Another Product Debut: FVIII This Time!

We were hoping for an August approval, but it seems the FDA is making Biogen Idec’s day. Two products approved in two and a half months! Both with extended half-life. Eloctate [Antihemophilic Factor (Recombinant), Fc Fusion Protein] is the newest factor VIII product. 

A new era of hemophilia treatment has begun. I read somewhere that the last truly “new” product that was introduced was Advate, in 2002. True?

The hemophilia industry is big, with much at stake. The introduction of two new products, with distinct and so far unique,  differences, may cause a shake up. And more products, with longer half-lives included, are coming.

More products give us more choice.  But you have to know what it is you are choosing between.  I have to say I don’t think hemophilia parents and patients are as well informed as they should be. I just conducted a recruitment of patients and caregivers this past week, looking for those who use “plasma-derived” products ONLY. I couldn’t believe the number of people who called to participate who use recombinant. They asked: what does plasma-derived mean? Isn’t the product I am using plasma-derived because it contains Albumin? Or worse, arguing with me that a recombinant product is plasma-derived.

We’ve written so many articles describing the difference between the two categories. All factor is either plasma-derived (originates from human blood) or recombinant (from human genes in the lab). The new product is a recombinant product manipulated to last longer in the blood—basically through a longer half life.

One savvy mom I’ve known for year, who has a son with the World’s Most Stubborn Inhibitor, wrote this to me:

It’s important for Biogen Idec to make clear that this product has not been tested in Previously Untreated Patients (PUPs), and the risk of inhibitors is highest in this group.
Biogen Idec will be opening up a clinical trial for PUPs and I am encouraging anyone who wishes to use the Alprolix (long-lasting recombinant FIX) in a previously untreated patient, to wait and enroll in that trial, so Biogen can gather the appropriate data to understand how safe the product will be in PUPs.
Please always speak with your HTC hematologist to learn which product is best for you or your loved one. (And I don’t usually use underlines in my writing! Yeah, it’s that important)

To learn more about Eloctate, visit:

http://www.biogenidec.com/therapies_eloctate.aspx?ID=20259
Good Book I Just Read
The Right Stuff by Thomas Wolfe [Kindle]
The gripping story of the origins of the space race, and the American men who volunteered to become the first Americans in space. The Mercury astronauts, made heroes by the press and NASA, possessed the character and qualities that made America proud. The book covers a lot of ground, from what it’s like to sit in the cockpit, going to Mach 1, the speed of sound, the tough men who were test pilots, the culture of the test pilots, and eventually the program to put a man in space. It’s a great story (and the movie is superb) and you’ll learn a lot. Wolfe’s flamboyant, hyperdramatic and  over-the-top style just wore on me terribly and I had a hard time eventually getting through this. Way too many exclamation points per page! Great story, odd writing though many consider Wolfe a national literary treasure. Three stars/five.

The [Infusion] Shot Heard Round the World

Unless you were watching the new streaming releases of Season 6 of Breaking Bad on Netflix (for which I might excuse you) or were abducted by aliens (for which I will not) you must have heard the news….. the first extended half-life factor has been approved for commercial sale. It’s the hemophilia B drug APROLIX. The news was announced at the HFA meeting in Tampa last Saturday, and it truly is the Shot Heard Round the World (an appropriate reference to the American Revolution). This ain’t just a new product, folks; it could shake up the industry. Physicians are watching; patients are watching; Wall Street is watching. What will the “uptake” be? 

Uptake is how many patients will switch to using a new product. The hemophilia community tends to be a “wait and see” creature, perhaps due to our devastating experiences of the 1980s. When Advate was launched in 2002 (yes, drugs are launched, like ships), investors predicted a rosy 20% uptake, but initially it was more like 12%. As the risk-takers who switched early reported good results, more and more patients switched. But Aprolix is truly a different kind of drug. Not just a new generation, it is a new type of hemophilia drug: an extended half-life, meaning fewer infusions for the same results. 

And Hemophilia B has not had a buffet of drugs to try like hemophilia A. There was only one recombinant (you all know what that means, right?) FIX for years, until October 2013, when Rixubis was approved. With extended half-life factor, prepare for some major shifts in our landscape. Much may depend on the price, which no one is talking about. If it’s too high, will insurance cover it? 

And what about individual results? Will each patient respond well? Advate had major hiccups when there appeared to be an increase in inhibitor formation in users. Was this due to higher scrutiny of inhibitors with a new product? The initial scare seemed to be due to increased scrutiny. And these are among the most scrutinized things on the planet.

