Hemophilia B

What are the future of treatments for hemophilia?

The future for our friends living with Hemophilia disorder is very exciting.

"Hemophilia may be "the royal disease," but for most commoners, it's only just a drag.

Even if the sufferer can avoid major blood loss, but the unseen internal bleeding can cause long-term damage to the joints and organs.

Yet the last 40 years have seen several changes in hemophilia treatment, and Biogen Idec (BIIB) is preparing an FDA filing for a new drug that it says will bring another one. "Whenever these products are approved, they will represent a real benefit to patients for the first time in 15 years," Biogen R&D head Douglas Williams told IBD.

For the last few decades, the hemophiliacs have been treated by infusion with blood products which containing the missing clotting factors (factor 8 for hemophilia A and factor 9 for B). These used to be derived from human plasma, but this turned out to carry a very serious risk of disease.

For that reason, drug makers more recently developed "recombinant" products created from the animal or artificial sources. In hemophilia A, which is a roughly $5 billion market, the leading drug is Baxter International's (BAX) Advate; other recombinant products include Bayer's (BAYRY) Kogenate and Pfizer's (PFE) Xyntha. Filtering process for plasma has also improved, so Baxter also still sells its plasma product Hemofil.

Prophylactic Treatment

A less number of patients develop antibodies, or "inhibitors," to these products.

In the clinical testing, more than one drug candidate has been sunk when too many patients developed inhibitors. Novo Nordisk (NVO), however, has developed a product called NovoSeven, which successfully treats many patients who have inhibitors to factor 8 products.

Safety improvements also led to the rise of prophylactic treatment for hemophilia. Patients used to just get treated in response to bleeding episodes, but many patients nowadays infuse themselves every other day to prevent bleeding episodes from occurring in the first place. Only about 35% of adult patients do this, however, because it's a tough regimen. This isn't just a little injection like a diabetic with insulin; patients have to find a vein for an IV infusion that can take around an hour. So, much of the current pipeline is focused on making the IV regimen easier, particularly by making it less frequent.

That's where Biogen's long-acting recombinant factor 8 fusion protein, or rFVIIIFc, comes in. In the phase-three results released Oct. 31, 30% of subjects successfully prevented bleeding episodes by infusing just once every five days. None developed inhibitors. Despite the improvement on the existing methods, ISI Group analyst Mark Schoenebaum was underwhelmed. "However, we estimate that 50% of the patients got to at least every four days based on the reported 3.5-day median." Biogen also reported great results for the drug in hemophilia B, which is less than a $1 billion market but one with much less competition. That market is dominated by Pfizer's BeneFix.

Demand Is Big Question

A very big question, however, is just how many patients are clamoring for a new drug. The Doctors who work with hemophiliacs say the bad experience with plasma products left many of them wary of new drugs. But Dr. Amy Shapiro, head of the Indiana Hemophilia & Thrombosis Center, says younger patients don't have those memories. They could also benefit from a longer-acting product, because it's more harder to find a vein in a child patient. "They may require a venous-access device to achieve the number of infusions they need," Shapiro told IBD. "That in itself is a surgery that can have side effects and complications. If we can avoid that by having longer-acting products and get more patients on prophylactics on peripheral venopuncture instead of using a device, that would be really a great achievement."

Article Source: http://www.articlesbase.com/diseases-and-conditions-articles/what-are-the-future-of-treatments-for-hemophilia-6386220.html

Author: adammark1