrome

Tissue-Based Tx Granted Breakthrough Status for Complete DiGeorge Syndrome

The FDA has granted Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations for the investigational tissue-based therapy, RVT-802 (Enzyvant), for the treatment of complete DiGeorge Syndrome (cDGS).

FDA to Review Fostamatinib for Chronic, Persistent ITP

FDA to Review Fostamatinib for Chronic, Persistent ITP

Rigel announced its submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA) for fostamatinib for the treatment of patients with chronic and persistent immune thrombocytopenic purpura (ITP).

Emicizumab Evaluated in Children with Hemophilia A

Emicizumab Evaluated in Children with Hemophilia A

The interim results from HAVEN 2, after a median of 12 weeks of treatment, showed that prophylaxis with emicizumab is associated with clinically meaningful reduction in the number of bleeds over time.

FDA Requires More Data Before Baricitinib Approval

FDA Requires More Data Before Baricitinib Approval

The NDA for baricitinib was submitted in January 2016 and a 3-month extension for time to review additional analyses was announced in January 2017.

NDA Submitted for Post-Cataract Surgery Inflammation Treatment

NDA Submitted for Post-Cataract Surgery Inflammation Treatment

Dexycu is a long-acting, biodegradable extended-release formulation of the anti-inflammatory agent dexamethasone, which is injected into the anterior chamber of the eye following cataract surgery.

FDA Fast Tracks d-Methadone as Adjunct Treatment for Depression

FDA Fast Tracks d-Methadone as Adjunct Treatment for Depression

The company has completed a Phase 1 study for REL-1017 and intends to initiate a randomized, double-blind, placebo-controlled Phase 2a trial in patients with major depressive disorder to assess its efficacy, safety, tolerability and pharmacokinetics.

Combo Tx Significantly Improves Lung Function in Cystic Fibrosis Study

Combo Tx Significantly Improves Lung Function in Cystic Fibrosis Study

Results showed that the ivacaftor/tezacaftor group reached the primary endpoint with a mean absolute improvement in ppFEV1 of 4% points from baseline vs. placebo (P<0.0001).

Alectinib Improves Progression-Free Survival in NSCLC Study

Alectinib Improves Progression-Free Survival in NSCLC Study

The ALEX study is intended to convert Alecensa to full approval as an initial treatment.

Evinacumab Gets Breakthrough Status for Severe Form of Hypercholesterolemia

Evinacumab Gets Breakthrough Status for Severe Form of Hypercholesterolemia

Regeneron previously reported positive interim Phase 2 results which showed that evinacumab added to current lipid-lowering therapy reduced low-density lipoprotein cholesterol (LDL-C) levels by an additional 55%.

Opdivo Granted Priority Review for Colorectal Cancer Indication

Opdivo Granted Priority Review for Colorectal Cancer Indication

Opdivo, a human programmed death receptor-1 (PD-1)-blocking antibody, is already indicated for various renal and urologic cancers, head and neck cancer, lymphoma, skin cancer, and lung cancer.

First-in-Class Injectable Antibiotic Shows Promise for Complicated UTIs

First-in-Class Injectable Antibiotic Shows Promise for Complicated UTIs

Zavante Therapeutics announced promising results from the pivotal ZEUS clinical trial, evaluating Zolyd (fosfomycin for injection) for the treatment of complicated urinary tract infections (cUTI), including acute pyelonephritis (AP).