Tags : Orphan Drug Designation

Project ALS’ Prosetin Receives the US FDA’s Orphan Drug Designation

Shots: The US FDA has granted ODD to Prosetin for the treatment of ALS. ODD provides incentives for the development of drugs, biologics, and devices for diseases affecting 200,000 or fewer Americans By obtaining ODD, Prosetin is now eligible for benefits including up to 7yrs. of marketing exclusivity if it receives regulatory approval, exemption from […]Read More

Taysha Gene Therapies’ TSHA-101 Receives the US FDA’s Orphan Drug

Shots: The US FDA has granted ODD and RPD to the Taysha Gene Therapies’ TSHA-101 for GM2 Gangliosidosis. The company expects the therapy to enter the clinic by the end of 2020 The US FDA’s two designations demonstrated the strength of the translational data package supporting TSHA-101 for GM2 Gangliosidosis TSHA-101 is an AAV9-based gene […]Read More

Chugai’s RG6042 Receives MHLW’s Orphan Drug Designation for Huntington’s Disease

Shots: The MHLW has granted Chugai’s RG6042 an ODD for the expected treatment of Huntington’s disease The RG6042 is currently being evaluated in P-III GENERATION HD1 study and is expected to delay the progression of Huntington’s disease as the first disease-modifying therapy RG6042 (ONIS-HTTRx) is an antisense oligonucleotide targeting HTT mRNA and has received FDA […]Read More

OBI Pharma’s OBI-999 Receives the US FDA’s Orphan Drug Designation

Shots: OBI Pharma has initiated the enrollment of P-I/II study at the University of Texas M.D. Anderson Cancer Center to evaluate the therapy in patients with LA/m-solid tumors The objective of the study is to assess the safety & preliminary efficacy profile of OBI-999 in patients with pancreatic, gastric, colorectal and esophageal cancers OBI-999 is […]Read More