Approval for Orphan Drug Status

[Guest guest]

February 26. 2011 - GlaxoKline (NYSE: GSK) announced that the United States

Food and Drug Administration (FDA) granted full approval for PROMACTA®

(eltrombopag), an oral tablet that can raise platelet counts in patients with

the rare blood disorder chronic immune (idiopathic) thrombocytopenic purpura

(ITP) who have had an insufficient response to corticosteroids, immunoglobulins

or splenectomy.

PROMACTA initially received FDA orphan drug designation in May 2008 and

accelerated approval in November 2008 for chronic ITP. The FDA Accelerated

Approval program offers a pathway to gain provisional marketing approval for

therapies that address unmet patient needs. Full approval of the therapy

requires completion of post-marketing clinical trials and commitments that

verify clinical benefit.

" Full approval of PROMACTA was based on clinical studies that provide physicians

and patients with a broader understanding of its treatment effect and safety

profile, " said Stein, MD, V.P. of Medicines Development, GlaxoKline.

" PROMACTA is a testament to how the FDA Accelerated Approval Program supports

development of therapies that meet unmet patient needs. Patients with limited

treatment options gained access to PROMACTA while GSK conducted clinical studies

that yielded additional efficacy and safety data. "

http://www.medicalnewstoday.com/articles/217601.php

***********************************

In a finding that may potentially improve survival from war injuries and

disasters, laboratory researchers report that refrigerated whole blood may have

a shelf life well beyond the current standard of 24 to 48 hours.

" We have found that whole blood retains its clotting properties at least 11 days

under standard refrigeration, " said the study leader, Jobes, M.D., a

cardiothoracic anesthesiologist in the Cardiac Center at The Children's Hospital

of Philadelphia. " If this lab discovery can be confirmed in human subjects, it

may lead to a change in clinical practice, and possibly to improved survival for

massively transfused patients. "

The study appears in the January 2011 issue of the journal Transfusion.

The majority of patients receiving blood transfusions only require specific

components of whole blood, such as red blood cells, plasma and platelets.

However, whole blood may be preferable in specific situations such as infant

heart surgery and combat casualties.

http://www.medicalnewstoday.com/articles/217342.php

**************************

FYI,

Lottie Duthu