Altemia Granted Orphan Drug Designation for Treatment of Sickle Cell Disease
Altemia, a new treatment for sickle cell disease (SCD), was granted orphan drug designation.
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The Need to Revisit Treatment Approaches for Gout
The time to revisit treatment approaches for gout is now.
REVERSE Phase III Clinical Trial of GS010 Shows Efficacy in LHON Patients
REVERSE Phase III clinical trial shows efficacy and safety of GS010 in patients with LHON.
Phase 3 OLYMPUS Trial of UGN-101 Shows Promise as UTUC Treatment
Phase 3 OLYMPUS clinical trial releases interim analysis of UGN-101 (MitoGel) for low-grade UTUC.
FDA Grants Orphan Drug Designation to TTAC-0001 for Glioblastoma Multiforme
FDA grants orphan drug designation to PharmAbcine Inc for TTAC-0001, the company’s leading clinical compound, for treatment of GBM.
Catalyst Pharmaceuticals Submits NDA for LEMS Treatment
Catalyst Pharmaceuticals announces its submission of an NDA to the FDA for amifampridine phosphate (Firdapse) for the treatment of LEMS.
FDA Grants Orphan Drug Designation for SMA Treatment
The FDA granted orphan drug designation to Scholar Rock for its lead antibody product candidate, SRK-015, for the treatment of spinal muscular strophy (SMA).
Phase 3 of the NEWTON 2 Study of EG-1962 in aSAH Is Unlikely to Meet Primary Endpoint
Phase 3 of the NEWTON 2 study of EG-1962 in aSAH has been terminated since it is unlikely to meet its primary endpoint.
FDA Grants CYP-001 Orphan Drug Designation
The FDA granted Cynata’s Therapuetics Limited (ASX:CYP) orphan-drug designation for CYP-001, which is the company’s potential treatment for acute graft versus host disease (GvHD).
FDA Grants Orphan-Drug Designation to Angelman Syndrome Treatment
The FDA granted GeneTx Biotherapeutics LLC orphan-drug designation for GTX-101, the company’s potential treatment for Angelman syndrome.
First Patient Treated in Phase 2 PREVENT Study of Exendin 9-39 for Post-Bariatric Hypoglycemia
Eiger announced first patient treated in the PREVENT study, a phase 2, multicenter study of subcutaneous (SC) exendin 9-39 in post-bariatric surgical patients who experience PBH.
Avelumab Shows Positive Response, Safety Profile for Stage IV Merkel Cell Carcinoma
First-line avelumab monotherapy treatments show efficacy in patients with Merkel cell carcinoma (mMCC).
AbbVie Will Not Seek Accelerated Approval for Potential SCLC Therapy
AbbVie won’t seek accelerated approval for Rovalpituzumab Tesirine in third-line relapsed/refractory small cell lung cancer due to the low percentages in efficacy in the TRINITY study.
Low Doses of Cyclosporine A Do Not Prevent Second-Eye Involvement in LHON
A recent study suggests low doses of oral cyclosporine A do not prevent second-eye involvement in adult patients with unilateral Leber’s hereditary optic neuropathy (LHON).
PBI-4050 Phase 2 Trial Demonstrates Efficacy for Alstr
Positive data was anounced regarding PBI-4050, a possible treatment for Alström syndrome.