Articles

Here’s a round-up of recent designations granted by the FDA to products developed to treat rare diseases.

Orphan Drug Designation Granted to CLN2 Form of Batten Disease, RGX-181

FDA Grants Fast Track Designation to A4250 for Progressive Familial Intrahepatic Cholestasis

The FDA recognized the potential of proCase, which is being developed to treat blood clots and does not increase the risk of bleeding.

Blood Clot Treatment Fast-tracked By FDA

The FDA has granted a fast track designation to miransertib for the treatment of PIK3CA-Related Overgrowth Spectrum, a group of ultra-rare genetic disorders.

Miransertib Granted Fast Track Designation for Treatment of PIK3CA-Related Overgrowth

The FDA has granted a fast track designation to pamrevlumab for the treatment of patients with idiopathic pulmonary fibrosis (IPF).

Fast Track Designation Granted to Idiopathic Pulmonary Fibrosis Treatment, Pamrevlumab

The FDA has granted a fast track designation to Immune Pharmaceuticals, Inc.’s bertilimumab for the treatment of bullous pemphigoid.

FDA Grants Fast Track Designation to Bullous Pemphigoid Treatment, Bertilimumab

Stay up-to-date on the latest rare disease news by checking out our top 5 articles of the week.

Top 5 Rare Disease News of the Week&mdash;September 2, 2018

The FDA has granted a fast track designation to FCX-013, a gene therapy for the treatment of patients with moderate to severe localized scleroderma.

Gene Therapy for Moderate to Severe Localized Scleroderma Granted Fast Track Designation

The FDA has granted a fast track designation to CX-01 for the treatment of patients over 60 years of age receiving induction therapy for newly-diagnosed AML.

Fast Track Granted to CX-01 for Treatment of Patients Over 60 Newly Diagnosed with AML

The FDA has granted a fast track designation to gene therapy product candidate AAV-CNGB3 for the treatment of achromatopsia caused by mutations in the CNGB3 gene.

FAST: Foundation for Angelman Syndrome Therapeutics

Latest News