Medical Tidbits, FDA approves first drug to treat a rare enzyme disorder in pediatric and adult patients

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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



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