Pharming Group N.V., a biotech company headquartered in Leiden, the Netherlands, has received “orphan drug” designation from the U.S. Food and Drug Administration (FDA) for a new drug designed to treat patients with a rare deficiency of the clotting protein fibrinogen.
Recombinant human fibrinogen (rhFIB), which is being developed using Pharming’s protein production technology and marketed under the brand name Rhucin®, is for treatment of bleeding episodes in patients with low levels of fibrinogen. The disorder can either be “acquired” or be genetically inherited. Some examples of acquired triggers for low fibrinogen are patients undergoing trauma or surgery, or who have liver disease, sepsis or cancer. The inherited conditions are also known as factor I deficiency, congenital afibrinogenemia or dysfibrinogenemia (although dysfibrinogenemia can also be acquired).
The genetic forms are conditions in which either a lack of or dysfunction in fibrinogen causes excessive bleeding. In its October 11, 2007 announcement, Pharming states that approximately 100,000 patients in the U.S. develop bleeding episodes each year due to a fibrinogen deficiency.
The current treatment for patients with fibrinogen deficiency is cryoprecipitate, or “cryo.” Cryo, drawn from partially thawed, human-derived frozen plasma, is the standard therapy for afibrinogenemia and dysfibrinogenemia. It contains fibrinogen and other clotting proteins, such as von Willebrand factor and factors VIII and XIII. Since there are no drugs available to treat patients with fibrinogen deficiencies, cryo remains the currently recommended treatment for afibrinogenemia, dysfibrinogenemia and factor XIII deficiency by the National Hemophilia Foundation’s Medical and Scientific Advisory Council. However, it is generally known that use of cryo is not ideal, since it has not been treated to reduce viral transmission. Because Pharming’s drug is a recombinant product and not derived from plasma, it carries less theoretical risk of viral transmission.
“Pharming’s development strategy for rhFIB will utilize the successful model of recombinant blood clotting proteins to control bleeding for various genetic and acquired deficiencies,” said Dr. Francis Pinto, Chief Executive Officer of Pharming. “As Rhucin® nears its first market authorization, rhFIB represents another avenue of growth for Pharming and demonstrates our strength in developing therapeutic recombinant proteins for unmet medical needs,”
The FDA’s Office of Orphan Products Development grants orphan drug status, which means the drugs go through an expedited review process. The designation allows for accelerated approval, so that much-needed drugs or biologics become available to patients with rare, life-threatening diseases who need effective treatments. More than 1,400 drugs and biologics have been designated as orphan drugs. Since 1983, more than 250 of them have been approved for marketing.
Source: Pharming Group press release dated October 11, 2007