GlobalData on MSN6d
FDA grants orphan drug status to Arbor Biotechnologies’ PH1
The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) and rare paediatric disease designation ...
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 ...
Targeted Oncology6d
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 ...
The American Journal of Managed Care15h
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 ...
Medscape2d
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 unresectable symptomatic plexiform neurofibromas.
The American Journal of Managed Care10h
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 ...
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 ...
PharmaTimes6d
FDA grants Orphan Drug Designation to Ariceum cancer therapy
Ariceum Therapeutics, a private biotech company based in Berlin, has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its proprietary ...
Biodexa Pharmaceuticals PLC: 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 biopharmaceutical company developing a pipeline of innovative products for the treatmen ...
Kauppalehti14h
FDA approves Roche’s Evrysdi tablet as first and only tablet for Spinal Muscular Atrophy (SMA)
"The Evrysdi tablet combines established efficacy with convenience, providing an additional flexible option for SMA management." The approval of the Evrysdi tablet was based on the results of a ...
News Medical on MSN3d
Ariceum Therapeutics receives FDA’s Orphan Drug Designation for targeted radionuclide cancer treatment
Ariceum Therapeutics (Ariceum), a private biotech company developing radiopharmaceutical products for the diagnosis and treatment of certain hard-to-treat cancers, today announced that the U.S. Food ...