Drugs in the Pipeline

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.