Pharma News

Novimmune, announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to NI-0501 for the treatment of patients with primary HLH with refractory disease, or with recurrent or progressive disease during conventional therapy. NI-0501 is a fully human, anti-interferon-gamma (IFNγ) monoclonal antibody discovered and developed by Novimmune.

Takeda Pharmaceutical Company Limited and Frazier Healthcare Partners announced the formation of Outpost Medicine, a biopharmaceutical company focused on the development of new treatments of urologic and gynaecologic diseases and disorders.

Aralez Pharmaceuticals Inc. announced that it has resubmitted to the U.S. Food and Drug Administration ("FDA") the New Drug Application ("NDA") for its investigational candidate, YOSPRALA™ (PA32540/PA8140) for the secondary prevention of cardiovascular disease in patients at risk for aspirin-induced gastric ulcers.

ResApp Health Limited , announced a positive outcome from its recent Pre-Submission Meeting with the United States Food and Drug Administration (FDA) regarding ResApp’s diagnostic mobile software application, ResAppDx.

Aeglea BioTherapeutics, Inc., announced the U.S. Food and Drug Administration (FDA) has accepted the company’s investigational new drug (IND) application for AEB1102 for the treatment of Arginase I deficiency, a urea cycle disorder that results in elevated levels of the amino acid arginine, which can lead to a range of complications including intellectual disability and spasticity.

Celator Pharmaceuticals, Inc. announced positive results from the Phase 3 trial of VYXEOS™ (cytarabine: daunorubicin) Liposome for Injection (also known as CPX-351) in patients with high-risk (secondary) acute myeloid leukemia (AML) compared to the standard of care regimen of cytarabine and daunorubicin known as 7+3. The trial met its primary endpoint demonstrating a statistically significant improvement in overall survival.  Data will be submitted for presentation at the American Society of Clinical Oncology 2016 Annual Meeting.

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Genentech, announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) and granted Priority Review for atezolizumab (anti-PDL1; MPDL3280A) for the treatment of people with locally advanced or metastatic urothelial carcinoma (mUC) who had disease progression during or following platinum-based chemotherapy in the metastatic setting, or whose disease worsened within 12 months of receiving platinum-based chemotherapy before surgery (neoadjuvant) or after surgery (adjuvant). Urothelial carcinoma accounts for 90 percent of all bladder cancers and can also be found in the renal pelvis, ureter and urethra.

ProNAi Therapeutics, Inc. announced that its oncology drug candidate PNT2258 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of diffuse large B-cell lymphoma (DLBCL).

GW Pharmaceuticals plc, announced the positive results of the first pivotal phase 3 study of its investigational medicine Epidiolex (cannabidiol or CBD) for the treatment of Dravet syndrome. In this study, Epidiolex achieved the primary endpoint of a significant reduction in convulsive seizures assessed over the entire treatment period compared with placebo (p=0.01). Epidiolex has both Orphan Drug Designation and Fast Track Designation from the US Food and Drug Administration (FDA) in the treatment of Dravet syndrome, a rare and debilitating type of epilepsy for which there are currently no treatments approved in the US.

KemPharm, Inc. announced that a joint meeting of the Anesthetic and Analgesic Drug Products Advisory Committee and Drug Safety and Risk Management Advisory Committee of the U.S. Food and Drug Administration (FDA) has been scheduled for May 5, 2016, to review KemPharm’s New Drug Application (NDA) for KP201/APAP, its lead investigational drug candidate for the short-term management of acute pain.