Orphan Drug Designation

PharmaTher’s Ketamine Receives the US FDA’s Orphan Drug Designation for Complex Regional Pain Syndrome

Shots: The US FDA has granted ODD to the Ketamine for the treatment of complex regional pain syndrome (CRPS) which is a rare chronic pain and inflammatory condition following an injury to a limb The ODD follows the US FDA’s ODD of ketamine for the treatment of ALS, granted on Aug 04, 2021 Ketamine acts …

PharmaTher’s Ketamine Receives the US FDA’s Orphan Drug Designation for Complex Regional Pain Syndrome Read More »

C4T’s CFT7455 Receives the US FDA’s Orphan Drug designation for the Treatment of Multiple Myeloma

Shots: The US FDA has granted ODD to CFT7455 for the treatment of MM. The FDA grants ODD to drugs & biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases, or conditions that affect fewer than 200,000 people in the US In Jun’21, the company has initiated a …

C4T’s CFT7455 Receives the US FDA’s Orphan Drug designation for the Treatment of Multiple Myeloma Read More »

LEXEO Therapeutics’ LX2006 Receives the US FDA’s Rare Pediatric Disease Designation and Orphan Drug Designation to Treat Friedreich Ataxia

Shots: The US FDA has granted RPD and ODD to LX2006 for the treatment of FA. The company plans to start a P-I/II clinical trial of LX2006 in patients with cardiomyopathy associated with FA in 2021 Additionally, if BLA for LX2006 is approved, a company may be eligible to receive PRV that may be sold …

LEXEO Therapeutics’ LX2006 Receives the US FDA’s Rare Pediatric Disease Designation and Orphan Drug Designation to Treat Friedreich Ataxia Read More »

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

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 …

Seelos’ SLS-005 (Trehalose) Receives EC’s Orphan Drug Designation to Treat Amyotrophic Lateral Sclerosis Read More »

Passage Bio’s PBKR03 Receives EC’s Orphan Drug Designation for Krabbe Disease

Shots: The EC has granted ODD for PBKR03 for the treatment of Krabbe disease. The ODD was based on an EMA’s COMP positive opinion The designation provides potential benefits to the company, which include clinical protocol assistance, reduced regulatory fees, research grants, and up to 10 years of market exclusivity following regulatory approval The company …

Passage Bio’s PBKR03 Receives EC’s Orphan Drug Designation for Krabbe Disease Read More »

Taysha Gene Therapies’ TSHA-101 Receives the US FDA’s Orphan Drug Designation and Rare Pediatric Disease Designation for GM2 Gangliosidosis

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 …

Taysha Gene Therapies’ TSHA-101 Receives the US FDA’s Orphan Drug Designation and Rare Pediatric Disease Designation for GM2 Gangliosidosis Read More »

Kiniksa’s Rilonacept Receives the US FDA’s Orphan Drug Designation to Treat Pericarditis

Shots: The US FDA has granted ODD for Kiniksa’s Rilonacept to treat pericarditis, which includes recurrent pericarditis. The company plans to submit sBLA in recurrent pericarditis to the US FDA in late 2020 Earlier, the company has reported the results of P-III RHAPSODY study that met its all 1EPs & 2EPs showing that the therapy …

Kiniksa’s Rilonacept Receives the US FDA’s Orphan Drug Designation to Treat Pericarditis Read More »