And lest we get too carried away, don’t forget there are other products in the pipeline by other companies, some extended half-life (the products, not the companies, though we hope they are long lasting too). Basically, the news about Aprolix marks the beginning of a new era in hemophilia. Maybe a Revolution, maybe the Renaissance. A Hemophilia Renaissance. 
Below, an article in its entirety about the news….
U.S. FDA approves Biogen’s hemophilia B drug
Alprolix
Biogen Idec Inc has won U.S.
approval for its long-acting hemophilia B treatment Alprolix, the U.S. Food and
Drug Administration said on Friday.
Hemophilia B is a rare, inherited disorder in
which a person’s blood does not clot properly, which can lead to prolonged
bleeding and bruising. It affects about 4,000 people in the United States and
25,000 worldwide.
Alprolix is a bioengineered version of the blood
coagulation factor IX, a protein needed for normal blood clotting. Biogen is
developing the drug in partnership with Swedish Orphan Biovitrum AB. It
received approval in Canada a week ago.
Hemophilia drugs in general must be infused two
to three times a week to prevent bleeding episodes. Biogen’s drug needs to be
administered once a week or once every 10 to 14 days.
The company is also developing a long-lasting
drug, Eloctate, for Hemophilia A, a more common form of the disease, that
affects about 20,000 people in the United States. Patients with hemophilia A
lack or have reduced levels of coagulation factor VIII.
Current treatments for hemophilia B generate
about $1 billion a year, according to Biogen. The market for hemophilia A
therapies is worth about $6 billion.
Eloctate would compete with Baxter International
Inc’s drug Advate. Baxter is seeking to develop its own long-acting version but
Biogen is expected to be first to the market.
Alprolix is expected to generate sales of $286
million by 2019, according to Thomson Reuters data. Eloctate is expected to
generate sales of $1.1 billion.
(Reporting by Toni Clarke in Washington; Editing
by David Gregorio)  Fri Mar 28,
2014 5:11pm EDT
For more info:
http://www.alprolix.com/

So Long! Extended Haf-Life Factor Sets the Stage

You’ve probably heard about Biogen Idec’s clinical studies of factor with extended half-lives. They aren’t the only ones investigating. CSL Behring just announced that they are starting a global phase II/III, multi-center study to assess the safety, efficacy and pharmacokinetics of recombinant fusion protein.

This recombinant fusion protein links coagulation factor IX with recombinant albumin, called “rIX-FP.” rIX-FP is indicated for the prophylaxis and treatment of bleeding episodes in patients with factor IX deficiency.

Of course, extended half-life factor means potentially fewer injections for patients, and may enable or enhance prophylactic treatment, improving quality of life for patients.

So many patients worldwide have been hoping for gene therapy–a “cure”–but it seems that the next great thing that will come along just might be extended half-life factor. We’re not sure yet which company will deliver first, but keep watching. These are exciting times and the long acting factor race is on!

Book I Just Read
The Hunger Games by Suzanne Collins (Kindle edition)

This best selling novel takes place in post-apocalyptic America, now called–for no reason disclosed– Panem. In fact, there are a lot of things that happen for no apparent reason in this book. Quick synopsis: the powers that be dictate that one teen from every district must compete to the death in the Hunger Games, in retribution for an uprising that happened a long time ago. To the victor goes fame and food to their district. The whole event is televised–reality TV where kids get killed. The heroine narrates the story, and has a pretty flat tone throughout. Think “The Truman Show” meets “Mad Max Beyond Thunderdome” meets The Olympics meets “Rollerball” and you get a great idea what the book is about. It’s not terribly original; the writing is mediocre and the story pretty predictable, characters shallow with no depth of thought or emotion. After killing her first kid, arrow through the throat, the 16-year-old narrator with the flat affect seems more concerned about whether the boy she is with loves her. Someone recommended this to me, but neglected to tell me it’s teen romance fiction, with a lot of killing. Read it if you want to kill time; it’s a few steps up from a comic book. If you want fantasy with substance and depth, try Tolkein. The book seems to be meant for a movie, and reads like a screenplay; I somehow think the movie will be better as a result, coming in March. Two stars/five.

Biogen Idec Hemophilia’s First Advocacy Advisory Summit

Every now and then I am reminded how wonderful it is to be a part of the hemophilia community, if you can say such a thing knowing it means your loved one has hemophilia! I spent the day on Friday with a group of dedicated colleagues, some I have known for 18 years, so many I admire. Our hemophilia community is filled with such people, all trying to improve the lives of those with the condition.

