BIO recently published the July 2012 version of its quarterly therapeutic newsletters. These six newsletters focus on the therapeutic areas of member companies and include updates from biotech stakeholders around Washington, including Congress, FDA, NIH, and patient organizations.
The therapeutic areas covered by these newsletters are:
Cardiology, Pulmonology, & Blood
Nephrology, Endocrinology, Metabolism, & Gastroenterology
Allergy, Infectious Disease, & Antiviral
Rheumatology, Inflammation, Anesthesia, & Pain
Neurology & CNS
The below is an excerpt from the “Focus on Cardiology, Pulmonology, & Blood” newsletter. To download it in its entirety, please click here. If you have any questions or would like to receive a specific set of therapeutic newsletters, please contact Charles Crain at firstname.lastname@example.org.
NHLBI Funding Announcements
PAR-12-155, Integrative Omics Data Analysis for Discovery in Lung Diseases (R01)– January 26, 2014
PA-12-143, Toward an Improved Understanding of HDL Function (R01)– September 8, 2012
PA-12-110, Getting from Genes to Function in Lung Disease (R01)– September 8, 2012
PAR-11-307, Discovery of Genetic Basis of Mendelian or Monogenic Heart, Lung, and Blood Disorders (X01)– May 15, 2014
PA-09-249, Directed Stem Cell Differentiation for Cell-Based Therapies for Heart, Lung, and Blood Diseases (SBIR [R43/R44])– September 8, 2012
RFA-HL-14-005, Comparison of Strategies to Study Pediatric Pulmonary Vascular Disease Outcomes Using Bioinformatics (U01)– February 22, 2013
FDA Advisory Committee News
On May 23, 2012, the FDA Cardiovascular and Renal Drugs Advisory Committee met to discuss supplemental new drug application (sNDA) 202439/S-002 XARELTO (rivaroxaban), submitted by Janssen Pharmaceuticals, Inc. to reduce the risk of thrombotic cardiovascular events in patients with acute coronary syndrome (ACS) [ST elevation myocardial infarction (STEMI), non-ST elevation myocardial infarction (NSTEMI), or unstable angina (UA)] in combination with aspirin alone or with aspirin plus clopidogrel or ticlopidine.
The materials and minutes from this meeting are available online, as well as a complete transcript. For more information, please click here.
NHLBI Scientists: Cystic Fibrosis Therapy Tested in Young Children
A treatment that benefits adults and older children with cystic fibrosis may not help infants and young children with the disease, a new study reports. The finding could slow the adoption of this therapy in younger children.
Previous studies have found that inhaling a concentrated salt water (hypertonic saline) mist provides some benefits to adults and older children with cystic fibrosis. Because of these earlier findings, the use of hypertonic saline in younger children has been rising. The therapy is now used by about 1 in 5 children under 6 years old with cystic fibrosis. But there isn't clear evidence that the therapy is effective for these young children.
To test whether inhaling hypertonic saline helps young children with cystic fibrosis, a research team led by Dr. Margaret Rosenfeld at Seattle Children's Hospital and Dr. Felix Ratjen at the University of Toronto enrolled 321 participants, ages 4 months to 60 months, at 30 cystic fibrosis care centers across the United States and Canada. Twice daily for 48 weeks, the children inhaled either 7% hypertonic saline or a 0.9% saline mist as a control.
The scientists reported that hypertonic saline was well-tolerated and caused few side effects. However, there was no difference between the 2 saline groups in the rate of acute lung problems that required treatment with antibiotics (an average of 2.3 cases per participant per year). The treatment also didn't improve other clinical measurements such as coughing, respiratory rate, height or weight (the disorder can stunt children's growth).
To learn more about this research, please click here.
BIO Announces FDASIA and JOBS Act Webinars
BIO would like to invite you to participate in our upcoming educational webinar series in September and October on key provisions contained in the Food and Drug Administration Safety and Innovation Act (FDASIA) and the Jumpstart Our Business Startups (JOBS) Act.
The FDASIA webinars, scheduled for September 13 and September 26, will provide information on the intent and goals of the provisions in FDASIA, including Enhanced Communications, NME Reviews, Expanded Accelerated Approval, and Breakthrough Therapies. Industry experts will also discuss implementation issues and timelines.