Beam Therapeutics BEAM announced that the FDA has granted an orphan drug designation to its investigational genome-editing ...

Sanofi SNY announced that the FDA has granted orphan drug designation to its investigational BTK inhibitor, rilzabrutinib, ...

Shuttle Pharma previously received Orphan Drug Designation from the FDA, providing Shuttle Pharma with potential marketing exclusivity upon obtaining FDA approval for treatment of this disease.

Beam Therapeutics' investigational modified cell therapy for treating sickle-cell disease has been granted orphan-drug status by the Food and Drug Administration.

today announced that it has received seven years of orphan-drug exclusive approval from U.S. Food and Drug Administration (FDA) for Ryoncil ® (remestemcel-L) for treatment of steroid-refractory ...

(RTTNews) - Mesoblast (MESO, MSB.AX) announced that it has received seven years of orphan-drug exclusive approval from U.S ... exclusivity means that the FDA will not approve another mesenchymal ...

Crofelemer previously granted orphan-drug designation by the FDA and the ... Napo's crofelemer is FDA-approved under the brand name Mytesi ® for the symptomatic relief of noninfectious diarrhea ...

If a product that has orphan drug designation subsequently receives the first FDA approval for the disease or condition for which it has such designation, the product is entitled to orphan drug ...

Mehta ruled in favor of Latham, upholding FDA approval of the narcolepsy drug ... of Avadel primarily by arguing federal law—the Orphan Drug Act—permitted the FDA to approve Lumryz during ...