So I was honored to be selected to attend Biogen Idec Hemophilia’s first Advocacy Advisory Summit on Friday, in Research Triangle Park, North Carolina. Arriving to much-welcomed balmy weather, I caught up with a cast of great people and friends, including executive directors Lynne Capretto (Ohio), Sue Cowell (N. Carolina), Kevin Sorge (New England), Kelly Waters (Virginia), Merlin Wedepohl (N. Cal), Kristian Prill (Washington), Greg Mermilliod (S. Cal), Vicki Jackson (Alabama), Sean Hubbert (Central Cal), Ken Trader, Joe Kleiber and Michelle Rice of NHF, and Susan Swindle of HFA. And special guests Dolly Shinhat-Ross of WFH and Margo Price of ISTH.

We were invited to “meet” Biogen Idec, learn about their clinical trials, and tour their plant. Jessica Swann, whom many in our community know from her days as executive director of Hemophilia Association of San Diego, and then her days at Avigen and Bayer, is now Associate Director, Global Product and Market Development at Biogen Idec Hemophilia. She joins Dr. Glenn Pierce, Senior Vice President and Chief Medical Officer, who was twice president of NHF, formerly of Avigen and Bayer and who also formerly had hemophilia!

You may not recognize the name Biogen Idec: they do not have a commercial hemophilia product currently, but have two in the pipeline, in phase III clinical studies. You may have heard though about “extended half-life factor.” This is one company that is working on this.

When factor is infused into the blood stream to stop a bleed, it has a “half life.” Just as food is broken down and used, and you need to eat again, factor is used to stop bleeding and eventually broken down (degraded) in the blood stream and absorbed back into the body. The half life is how long it takes for 50% (half) of the factor to be used up. The idea behind extended half-life factor is to “catch” the factor when it is absorbed and return it back into the bloodstream before it gets degraded so it can still be used. Its half life then theoretically goes from 12-18 hours (currently normal half lives for factor) to up to 2-3 days or more. This may allow the protective effect of factor VIII and factor IX to last much longer than they currently last.

As a group, we tried to imagine what that would mean for patients: greater quality of life, fewer infusions, less stress and worry, greater protection. Make no mistake: extended half-life factor could be the next great thing in hemophilia.

But who is this company Biogen Idec? I am familiar with Biogen as Tommy’s dad, Kevin, used to work there in 1982, back when it all fit into one building in Cambridge, Massachusetts. Tommy was born in 1987. If you can believe this, Kevin was working on recombinant factor VIII before we got married, and with no knowledge that we would one day have a son with hemophilia! (Cue the Twilight Zone music…)

So Biogen is no stranger to me, but it is to our community because they did not continue the recombinant factor VIII program from 1982. It’s smart for any company to get familiar with the community before its product is launched. This company has a leg up as many of the people on its research, medical, clinical and marketing teams are from the community itself.

Still, Jess posed a question to our group of 35: How would you describe Biogen Idec? About 50% chose “Innovative,” while 19% chose “New and unknown player.” Yes, Biogen Idec is researching this new technology and new hemophilia products, but — who are you?

Biogen Idec is very familiar in the world of multiple sclerosis (MS). It has only three commercial products: Rituxan, Avonex (interferon beta), and Tysabri (natalizumab), which collectively had 2010 revenues of $4.7 billion. So it’s not a small company.
But they have a big pipeline, with nine programs in Phase III (late stage) clinical studies, including extended half-life factor VIII and IX. Data just presented at ISTH in Japan this past summer looks promising.

After listening in the morning to presentations by Biogen Idec executives about the company, its history, products and mission, we listened to quite compelling testimonials from two MS patients, and a local executive director of an MS society. Some of us were stunned to be on the “other side” of the testimonial telling: usually we are the ones giving the heart-wrenching testimonials. It was stirring and emotional to listen to an articulate mother tell her story of MS, and how the Biogen Idec drug helps her to live a normal life. She also related the patient assistance programs that help each person affected, and how she has counted on this help for 15 years! She most definitely put a face to the company in terms of practical and compassionate care.

After lunch we toured the manufacturing plant. This is always fascinating to me. None of us are scientists, and yet we have some idea of what goes on in making factor. But we get reminded when we tour plants of the years and years of research that goes into preparing for manufacturing, the quality control that must be in place, and above all the cleanliness of the environment. I think this struck us all the most. As Joe Kleiber of NHF commented, the “cleanliness and complexities” of large scale production of factor is astounding.


