Drugs in the Pipeline
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 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.
The FDA has set a Prescription Drug User Fee Act target date of April 1, 2019.
Change in pain and physical function score from baseline were chosen as the co-primary endpoints.
The ATLAS study enrolled 570 patients infected with HIV-1; participants were randomized to either the experimental or active comparator arms.
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.
Clinical data has indicated that the drug exhibits a low propensity for resistance development as well as a favorable tolerability profile.
The NDA includes data from the Phase 3 ARTEMIS 1 trial which evaluated the intranasal spray in 292 patients.
The Company announced results from a Phase 2 trial involving 45 patients with BPDCN earlier this year.
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).
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.
Delay or lack of speech, seizures, and walking/balance disorders are some of the symptoms associated with AS.
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.
The Committee's recommendation was based on data from the omadacycline global development program that included nearly 2000 adults in three Phase 3 studies.
There are currently no treatments available for frontotemporal dementia.
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- Cardiovascular Disease (CVD)
- Diabetic Nephropathy
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- Lupus Nephritis
- Peritoneal Dialysis
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