Healio1d
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 ...
The American Journal of Managed Care2d
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 ...
UPDATE: SpringWorks Therapeutics Announces FDA Approval of GOMEKLIâ„¢ (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 ...
GlobalData on MSN9d
FDA grants orphan drug status to Arbor Biotechnologies’ PH1
The development comes after the US regulator granted approval for the investigational new drug ... “ABO-101 receiving rare ...
OS Therapies gears up for FDA meeting on cancer drug
NEW YORK - OS Therapies, Inc. (NYSE-A: OSTX), a small-cap biotech firm with a market capitalization of $42 million focusing on cancer treatment solutions, has announced the completion of its Phase 2b ...
Hartford Business Journal3d
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 ...
Targeted Oncology9d
FDA Grants Key Oncology Approvals and Designations in January 2025
On January 7, 2025, the FDA granted priority review to the new drug application for sunvozertinib (DZD9008), an oral EGFR ...
Following Positive Phase 2 Results and Orphan Drug Designation, Biodexa's FAP Drug Receives FDA Fast Track Status
CARDIFF, UK / ACCESS Newswire / February 12, 2025 /Biodexa Pharmaceuticals PLC. (NASDAQ:BDRX), a clinical-stage ...
The American Journal of Managed Care2d
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 ...
The Manila Times3d
FDA approves Roche's Evrysdi tablet as first and only tablet for Spinal Muscular Atrophy (SMA)
Evrysdi tablet can be stored at room temperature and offers the same demonstrated efficacy and safety as the currently ...
Medscape4d
FDA Approves Mirdametinib for Neurofibromatosis Type 1
The kinase inhibitor is now approved for adults and children aged ≥ 2 years with neurofibromatosis type 1 who have ...