CSL Behring

New Survey Spotlights Ongoing Concerns for People Living with Hemophilia B

admin April 30, 2023

This is a paid public announcement from CSL Behring and does not constitute an endorsement of products or services. When you click on the links in this blog entry, you will be directed to the CSL Behring website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company; do not reveal identifying information against your will.

Carefully controlled schedules. Undercurrents of uncertainty. Persistent pain. People who are living with hemophilia B, and those who love them, face a lifetime of managing the complexities of this condition.

While significant advancements have been made in the treatment of hemophilia B, there is still a desire for new treatment options to address unmet needs for people with moderate to severe forms of the condition.

In fact, a recent CSL Behring-sponsored survey on the burdens of living with hemophilia B found that even with treatment, people with hemophilia B are still experiencing spontaneous bleeds and joint pain, and would consider switching to a new therapy that may be more effective. The online survey conducted by CSL Behring in partnership with the Coalition for Hemophilia B included 110 people with the rare bleeding disorder who are on either short-term or long-term prophylactic treatment.* The survey revealed that during a six-month period, respondents reported an average of 4.2 spontaneous bleeds and 2.2 joints bleeding three or more times.

Additionally, 87% of respondents reported experiencing joint pain at least a few times a month.

“The survey really highlighted the concerns and challenges that people with hemophilia B still face,” said Kim Phelan, Chief Operating Officer of The Coalition for Hemophilia B. “Spontaneous bleeds, joint damage and joint pain are just a few of the burdens that some in the hemophilia B community must live with.

With up to 156 intravenous infusions per year, people with hemophilia B are also at risk for vein collapse.

The life-long effects of living with and managing hemophilia B however aren’t just physical. More than 40% of people living with hemophilia B experience depression, anxiety, or other psychological disorders.

As new treatments become available to potentially address the unmet needs within the hemophilia B community, discussion with healthcare professionals on treatment goals is essential. It’s time to ask, “Is there a better treatment option for me?”

*Short-term prophylaxis is prolonged treatment following a bleed until full recovery and prophylaxis prior to physical activity. Long-term prophylaxis is regular preventative injections. Of the 110 people surveyed, 29 were being treated with short-term prophylaxis only, 74 were being treated with long-term prophylaxis only, and 7 were being treated with both short-term and long-term prophylaxis.

www.CSLBehring.com USA-HGX-0330-APR23

Stunning Breakthrough: Hemgenix

Laurie Kelley November 27, 2022

Last week we shared an essay by Paul Clement about the approval for gene therapy for hemophilia A, approved only in Europe, and asked, when for the US?

While that question is still valid for hemophilia A, the stunning news this week was that gene therapy–at long last—is approved, for hemophilia B!

The news straight from CSL Behring: “This historic approval provides a new treatment option that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy and generates elevated and sustained factor IX levels for years after a one-time infusion.”

The news was so startling, it made front page on CNN.com. But I suspect not for the scientific reason but for the economic reason: it comes with a $3.5 million price tag, making it the most expensive drug on earth currently.

Hemophilia Economics 101

While high prices are nothing new in hemophilia—factor therapy has always been among the world’s most expensive drugs—the sticker price was shocking to many. So many people have asked me through the years when is the price of factor going to come down, as if it were a high-tech consumer item like camcorders, Walkmans or DVD players. Remember those? They get mass produced, offshored, and millions upon millions of consumers buy them, which eventually drives the price down. And don’t forget competition. Basic supply and demand.

Hemophilia drugs are nothing like that. There are many factors that determine price but here are three: the research and development (R&D) that was spent to create the drug; the finite marketplace; and whether insurance will cover the cost.

R&D for drugs such as Roctavian, the brand name of BioMarin Pharmaceutical’s gene therapy product approved in Europe for hemophilia A, and Hemgenix, the brand name of CSL Behring’s gene therapy product approved by the U.S. FDA for hemophilia B, can surge to the hundreds of millions, if not billions, of dollars. The money needs to be recouped, and reinvested in the company, and to investors.

The smaller the target audience, the higher the price. How can you recoup the R&D with such a small consumer audience as hemophilia B? In the U.S., there are approximately 20,000 with hemophilia, of which about 15% have hemophilia B. Not everyone of these patients will want gene therapy; not everyone can afford it.

By afford it, I mean have insurance cover it, which is the final piece of the pricing puzzle. Who will pay the $3.5 million per patient? State Medicaid plans? Commercial insurance? What if a patient on Blue Cross Blue Shield is approved, gets the gene therapy, has it reimbursed, but the following year switches plans? How does this benefit the bottom line at BCBS? Will insurance companies say no to gene therapy based on these concerns?

Advocacy is Key

This is where our decades of strong advocacy in the hemophilia community will make a difference. In a way, we’ve been preparing for this moment our whole lives. While the new drugs are not being touted as a cure, those of us old enough remember the slogan “A Cure by 2000!” We have fought for compensation for those infected by HIV and hepatitis from unsafe blood products. We fought for the new recombinant drugs, when insurance denied us. We fought for longer half-life drugs, for prophy, for bi-specific antibody products. All of these came with higher price tags, and eventually we prevailed.

