Drugs in the Pipeline

Gene Therapy Looks Promising for Transfusion-Dependent β-Thalassemia

Gene Therapy Looks Promising for Transfusion-Dependent β-Thalassemia

The safety and efficacy of LentiGlobin were evaluated in 2 independent 2-year clinical studies: HGB-204 (Northstar, N=18), an open-label, single-dose, nonrandomized, multicenter Phase 1/2 study in patients with TDT, and HGB-205, an ongoing open-label, single-dose, nonrandomized, single-center Phase 1/2 study in patients with TDT and severe sickle cell disease.

Oraxol Gets Orphan Drug Designation for Angiosarcoma

Oraxol Gets Orphan Drug Designation for Angiosarcoma

Oraxol is a novel oral therapy that combines paclitaxel, a tubulin-stabilizing chemotherapeutic agent, with a non-absorbable gastrointestinal tract P-glycoprotein pump inhibitor.

Hemlibra Gets Breakthrough Tx Status for Hemophilia A Without Inhibitors

Hemlibra Gets Breakthrough Tx Status for Hemophilia A Without Inhibitors

The trial enrolled 152 patients with hemophilia A who were previously treated, either on-demand or as prophylaxis, with factor VIII therapy.

Esketamine Evaluated in MDD Patients at Imminent Risk for Suicide

Esketamine Evaluated in MDD Patients at Imminent Risk for Suicide

If approved, esketamine has the potential to be the first treatment for patients with major depressive disorder at imminent risk for suicide.

FDA to Review Opioid System Modulator for Major Depressive Disorder

FDA to Review Opioid System Modulator for Major Depressive Disorder

ALKS 5461 consists of a fixed-dose combination of buprenorphine, a partial mu-opioid receptor agonist and kappa-opioid receptor antagonist, and samidorphan, a mu-opioid receptor antagonist.

Long-Term Obeticholic Acid Linked to Fibrosis Regression in PBC Patients

Long-Term Obeticholic Acid Linked to Fibrosis Regression in PBC Patients

Results showed that 46% (n=6) of substudy patients improved their histological fibrosis stage, while 38% maintained (n=5), and 15% experienced 1 stage progression (n=2).

Treatment for Idiopathic Pulmonary Fibrosis Gets Orphan Drug Designation

Treatment for Idiopathic Pulmonary Fibrosis Gets Orphan Drug Designation

RP5063 is a new chemical entity with a novel mechanism of multimodal modulation of serotonin and dopamine signaling pathways.

Phase 3 Results Released for Investigational Alzheimer's Disease Treatment

Phase 3 Results Released for Investigational Alzheimer's Disease Treatment

Results showed that after 18-months, there was no significant statistical difference in cognitive or functional outcomes between those patients taking azeliragon and the placebo group.

Top-Line Results Announced for Investigational RA Treatment Upadacitinib

Top-Line Results Announced for Investigational RA Treatment Upadacitinib

At week 12, results showed 71% in the upadacitinib group had achieved ACR20, compared to 63% and 36% in the adalimumab and placebo groups, respectively.

Elamipretide Granted Orphan Drug Status for Barth Syndrome

Elamipretide Granted Orphan Drug Status for Barth Syndrome

The TAZPOWER study is a randomized, double-blind, placebo-controlled Phase 2/3 crossover study evaluating the safety and efficacy of elamipretide (daily subcutaneous injections) in 12 male patients (aged ≥12 years) with genetically-confirmed Barth syndrome.

Duvelisib NDA Gets Priority Review for Relapsed/Refractory CLL/SLL, Follicular Lymphoma

Duvelisib NDA Gets Priority Review for Relapsed/Refractory CLL/SLL, Follicular Lymphoma

Duvelisib is a first-in-class oral dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma.

Tenalisib Granted Orphan Drug Designation for Cutaneous T-Cell Lymphoma

Tenalisib Granted Orphan Drug Designation for Cutaneous T-Cell Lymphoma

Preclinical studies have demonstrated that RP6530 reprograms macrophages from an immunosuppressive M2-like phenotype (pro-tumor) to an inflammatory M1-like state (anti-tumor), which can potentially enhance the activity of checkpoint inhibitors or overcome resistance to these agents.

Omadacycline Granted Priority Review for CABP, Skin/Skin Structure Infections

Omadacycline Granted Priority Review for CABP, Skin/Skin Structure Infections

Omadacycline is an investigational, once-daily, broad-spectrum aminomethylcycline antibiotic that is related to tetracyclines.

Dacomitinib Granted Priority Review for NSCLC With EGFR-Activating Mutations

Dacomitinib Granted Priority Review for NSCLC With EGFR-Activating Mutations

Dacomitinib is a pan-human epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor.

Positive Top-Line Results for Ramucirumab in HCC Announced

Positive Top-Line Results for Ramucirumab in HCC Announced

REACH-2 was a randomized, double-blind, placebo-controlled trial that enrolled 292 patients with hepatocellular carcinoma (HCC) who were intolerant to, or had disease progression while on or following treatment with sorafenib and had a high alpha-fetoprotein (AFP-High) defined as AFP ≥400ng/mL.