As many had predicted following news of a clinical hold on the gene therapy last month, Regenxbio Inc. disclosed receipt of a complete response letter (CRL) regarding its BLA for RGX-121 (clemidsogene lanparvovec) in mucopolysaccharidosis II, an ultrarare neurodegenerative disease in dire need of new therapies.
With the PDUFA date fast approaching for Regenxbio Inc.’s gene therapy RGX-121, the U.S. FDA placed the drug on clinical hold along with another, RGX-111, after preliminary analysis of a single case of neoplasm (specifically, an intraventricular central nervous system tumor) in a participant treated in the phase I/II study with the latter treatment.
Immusoft of CA Inc., a wholly owned subsidiary of Immusoft Corp., has announced that the FDA has granted orphan drug designation to ISP-002, the company’s investigational engineered B-cell therapy for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.
Regenxbio Inc. plans to file a BLA this year seeking accelerated approval for gene therapy candidate RGX-121 to treat young children with mucopolysaccharidosis type II, also known as Hunter syndrome, based on positive data from the phase I/II/III Campsiite trial, which not only hit the biomarker endpoint but also indicated potential systemic benefits.
For the second time in a week, measures of serum neurofilament light chain (NfL) took center stage, this time as Denali Therapeutics Inc. unveiled interim data from the open-label, single-arm phase I/II study testing DNL-310 in children with mucopolysaccharidosis II (MPS II), also known as Hunter syndrome.
Denali Therapeutics Inc.’s extensive update on clinical programs in central nervous system diseases at the start of this year included plans for lead asset DNL-310 in mucopolysaccharidosis II (MPS II), also known as Hunter syndrome – a space where other notable players include such names as Regenxbio Inc. and Takeda Pharmaceutical Co. Ltd.
Denali Therapeutics Inc.’s extensive update on clinical programs in central nervous system diseases at the start of this year included plans for lead asset DNL-310 in mucopolysaccharidosis II (MPS II), also known as Hunter syndrome – a space where other notable players include such names as Regenxbio Inc. and Takeda Pharmaceutical Co. Ltd.
BEIJING – Canbridge Pharmaceuticals Inc. last week won marketing approval in China for its first rare disease drug, the mucopolysaccharidosis II therapy Hunterase (idursulfase beta injection).
BEIJING – Canbridge Pharmaceuticals Inc. last week won marketing approval in China for its first rare disease drug, the mucopolysaccharidosis II therapy Hunterase (idursulfase beta injection).
BEIJING – Canbridge Pharmaceuticals Inc. is to add another source of income after Chinese regulators approved Nerlynx (neratinib) to be marketed for the extended adjuvant treatment of adult patients with early stage HER2-positive breast cancer.
