Elixirgen Therapeutics Granted FDA Orphan Drug Designation for EXG-34217 for Treatment of Telomere Biology Disorders
The FDA recently granted EXG-34217 Rare Pediatric Disease Designation (RPDD) and Regenerative Medicine Advanced Therapy (RMAT) Designation.
Cure Today2d
The FDA Grants ODD to OPN-6602 For Relapsed/Refractory Myeloma
The FDA has granted orphan drug designation to the oral agent OPN-6602 for the treatment of patients with relapsed or ...
Medscape11h
Dupilumab Under FDA Priority Review for Bullous Pemphigoid
Patients treated with on dupilumab showed significant improvement in sustained BP remission compared with those on placebo.
Healio6d
FDA approves Evrysdi tablets for spinal muscular atrophy
The FDA has approved Evrysdi in tablet form for patients with spinal muscular atrophy aged 2 years and older who weigh more ...
Healio3d
FDA approves Gomekli for neurofibromatosis type 1 with plexiform neurofibromas
The FDA has approved an oral, small molecule MEK inhibitor for adult and pediatric patients 2 years of age and older with ...
Hartford Business9d
FDA approves CT drugmaker’s therapy for rare cancer tumors
Stamford-based SpringWorks Therapeutics, a commercial-stage biotech company developing treatments for rare diseases and ...
Pharmabiz1d
US FDA accepts for priority review Sanofi & Regeneron’s Dupixent sBLA for the targeted treatment of bullous pemphigoid
US FDA accepts for priority review Sanofi & Regeneron’s Dupixent sBLA for the targeted treatment of bullous pemphigoid: Paris Wednesday, February 19, 2025, 12:00 Hrs [IST] The U ...
SpringWorks Therapeutics Announces FDA Approval of GOMEKLI™ (mirdametinib) for the Treatment of Adult and Pediatric Patients with NF1-PN
GOMEKLI is the first and only medicine approved for both adults and children with NF1-PN –– Approval based on positive data ...
The American Journal of Managed Care8d
Risdiplam Tablet for Spinal Muscular Atrophy Receives FDA Approval
The 5 mg risdiplam tablet provides the same efficacy and safety for spinal muscular atrophy as the currently available oral ...
Pharmabiz6d
Roche’s Evrysdi tablet receives US FDA approval for the treatment of spinal muscular atrophy
Roche’s Evrysdi tablet receives US FDA approval for the treatment of spinal muscular atrophy: Basel Friday, February 14, 2025, 13:00 Hrs [IST] Roche, announced that the US Food ...
TMCnet2d
Longeveron® Announces World Health Organization Approval of "laromestrocel" as International Non-proprietary Name for Stem Cell Therapy Lomecel-B™
The U.S. FDA has granted Lomecel-B™ Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation for the treatment of mild Alzheimer’s disease, and Orphan Drug designation, ...
The American Journal of Managed Care8d
FDA Approves Mirdametinib for Neurofibromatosis Type 1 With Plexiform Neurofibromas
Adult and pediatric patients with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas that are not amenable ...