Drugs in the Pipeline

BLA for Novel Peanut Allergy Treatment Submitted to FDA

BLA for Novel Peanut Allergy Treatment Submitted to FDA

Viaskin Peanut, an epicutaneous immunotherapy (EPIT), delivers small amounts of peanut protein through a wearable patch to induce desensitization.

FDA Panel Votes on Prucalopride for Chronic Idiopathic Constipation

FDA Panel Votes on Prucalopride for Chronic Idiopathic Constipation

The panel's decision was made based on data from from five Phase 3 studies and one Phase 4 trial which evaluated the safety and efficacy of prucalopride in patients with chronic constipation.

BTK Inhibitor Designated Orphan Drug for Immune Thrombocytopenic Purpura

BTK Inhibitor Designated Orphan Drug for Immune Thrombocytopenic Purpura

PRN1008 is an oral, reversible covalent Bruton's tyrosine kinase (BTK) inhibitor; BTK is present in the signaling pathways of most types of white blood cells except for T cells and plasma cells.

Novel Narcolepsy Treatment Gets Orphan Drug Designation

Novel Narcolepsy Treatment Gets Orphan Drug Designation

The Company stated that they plan to begin a Phase 2 trial of AXS-12 before the end of 2018, with results anticipated for the first half of 2019.

Positive Results for Lemborexant in Insomnia Disorder Study

Positive Results for Lemborexant in Insomnia Disorder Study

SUNRISE 2 is an ongoing long-term efficacy and safety study (N=900) of adults (aged 18 to 88 years) with insomnia disorder who have difficulty falling and/or staying asleep.

Treatment for Alagille Syndrome Granted Orphan Drug Designation

Treatment for Alagille Syndrome Granted Orphan Drug Designation

Alagille syndrome affects approximately 1 in 70,000 newborns.

Lynparza Granted Orphan Drug Status for Pancreatic Cancer

Lynparza Granted Orphan Drug Status for Pancreatic Cancer

Lynparza, a poly (ADP-ribose) polymerase (PARP) inhibitor, is being assessed in the ongoing Phase 3 POLO trial.

Dupilumab Studies Show Benefit in Chronic Rhinosinusitis With Nasal Polyps

Dupilumab Studies Show Benefit in Chronic Rhinosinusitis With Nasal Polyps

In both studies, at 24 weeks, patients treated with dupilumab plus corticosteroid nasal spray experienced a 51% and 57% improvement in nasal congestion/obstruction severity vs 15% and 19% improvement with nasal spray alone.

NDA Submitted for Dry Eye Disease Treatment KPI-121 0.25%

NDA Submitted for Dry Eye Disease Treatment KPI-121 0.25%

The NDA includes data from one Phase 2 and two Phase 3 clinical trials (STRIDE 1 and STRIDE 2) involving ~2000 individuals with dry eye disease.

Treatment for WHIM Syndrome Gets Orphan Drug Designation

Treatment for WHIM Syndrome Gets Orphan Drug Designation

X4 Pharmaceuticals is developing the drug, which is currently in a Phase 2/3 clinical trial involving patients with WHIM syndrome.

FDA Panel Votes on Drug/Device Combo to Treat Pain in Medically Supervised Setting

FDA Panel Votes on Drug/Device Combo to Treat Pain in Medically Supervised Setting

The FDA is not bound by the Committee's recommendation but takes it into consideration when making its decision.

STRO-001 Gets Orphan Drug Designation for Mutiple Myeloma

STRO-001 Gets Orphan Drug Designation for Mutiple Myeloma

STRO-001 targets CD74, which is a protein highly expressed in B-cell malignancies like multiple myeloma.

Novel Inhaled Treatment for Cystic Fibrosis Granted Orphan Drug Designation

Novel Inhaled Treatment for Cystic Fibrosis Granted Orphan Drug Designation

SNSP113 is intended to improve lung function in CF patients by targeting key drivers of pulmonary decline such as infection, airway congestion and inflammation.

Ustekinumab Induces Clinical Remission After One Dose in Ulcerative Colitis Study

Ustekinumab Induces Clinical Remission After One Dose in Ulcerative Colitis Study

Study authors at the American College of Gastroenterology (ACG) Scientific Meeting 2018 presented late-breaking data from the induction phase of the Phase 3 UNIFI study.

FDA to Review Siponimod for Secondary Progressive Multiple Sclerosis

FDA to Review Siponimod for Secondary Progressive Multiple Sclerosis

The NDA is based on data from the EXPAND study, a randomized, double-blind, placebo-controlled Phase 3 study in 1652 participants living with SPMS.