Tags : Orphan Drug Designation

Pharma Regulatory

AstraZeneca and MSD’s Selumetinib Receive MHLW’s Orphan Drug Designation for

Shots: The ODD designation follows P-I/II SPRINT Stratum 1 study assessing Selumetinib (bid) as monothx. in pediatric patients with NF1-related inoperable PNs The P-I/II SPRINT Stratum 1 study demonstrated a reduction in the size of tumors with 66% ORR in children. AstraZeneca and MSD are co-developing and co-commercializing selumetinib which received the US FDA’s approval […]Read More

Regulatory

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