U.S. Food and Drug Administration Orphan Drug Approvals in 2019

October 2, 2019

The following is a list of U.S. Food and Drug Orphan Drug approvals in the first three quarters of 2019.

FDA Grants Fast Track Designation to Arcturus’ mRNA Therapeutic Candidate for OTC

Rare Daily Staff The U.S. Food and Drug Administration has granted Fast Track designation to Arcturus Therapeutics’ […]

Rare Daily Staff Pharming Group said it has entered into a definitive agreement with Novartis Pharma to […]

Rare Daily Staff The U.S. Food and Drug Administration granted Fast Track designation to Inhibrx’ INBRX-101, an […]

Rare Daily Staff Belite Bio, a clinical stage company focused on advancing novel therapeutics targeting retinal degenerative […]

Rare Daily Staff The European Medicines Agency granted Priority Medicines (PRIME) designation to Rocket Pharmaceutical’s RP-A501, the […]

CRISPR genome editing has the potential to revolutionize the treatment of diseases, but the imprecision of its […]

Rare Daily Staff The U.S. Food and Drug Administration granted priority review to Bristol Myers Squibb’s repotrectinib, […]

Rare Daily Staff Pfizer reported that the pivotal phase 3 BASIS clinical trial evaluating marstacimab has met […]

Rare Daily Staff The U.S. Food and Drug Administration notified PepGen’s that it was placing a clinical […]