EPX-300 of Epygenix Therapeutics and JOTROL of Jupiter has received Orphan Drug Designation for the treatment of Dravet Syndrome and Friedreich’s Ataxia respectively.
Epygenix Therapeutics, Inc., a privately held biopharmaceutical company developing precision medicine for catastrophic and rare forms of genetic epilepsy, has announced that the USFDA has granted Orphan Drug Designation for their drug EPX-300 for the treatment of patients with Dravet Syndrome. Additionally, Jupiter Orphan Therapeutics has also announced that they have received notification from the USFDA that its Orphan Drug Designation request for Trans-Resveratrol Product JOTROL for Treatment of Friedreich’s Ataxia has been granted.
New treatment Approved for adults with relapsed or refractory acute lymphoblastic leukemia by the USFDA
The USFDA has approved Pfizer’s Besponsa (inotuzumab ozogamicin) for the treatment of relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) in adults through a Priority Review and Breakthrough Therapy Designations. Besponsa is a targeted therapy that is thought to work by binding to B-cell ALL cancer cells that express the CD22 antigen, blocking the growth of cancerous cells. The approval is based on the clinical trial results carried out to check for the safety and efficacy of this drug in 326 adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia. Besponsa has also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