Steve, our tour guide, stressed how in making such a product, the process needs clean water and clean air. Quality control is paramount: they measure the concentration of microbes in the air; they reduce human contact with the manufacturing process as much as possible (humans carry bacteria, and we were to purify our hands many times on the tour); all reactors have to be sterilized each time a new batch is made. The process needs pure water, to make steam, to clean the pipes and parts. He showed us software that displayed continually how the current manufacturing process was measuring up, using 50 different measures all combined into one measure, compared to historical data. Looking at a line chart on a screen, you could actually see the current production and how it compared over past weeks to a historical line. In this way, you can make adjustments needed to get production back on track. This was fascinating to us!

When we returned, we had many questions and the Biogen Idec team had questions for us. One piece of advice that was sincere and deep was not to overpromise the hemophilia community. New products are fantastic; we hunger for advanced products to help our children live more normal lives. But we have been burned in the past, when hopes were raised that a cure was around the corner.

The message was clear, for all companies: now that patents have expired and the race is on to bring new products to market, don’t build up hopes too high. Fortunately, we have great products now and our loved ones live good lives, especially compared to most other patients globally. We can wait and we want a realistic perspective. Still, there was excitement that a break-through product perhaps is not too far away.

In closing, Jess deftly asked us how we would now describe Biogen Idec? Not too surprisingly, almost everyone changed their original answer and selected, “Committed to the hemophilia community.”

I’d say that was a very successful first hemophilia Advocacy Advisory Summit.

For more info: www.biogenidechemophilia.com

Thanks to Biogen Idec for the tour and time, and thanks to all my colleagues who made for such a great meeting and day.

Good Book I Just Read
The Life You Can Save
Peter Singer

This compassionate and thought-provoking book on humanitarianism is nonetheless by a philosophy professor who once wrote that there is an argument to be made that aborting a fetus known to have hemophilia is not ethically wrong, in his book (which I have not read) ‪Writings on an Ethical Life‬. I am not going there now, but will say this book was good. Singer’s very basic premise is that most of us would not hesitate to help a child drowning in a pool, even if it were inconvenient (missing a bus, ruining our clothes), but we blindly turn from the 27,000 children who die every day from preventable causes in developing countries. He sets his case well, wondering why we do not easily give up buying things we don’t need in order to help others to live. He explores our wariness with charitable organizations, and challenges our rationalizations for not giving. This is a good book, not without its flaws in the argument, but worth reading and a very quick read. Recommended for those who are compassionate, who want to influence others to be more compassionate and giving, and for those in healthcare globally. Three stars.

Big Week for New Hemophilia Products

There was lots of news this week under the heading of “What can I look forward to when I don’t have gene therapy?”

How about extended half-life factor? So, instead of prophy three times a week, you dose once, and it lasts all week? Extended half-life factor is the next great thing in hemophilia, while we wait someday for a cure. I think this will be a reality in the next two years.

This week Biogen Idec (a company right here in Massachusetts; yes, they are freezing just like the rest of us this week) announced that they’ve dosed their first patient with extended half-life recombinant factor IX.There are about 6 companies studying long-acting factor IX. One of these is also GTC Biotherapeutics, also located in Massachusetts, which has a FIX product in preclinical studies. And this is really different: GTC is using transgenic animals, like rabbits and goats. These animals are specially bred to excrete human FVIII or IX through their milk glands. Fascinating!

Now even when a company has a new product in clinical trials, this doesn’t mean that it automatically will come to fruition. These are trials, and sometimes things don’t work out. For example, just this week Bayer cancelled its LipLong study, which seemed so promising. Bayer was exploring an extended half-life factor VIII (BAY 79-4980), but results weren’t as expected. But don’t worry; they are also working on another extended half-life FVIII in different preclinical trial.

One thing to note: you can actually voluteer to participate in one of these studies. Go to clinicaltrial.gov. You could help make history!

Great Book I Just Read
Take Yourself to the Top by Laura Fortgang

I read this book every two years at New Year’s to get motivated, to reasses and to plan. Not only for my business, but also for myself. This book has been around a while, but I love its no-nonsense approach, its friendly and cajoling tone, its practical applications for thinking bigger, laying the groundwork for success and removing all obstacles. I recommend it for anyone starting a business or starting a new passion within a business. First step, get off the caffeine. Man, it’s like she reads my mind in this book at times! Four stars.

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