Drugs in the Pipeline
CPI-613 Gets Orphan Drug Designation for Peripheral T-Cell Lymphoma
This first-in-class drug was previously granted Orphan Drug designation for the treatment of pancreatic cancer, acute myeloid leukemia, Burkitt lymphoma, and myelodysplastic syndromes.
DMD Treatment Gets Orphan Drug, Rare Pediatric Disease Designations
DMD is predominately caused by out-of-frame deletions in the dystrophin gene, which results in absent or defective dystrophin protein, leading to progressive and irreversible muscle function loss.
FDA to Review Gimoti for Diabetic Gastroparesis in Adult Women
The FDA has set a Prescription Drug User Fee Act target date of April 1, 2019.
Positive Results for Novel Osteoarthritis Pain Treatment in Phase 3 Trial
Change in pain and physical function score from baseline were chosen as the co-primary endpoints.
Long-Acting 2-Drug Injectable Noninferior to Oral 3-Drug Combo in HIV Study
The ATLAS study enrolled 570 patients infected with HIV-1; participants were randomized to either the experimental or active comparator arms.
Treatment for Bronchiolitis Obliterans Gains Orphan Drug Status
Bronchiolitis obliterans is the leading cause of morbidity and mortality in the pulmonary transplant population with ≥50% of patients who receive a lung transplant developing the condition within 5 years.
Iclaprim Gets Priority Review for Acute Bacterial Skin/Skin Structure Infections
Clinical data has indicated that the drug exhibits a low propensity for resistance development as well as a favorable tolerability profile.
FDA to Review Midazolam Nasal Spray for Seizure Clusters
The NDA includes data from the Phase 3 ARTEMIS 1 trial which evaluated the intranasal spray in 292 patients.
Elzonris Granted Priority Review for Blastic Plasmacytoid Dendritic Cell Neoplasm
The Company announced results from a Phase 2 trial involving 45 patients with BPDCN earlier this year.
Xolair Designated Breakthrough Therapy for Food Allergies
The FDA designation was supported by data from 7 studies evaluating the safety and efficacy of Xolair against various food allergens (eg, peanut, milk, egg, others).
Gene Therapy Gets Orphan Drug Designation for Severe Genetic Skin Disease
Patients with TGM-1 deficiency may have chronic pronounced scaling of the skin with increased transepidermal water loss, as the TGM-1 enzyme is essential for facilitating the formation of the epidermal barrier, which prevents dehydration.
FDA Grants Orphan Drug Status to Angelman Syndrome Treatment
Delay or lack of speech, seizures, and walking/balance disorders are some of the symptoms associated with AS.
Vicinium Fast-Tracked for Non-Muscle Invasive Bladder Cancer
A Phase 3 trial (Vista) of the treatment is currently taking place; the trial is evaluating the treatment in patients with NMIBC who have previously received 2 courses of BCG and who are now BCG-unresponsive.
Omadacycline Gets FDA Committee Nod for Skin Infections, Pneumonia
The Committee's recommendation was based on data from the omadacycline global development program that included nearly 2000 adults in three Phase 3 studies.
Monoclonal Antibody Granted Orphan Drug Status for Frontotemporal Dementia
There are currently no treatments available for frontotemporal dementia.
Renal and Urology News Articles
- Phosphate Binders Lower Infection-Related Death Risk in HD
- Acute Renal Failure in CKD Patients Less Likely With PCI
- Gout Associated With Increased Risk of Developing Erectile Dysfunction
- More Targeted Sodium Reduction Strategies Proposed
- Cytoreductive Surgery for Metastatic PCa Linked to More Complications
- FDA Recalls Drugs Containing Active Ingredient Valsartan
- Correcting Metabolic Acidosis May Preserve Muscle Mass
- Infection Risk Lower in Dialysis Patients With High, Normal Vitamin D
- Urine Cytology May Not Improve Hematuria Evaluation for Bladder Cancer
- Aggressive Therapy Warranted for Gleason 10 Prostate Cancer
NEPHROLOGY & UROLOGY NEWS
- Acute Kidney Injury (AKI)
- Anemia
- Chronic Kidney Disease (CKD)
- Contrast Nephropathy
- Cardiovascular Disease (CVD)
- Diabetes
- Diabetic Nephropathy
- End-stage Renal Disease (ESRD)
- Hemodialysis
- Hyperphosphatemia
- Hypertension
- Hyperuricemia
- Lupus Nephritis
- Peritoneal Dialysis
- Secondary Hyperparathyroidism (SHPT)
- Transplantation