Tags : ODD

Regulatory

Seelos’ SLS-005 (Trehalose) Receives EC’s Orphan Drug Designation to Treat

Shots: EMA’s COMP has granted ODD to SLS-005 for the treatment of amyotrophic lateral sclerosis Under ODD, Seelos will get benefit from several incentives such as protocol assistance, reduced regulatory fees and market exclusivity Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the BBB, stabilizes proteins and activates autophagy and has received […]Read More

Regulatory

Vico’s VO659 Receives EC’s Orphan Drug Designation for Spinocerebellar Ataxia

Shots: The EC granted ODD for VO659 for the treatment of spinocerebellar ataxia (SCA). The ODD was based on a EMA’s COMP positive opinion The company previously received ODD in Huntington Disease The company focused on the development of RNA modulating therapies for rare neurological disorders VO659 is an antisense oligonucleotide product, designed to suppress […]Read More

Regulatory

BioLineRx’s Motixafortide (BL-8040) Receives EC’s ODD to Treat Pancreatic Cancer

Shots: The EC has granted ODD to Motixafortide (BL-8040) for the treatment of pancreatic cancer following the positive COMP’s opinion The ODD designation is based on initial data of ongoing P-IIa COMBAT/KEYNOTE-202 study assessing the combination of BL-8040 + Keytruda (pembrolizumab) + CT in patients with m-pancreatic cancer Motixafortide is a selective inhibitor of the […]Read More

Pharma

Taiwan Liposome’s TLC178 Receives EMA’s ODD for Soft Tissue Sarcoma

Shots: The ODD follows P-I/II study assessing TLC178 in patients with advanced malignancies in adults and has resulted in no dose-limiting toxicity till date The EMA’s Orphan Drug Designation (ODD) is granted to the therapies offering life-threatening condition affecting ≤5 in 10,000 persons in EU, with no approved treatment and receiving 10-year of marketing exclusivity […]Read More

Regulatory

Albireo’s A4250 Receives EU Orphan Drug Designation (ODD) for Biliary

Shots: The designation is based on P-III clinical trial assessing A4250 in patients with progressive familial intrahepatic cholestasis (PFIC) Albireo to get 10 years of market exclusivity with additional two years on completion of pediatric investigation plan (PIP) and plans to target A4250 in rare cholestatic liver diseases in 2019 A4250 is an ileal bile acid transporter (IBAT) inhibitor, indicated to […]Read More