Genetic medicines developer Modalis Therapeutics Corporation (Tokyo:4883) announced on Friday that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to MDL-101, a potential treatment for congenital muscular dystrophy type 1a (LAMA2-CMD).
The ODD will provide Modalis with certain development incentives, including tax credits, user fee waivers and market exclusivity.
LAMA2-CMD is a rare, life-threatening disease with no approved treatments currently available. MDL-101, which uses Modalis' proprietary CRISPR-GNDM technology, aims to address the underlying cause of the disease by activating LAMA1, the sister gene of LAMA2.
Eisai completes rolling BLA submission for lecanemab-irmb to FDA
TME Pharma secures EUR2.4m grant for NOX-A12 Phase 2 trial
Satsuma Pharmaceuticals and Shin Nippon Biomedical resubmit STS101 NDA to FDA
Henlius and Organon announce FDA acceptance of BLA for denosumab biosimilar, HLX14
I-Mab announces presentation of Phase 1 dose data for givastomig at SITC 2024
Sobi to present emapalumab data for treating macrophage activation syndrome at ACR Conference
Foresee Pharmaceuticals submits US FDA New Drug Application for three-month version of CAMCEVI
Organon adds VTAMA cream to dermatology portfolio with acquisition of Dermavant
Johnson & Johnson reports strong Phase 3 results for TREMFYA in Crohn's Disease
Kind Pharmaceutical's AND017 receives US FDA Orphan Drug Designation
RiboX Therapeutics' RXRG001 Phase I/IIa Study IND application receives US FDA approval
Modalis Therapeutics receives Orphan Drug Designation for MDL-101
EpiVax and CUBRC receive USD2m contract from US FDA
U.P. Oncolytics' oncolytic virus-based therapy granted US FDA Orphan Drug designation
United Therapeutics reaches milestone with 500th lung transplant via ex vivo lung perfusion service