FDA Grants Breakthrough Therapy Designation to Denali’s …
2 days ago · In September 2024, the company announced that it completed a successful meeting with the FDA’s Center for Drug Evaluation and Research (CDER) division outlining a path towards potential accelerated approval for the tividenofusp alfa in Hunter syndrome, also known as mucopolysarcharidosis type II (MPS) syndrome.
DNLI's Hunter Syndrome Drug Gets Breakthrough Therapy …
Denali’s wholly owned program, DNL310 or tividenofusp alfa, is an Enzyme Transport Vehicle-enabled iduronate-2-sulfatase (IDS) replacement therapy in development for MPS II (Hunter syndrome).
FDA Grants Breakthrough Therapy Designation to Tividenofusp …
2 days ago · The FDA granted breakthrough therapy designation (BTD) for tividenofusp alfa (DNL310; Denali Therapeutics) for the treatment of patients with Hunter syndrome (MPS II), a rare genetic disease that can lead to physical, cognitive, and behavioral complications, according to a news release from Denali Therapeutics. 1
Denali Therapeutics Announces U.S. FDA Breakthrough Therapy
1 day ago · About Hunter Syndrome (MPS II) Hunter syndrome (MPS II) is a rare genetic disease that affects over 2,000 individuals in commercially accessible geographies, primarily males, and leads to physical, cognitive, and behavioral symptoms. ... BioSpace is the digital hub for life science news and jobs. We provide essential insights, opportunities and ...
Denali Therapeutics Announces U.S. FDA Breakthrough Therapy …
2 days ago · Hunter syndrome (MPS II) is a rare genetic disease that affects over 2,000 individuals in commercially accessible geographies, primarily males, and leads to physical, cognitive, and behavioral ...
Denali's Hunter Syndrome Drug Gets FDA Breakthrough Status ...
2 days ago · For investors, this news reduces regulatory risk and potentially accelerates revenue timeline, which could catalyze multiple expansion. ... About Hunter Syndrome (MPS II) Hunter syndrome (MPS II) is a rare genetic disease that affects over 2,000 individuals in commercially accessible geographies, primarily males, and leads to physical ...
Denali receives FDA breakthrough status for Hunter syndrome drug
2 days ago · Positive topline data for another orphan disease drug, DNL126 for Sanfilippo Syndrome, has been reported. Jefferies maintained a Buy rating on Denali Therapeutics, highlighting potential Hunter ...
DNLI's Hunter Syndrome Drug Gets Breakthrough Therapy …
2 days ago · Denali obtains Breakthrough Therapy Designation for its experimental candidate, tividenofusp alfa (DNL310), for the treatment of pateints with Hunter syndrome.
Denali Therapeutics gets FDA breakthrough therapy designation …
This designation is in addition to Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation, all previously granted by the FDA for tividenofusp alfa in Hunter syndrome.
Denali’s DNL310 Eyes Accelerated FDA Approval With
Sep 4, 2024 · Denali Therapeutics plans to seek accelerated FDA approval for DNL310 in Hunter syndrome, showing significant biomarker improvements and clinical benefits in ongoing trials.