Clinical TrialsInformation and insight on the latest clinical trials
- FDA Grants Fast Track Designation to Bullous Pemphigoid Treatment
The U.S. Food and Drug Administration (FDA) granted Fast Track designation to bertilimumab for the treatment of bullous pemphigoid, a skin disorder characterized by large blisters.
- Celtaxsys: Focus on Cystic Fibrosis and Rare Inflammatory Diseases
Greg Duncan, President and Chief Executive Officer of Celtaxsys, provides an overview of his company and it’s focus on cystic fibrosis (CF), an inherited chronic disease that affects the lungs and digestive system of about 70,000 children and adults worldwide.
- Angelman Syndrome Overview
Amit Rakhit, MD, Chief Medical and Portfolio Officer at Ovid Therapeutics, provides an overview on Angelman syndrome. Angelman syndrome is a complex genetic disorder that primarily affects the nervous system. Characteristic features of this condition i …
- FDA Approves Tiglutik for the Treatment of Amyotrophic Lateral Sclerosis
The US Food and Drug Administration (FDA) approved Tiglutik (riluzole) oral suspension for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.
- Buergers Disease Treatment Receives Orphan Drug Designation
U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for ATI-1013, a fully human, anti-nicotine monoclonal antibody for the treatment of Buerger’s Disease.
- Novel Therapy for Cystic Fibrosis Completes Phase 2 Trial
Greg Duncan, President and Chief Executive Officer of Celtaxsys, provides an overview of cystic fibrosis (CF), an inherited chronic disease that affects the lungs and digestive system of about 70,000 children and adults worldwide.
- ATTR Study Shows Tafamidis Minimizes Mortality and Hospital Visitation
Pfizer announced positive data from it’s phase 3 study (called ATTR-ACT), which evaluated it’s experimental therapy Tafamidis (vyndaqel) in patients with wild-type or variant (hereditary) transthyretin amyloid cardiomyopathy (ATTR-CM).
- A Novel Protein Therapy in Development for Achondroplasia (Short-Limbed Dwarfism)
Luca Santarelli, MD of Therachon discusses his company’s lead pipeline candidate, TA-46, a novel protein therapy in development for achondroplasia, the most common form of short-limbed dwarfism.
- FDA Allows Use of Kalydeco in Children Under Two Years Old
The U.S. Food and Drug Administration (FDA) has approved ivacaftor (KALYDECO) to be used in children with cystic fibrosis ages under the age of 2 years old who have at least 1 mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to the drug.
- Positive Results in Phase 3 Cushing’s Syndrome Trial
Strongbridge Biopharma’s pivotal phase 3 SONICS study evaluating levoketoconazole (Recorlev) for the treatment of endogenous Cushing’s syndrome achieved statistical significance of its pre-specified primary endpoint.