Drugs in the Pipeline

FDA to Review Rexulti as Maintenance Therapy for Schizophrenia

The Food and Drug Administration (FDA) has accepted for review a supplemental New Drug Application (sNDA) for the proposed labeling update of Rexulti (brexpiprazole; Otsuka and Lundbeck) for the maintenance treatment of adults with schizophrenia.

Tazemetostat Granted Orphan Drug Status for Rare, Aggressive Cancers

The Food and Drug Administration (FDA) has granted Orphan Drug designation to tazemetostat (Epizyme) for the treatment of malignant rhabdoid tumors (MRTs).

Complete Response Letter Issued for Expanded Kalydeco Indication

Complete Response Letter Issued for Expanded Kalydeco Indication

The Food and Drug Administration (FDA) has issued a Complete Response Letter for the supplemental New Drug Application (sNDA) of Kalydeco (ivacaftor; Vertex) in patients with cystic fibrosis (CF) ≥2 years old who have one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Top-line Results Announced for Repatha in Patients Who Are Statin-Intolerant

Top-line Results Announced for Repatha in Patients Who Are Statin-Intolerant

Amgen announced positive top-line data from the Phase 3 GAUSS-3 study evaluating Repatha (evolocumab) in statin-intolerant patients with high cholesterol.

Complete Response Letter Issued for MCNA in Bladder CA

The Food and Drug Administration (FDA) has issued a Complete Response Letter to the Biologics License Application (BLA) for MCNA (Telesta Therapeutics) in the treatment of non-muscle invasive bladder cancer (NMIBC).

Ovarian Cancer Vaccine Granted Fast Track Status

Ovarian Cancer Vaccine Granted Fast Track Status

TapImmune announced that the Food & Drug Administration (FDA) has granted Fast Track designation for its cancer vaccine TPIV 200 in the treatment of ovarian cancer.

Investigational NASH Tx Fast Tracked by FDA

Investigational NASH Tx Fast Tracked by FDA

The Food and Drug Administration (FDA) has granted Fast Track designation to NDI-010976 (Nimbus Therapeutics) for the treatment of non-alcoholic steatohepatitis (NASH)

NDA Submitted for Chronic Idiopathic Constipation Drug Plecanatide

Synergy Pharmaceuticals announced the submission of its first New Drug Application (NDA) to the Food and Drug Administration (FDA) for plecanatide for the treatment of chronic idiopathic constipation (CIC).

Ryanodex Fast Tracked for Exertional Heat Stroke

Ryanodex Fast Tracked for Exertional Heat Stroke

The Food and Drug Administration (FDA) has granted Fast Track designation to Ryanodex (dantrolene sodium; Eagle) injectable suspension for the treatment of exertional heat stroke (EHS).

Positive Data for Investigational Hepatic VOD Tx Announced

Positive Data for Investigational Hepatic VOD Tx Announced

Jazz Pharmaceuticals announced positive data from the Phase 3 pivotal study of defibrotide in patients with hepatic veno-occlusive disease (VOD) with multi-organ failure (MOF) post-hematopoietic stem-cell transplantation (HSCT). Findings were published online in Blood, the Journal of the American Society of Hematology (ASH).

Combo Tx Fast Tracked for Fibromyalgia

The Food and Drug Administration (FDA) has granted Fast Track designation to IMC-1 (Innovative Med Concepts) for the treatment of fibromyalgia.

FDA Grants Breakthrough Tx Designation to Novel Antidepressant

FDA Grants Breakthrough Tx Designation to Novel Antidepressant

The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to the investigational drug rapastinel (GLYX-13; Allergan) for the adjunctive treatment of Major Depressive Disorder (MDD).

Study on Opdivo for Head, Neck CA Halted Early

Study on Opdivo for Head, Neck CA Halted Early

Bristol-Myers Squibb announced that the CheckMate -141 trial evaluating Opdivo (nivolumab) vs. investigator's choice in patients with recurrent or metastatic platinum-refractory squamous cell carcinoma of the head and neck (SCCHN) was stopped early due to the study meeting its primary endpoint.

FDA Grants Priority Review to Expanded Cabozantinib Indication

The Food & Drug Administration (FDA) has accepted and granted Priority Review to the New Drug Application (NDA) for cabozantinib as a treatment for patients with advanced renal cell carcinoma (RCC) who have received one prior therapy.

Novel Antitoxin Granted Priority Review for CDI

The Food and Drug Administration (FDA) has accepted and granted Priority Review to the Biologics License Application (BLA) for bezlotoxumab (Merck) for the prevention of Clostridium difficile (C. difficile) infection recurrence.