ARTHEx Biotech Secures the US FDA’s Rare Pediatric Designation (RPD) to ATX-01 for Treating Myotonic Dystrophy Type 1 (DM1)
Shots:
- The US FDA has granted RPD to ATX-01 for treating myotonic dystrophy type 1 (DM1). It has also secured Orphan Drug Designation for the same across the US & EU
- ATX-01 (IV) is being investigated under P-I/IIa (ArthemiR) study for its safety, tolerability, PK/PD & efficacy in patients (18-64yrs.) with classic DM1. It will include a single-ascending dose part where patients will receive one dose and a multiple-ascending dose part where they will receive three doses of ATX-01 or PBO
- ATX-01 is an oleic acid-conjugated antisense oligonucleotide that has shown its ability to elevate MBNL protein expression & reduce toxic DMPK mRNA, addressing key cellular issues like spliceopathy, in DM1 myoblast cell line samples from patients having CTG repeat lengths
Ref: ARTHEx Biotech | Image: ARTHEx Biotech
Related News:- The US FDA Clears ARTHEx Biotech’s IND Application to Commence the P-I-IIa (ArthemiR) Study of ATX-01 for Myotonic Dystrophy Type 1 (DM1)
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
