FDA

Exciting News From Wash DC!

Laurie Kelley January 18, 2009

Tremendously great news from our nation’s capital. Life is about to change for a lot of people–but it’s nothing to do with a new president. Finally, a product has been approved by the US FDA for those with factor I deficiency!

It’s called RiaSTAP (Fibrinogen Concentrate (Human)), and it’s indicated for the treatment of acute bleeding episodes in patients with congenital fibrinogen deficiency (factor I deficiency), including afibrinogenemia and hypofibrinogenemia. RiaSTAP is a heat-treated, lyophilized fibrinogen powder made from pooled human plasma.

From www.businessspectator.com.au: “The approval by the FDA requires further study after marketing to confirm the product provides meaningful benefits to patients. The safety of Riastap, derived from human blood plasma, ‘appears to be acceptable,’ FDA reviewers said. The drug is already sold in parts of Europe as Haemocomplettan P.”

For more info, check out this site:

http://www.fda.gov/cber/products/riastap.htm

From the NHF website:

“Factor I deficiency is actually a collective term for several rare inherited fibrinogen deficiencies. Fibrinogen may be absent from the blood altogether (afibrinogenemia), present in only very low levels in the blood (hypofibrinogenemia), or measurable in normal quantities but defective (dysfibrinogenemia).

“The incidence of Factor I deficiency is estimated at 1 to 2 per million. It is inherited in an autosomal recessive fashion, which means it affects men and women equally.

“Fibrinogen helps platelets to glue together to form the initial ‘plug’ in response to an injury. Therefore, people with factor I deficiency, have a combined bleeding disorder because both platelets and clotting are abnormal. The severity of the disorder is directly related to the amount of fibrinogen present.

“Afibrinogenemia and hypofibrogenemia are usually diagnosed in newborns who can present with head bleeds, bleeding after circumcision and from the site of the umbilical cord. Easy bruising, nose and mouth bleeds, and soft tissue bleeds are also common. Joint bleeding is relatively uncommon. Women with afibinogenemia have an increased risk of spontaneous abortion. Persons with dysfibrinogenemia may have a disposition to thrombosis.”

It’s great to know there is now something commercially available for patients. This is another great from CSL Behring.
Congratulations to all!

Register with PNS Now!

Laurie Kelley August 23, 2008

It’s been a long time since I’ve seen a withdrawal or recall of factor. About six years ago we had a lot of them, and not everyone got word that their particular product was recalled. Then along came the Patient Notification System (PNS), a program that allows you to register to get alerted via email, telephone, fax or snail mail to any recalls or withdrawals. It’s a good time now to ask yourself if you have registered with PNS.

Here’s why: Just this week we had a voluntary recall of factor.

NHF’s Medical Advisory #407 of August 20 announced that CSL Behring initiated a voluntary recall of four lots of Monoclate-P
that were distributed beginning on August 4, 2008. It reads: “This action is being taken with the knowledge of the United States Food and Drug Administration (FDA). These lots are being voluntarily recalled as a precautionary measure because they do not meet the potency specification when stored for three months at 5 degrees C. No specific safety issues have been identified and neither have adverse incidents been reported. CSL Behring is requesting that the use of these lots be immediately discontinued and the product be returned to the company.”

When you register with PNS, you will be notified only about the product you use, so if you change brands, you may need to update your registration. If you are not registered, you may not hear at all about recalled brands. Please sign up with PNS today. Go to http://www.patientnotificationsystem.org to register. Don’t be the last to know!

Needless to say you should also register with NHF’s eNews, which will keep you updated with recalls and all news related to hemophilia.


Book I Just Read
Fifty Things to Do When You Turn Fifty by Ronnie Sellars (ed). This is a compilation of 50 essays by 50 authors, doctors, celebrities about turning 50 and what you need to know. It was pretty good, a general summary on changes you will face in areas like fitness, attitude, career and friendship. Thing is, most of us at age 50 are already aware of these things! So I guess it’s a good book to read when you are 45. Best part is that all profits are donated. Two stars.

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