Today, the U.S. Food and Drug Administration approved Kanuma (sebelipase alfa) as the first treatment for patients with a rare disease known as lysosomal acid lipase (LAL) deficiency.
Patients with LAL deficiency (also known as Wolman disease and cholesteryl ester storage disease [CESD]) have no or little LAL enzyme activity. This results in a build-up of fats within the cells of various tissues that can lead to liver and cardiovascular disease and other complications. Wolman disease often presents during infancy (around 2 to 4 months of age) and is a rapidly progressive disease. Patients with Wolman disease rarely survive beyond the first year of life. CESD is a milder, later-onset form of LAL deficiency and presents in early childhood or later. Life expectancy of patients with CESD depends on the severity of the disease and associated complications. Wolman disease affects one to two infants per million births, and CESD affects 25 individuals per million births. read more
University of Washington to Lead U.S. Expansion of Medtronic’s HeartRescue Project
DUBLIN – December 8, 2015 – Medtronic (NYSE: MDT) today announced that the University of Washington and King County (Seattle) Emergency Medical Services will lead the U.S. expansion of the HeartRescue Project, a successful Medtronic Philanthropy partnership launched in five states in 2010 that brought together some of the nation’s leading resuscitation experts to improve survival rates for Sudden Cardiac Arrest (SCA). read more
Phase 3 Results for Zydelig® With Bendamustine and Rituximab for Relapsed Chronic Lymphocytic Leukemia (CLL) Presented at American Society of Hematology Annual Meeting
ORLANDO, Fla.–(BUSINESS WIRE)–Dec. 8, 2015– Gilead Sciences, Inc. (Nasdaq: GILD) today announced results from a prespecified interim analysis of a Phase 3 study (Study 115) evaluating Zydelig® (idelalisib) in combination with bendamustine and rituximab (BR) for patients with previously treated CLL. The analysis found a 67 percent reduction in the risk of disease progression or death (progression-free survival, PFS) in patients receiving Zydelig plus BR compared to BR alone (hazard ratio (HR) = 0.33; 95 percent CI: 0.24, 0.45; p<0.0001). Additionally, all secondary endpoints, including overall survival (OS), achieved statistical significance in this interim analysis. Detailed results were presented today during the late-breaking abstracts session at the Annual Meeting of the American Society of Hematology (ASH) in Orlando, Florida (#LBA-5). read more
New Data Show ELOCTATE® and ALPROLIX® May Help Control Target Joint Bleeds in People with Hemophilia A and B
ORLANDO, Fla.–(BUSINESS WIRE)–New data demonstrate ELOCTATE® [Antihemophilic Factor (Recombinant), Fc Fusion Protein] (marketed as ELOCTA® in Europe) and ALPROLIX® [Coagulation Factor IX (Recombinant), Fc Fusion Protein] may effectively manage target joint bleeding and maintain low annualized bleeding rates (ABRs) in people with severe hemophilia A and B. The data, which were presented by Biogen (NASDAQ:BIIB) and Swedish Orphan Biovitrum AB (publ) (Sobi) (STO: SOBI) at the 57th American Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Fla., continue to reinforce the value of extended interval prophylactic dosing of ELOCTATE and ALPROLIX. read more
Novo Nordisk files for regulatory approval of faster-acting insulin aspart in the US for the treatment of type 1 and 2 diabetes
Bagsværd, Denmark, 9 December 2015 – Novo Nordisk today announced the submission of the New Drug Application (NDA) for faster-acting insulin aspart to the US Food and Drug Administration (FDA). Faster-acting insulin aspart is a mealtime insulin for improved control of postprandial glucose excursions and has been developed for the treatment of people with type 1 and type 2 diabetes.
The filing of faster-acting insulin aspart is based on the results from the ‘onset’ clinical trial programme which involved around 2,100 people with type 1 and 2 diabetes. In the onset programme, people treated with faster-acting insulin aspart achieved improvements in postprandial control versus NovoLog® (marketed as NovoRapid® outside the US) and an HbA1c reduction on par with NovoLog®. For people with type 1 diabetes, faster-acting insulin aspart results from the double-blinded onset 1 trial showed statistically significantly greater HbA1c reduction when dosed at mealtime or similar HbA1c reduction when dosed 20 minutes after a meal compared to NovoLog®. Across the onset trials, faster-acting insulin aspart had a safe and well tolerated profile, with the most common adverse event being hypoglycaemia, similar to the levels observed with NovoLog®.
“We are happy to be able to file faster-acting insulin aspart for regulatory approval in the US and have the opportunity to address unmet medical needs for people requiring improved blood glucose control around meals,” said Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk. “Onset 1 shows that faster-acting insulin aspart has the potential to offer improved postprandial glucose and either an additional reduction of HbA1c or added flexibility compared with NovoLog®.”
Novo Nordisk intends to make faster-acting insulin aspart available in the prefilled delivery device FlexTouch®.
Pfizer Receives U.S. FDA Approval of New QuilliChew ER™ (methylphenidate hydrochloride) extended-release chewable tablets CII
Extended-Release Is Now Chewable: First and Only Long-Acting Chewable Methylphenidate Treatment for Attention Deficit Hyperactivity Disorder (ADHD) in patients ages 6 years old and above read more
Baxalta Receives FDA Approval for VONVENDI, the First and Only Recombinant Treatment for Adults Affected by Von Willebrand Disease
- von Willebrand disease (VWD) is a genetic disorder that results in impaired clotting with limited treatment options today
- VONVENDI [von Willebrand factor (Recombinant)] represents the most significant therapeutic advancement for the treatment of adults with VWD in more than a decade, offering a new approach to treat the disease
- In clinical studies, VONVENDI demonstrated 100 percent treatment success in resolving bleeding episodes with a median of only two infusions to treat severe bleeds read more
The U.S. Food and Drug Administration (FDA) has confirmed that the organization has received SciBase’s application for a pre-market approval (PMA) of the Nevisense device for the US market.
“We have been working with the clinical studies and documentation that the FDA requires for several years. It has been a long process that culminated in the completion of the application during this quarter. The US market is the world’s largest and represents a significant opportunity for SciBase. Even a limited penetration of our method and Nevisense has the possibility to transform our business”, says Simon Grant, CEO of SciBase.
Bristol-Myers Squibb Completes Previously Announced Acquisition of Cardioxyl Pharmaceuticals, Inc.
The transaction includes full rights to Cardioxyl’s lead asset CXL-1427, a novel nitroxyl (HNO) donor (prodrug) in Phase 2 clinical development as an intravenous treatment for acute decompensated heart failure (ADHF).
Bristol-Myers Squibb Establishes Center for Molecular Synthesis in New Collaboration with Princeton University
PRINCETON, N.J.–(BUSINESS WIRE)–Bristol-Myers Squibb Company (NYSE:BMY) and the Department of Chemistry at Princeton University today announced a new research collaboration, which includes the establishment of the Center for Molecular Synthesis (BMS-CMS). The agreement creates opportunities for scientists at Princeton University and Bristol-Myers Squibb to collaborate on top-flight synthetic chemistry research, leveraging the two sites’ close proximity to foster a robust exchange of scientific ideas. read more
Flagship Ventures Launches Rubius To Develop Red-Cell Therapeutics™ Following 18 Months of Technology Development, Company Advances Breakthrough Therapeutic Platform with $25M of Initial Capital
CAMBRIDGE, Mass., Dec. 9, 2015 /PRNewswire/ — Flagship Ventures, a leading innovation and venture firm focused on healthcare and sustainability, announced it has launched Rubius Therapeutics, to develop functionalized red blood cells for the treatment of autoimmune conditions, metabolic diseases, cancer, and other serious diseases. Flagship has made an initial capital commitment of $25 million to enable Rubius to enter clinical testing of its novel therapeutic modality.read more
Nektar Announces First Patient Dosed in Phase 1/2 Clinical Study of NKTR-214, a CD122-Biased Immuno-Stimulatory Cytokine PRNewswire/ — Nektar Therapeutics (NASDAQ:NKTR) today announced that dosing has commenced in the Phase 1/2 clinical study evaluating the efficacy and safety of NKTR-214 in the treatment of solid tumors. NKTR-214 is a novel CD122-biased cytokine designed to preferentially activate the beta and gamma sub-units of the IL-2 receptor in order to proliferate tumor-killing T cells within the body (CD8-positive effector T cells and natural killer T cells) without stimulating regulatory T cells (CD4-positive T cells). read more