And now?

We will all need to be educated about this new gene therapy, and how to approach our insurance companies, if we want it. As we have been preaching since 2005, when everything changed in insurance for hemophilia, you need to learn to speak the insurance company’s language; debate with them in a way they are used to; work with your healthcare team; stand with your state hemophilia group.

There are so many excellent products available to treat hemophilia, will insurance companies use this to deny gene therapy? At this point, no one knows, but we do know we need to get prepared. Why?

BioMarin is actively working on getting Rotavian approved for hemophilia A in the US. And that will impact thousands more in our community. How the insurance reimbursement of Hemgenix plays out could be a harbinger of things to come.

Read CSL Behring’s press release here.

The Time is NOW!

Laurie Kelley February 27, 2012

I’m writing from Phoenix, where a weekend of education and networking for people with von Willebrand disease just wrapped up. The Arizona Hemophilia Association hosted the first annual national von Willebrand conference. Cindy Komar, the very capable executive director of the AHA and her team assembled a great cast of speakers and facilitators, and provided entertainment for the kids for the weekend-long event. It was a tremendous conference and hugely successful.

Saturday morning started with a rousing speech by Jerry Ervin, who shared his hard-luck story and how he overcame many environmental challenges to become educated and a motivational speaker. Then Drs. di Paola and Kouides shared latest developments in VWD treatment. Afterwards came break out sessions, in which I presented the changing insurance climate and how we can protect our access to treatment. Other sessions included dental care, financial planning, parenting and atypical VWD.

25 states were represented at the event, and I saw many friends in the community, from patients and families, and even my dear friend Julie Winton, with whom I climbed Kilimanjaro last August!

This morning we had two great speakers, Jeff Leiken, who spoke about attitude and removing bias from our way of looking at the world, and Pamela Crim, who shared her personal story, from a stroke at age 19 while just a newlywed, to adopting two little orphans from Juarez, Mexico–truly an incredible life story!

Outside the teens were treated to a demonstration by a hot air balloon pilot, Michael Glen. His inspiring talk about ballooning focused not on the fact that he’s a paraplegic (the only one in the world who is also a balloonist) but on how nothing can stop you from achieving your dreams. He was fantastic! I’d highly recommend him for teen workshops (www.rollingpilot.com)

We closed the event by paying our respects to the lady who was the pioneer in VWD advocating and educating, Renee Paper, my co-author and friend. We keenly felt her absence, but we’re pretty sure her spirit was with us.

Thanks to CSL Behring for the funding for making this fantastic event happen, and to the Arizona Hemophilia Association for their time and dedication.

Lucky Number 13 part 2

Laurie Kelley March 14, 2011

Just last week I blogged about CSL Behring’s new factor XIII concentrate Corifact. Now I’ve learned that Novo Nordisk announced that a Biologic License Application (BLA) has been submitted to the US Food and Drug Administration (FDA) requesting the approval of a recombinant factor XIII compound for those with congenital Factor XIII (FXIII) deficiency.

Corifact is made from human blood plasma. This new drug is made from recombinant technology, which uses genetic engineering.

Factor XIII deficiency has a prevalence of one case per two million people, with an estimated 600 diagnosed patients worldwide, making it one of the rarest bleeding disorders.

A press release from Novo Nordisk states: “Positive results from a phase III trial examining the efficacy and safety of recombinant factor XIII for the prevention of bleeds associated with congenital FXIII deficiency showed that when compared to a historic control group of individuals who did not receive routine FXIII infusions, preventive treatment with monthly recombinant FXIII injections significantly decreased the number of bleeding episodes requiring treatment. These data were presented at the American Society of Haematology (ASH) meeting in December 2010, and marked the first completed phase III study conducted to study the use of a recombinant FXIII treatment to prevent bleeding episodes in congenital FXIII deficiency patients.”

It’s all great news for those who have factor XIII deficiency, and with the sad news from around the world, especially Japan, we need good news.

Good Book I Just Read

No One Here Gets Out Alive
Jerry Hopkins and Danny Sugerman

I’m continuing my reading streak on The Doors, the 1960s band that took America by storm. In this book, Jerry Hopkins (Rolling Stone) and Danny Sugerman (then teen office boy for Doors) profile the madness and magnificence of Jim Morrison, front man and silky-voice singer, whose depravity and self-destruction eventually upstaged the music itself and his three talented band members. The book details Morrison’s schizophrenic adolescence in which he, with an IQ of 150, devoured sophisticated philosophy and poetry books, and wrote poetry himself, but yet immaturely and cruelly harassed his younger brother, scared the elderly and openly mocked cripples. The first few chapters alone might end any hero worship of The Lizard King. The book recounts chronologically how the Doors formed, their skyrocketing rise to fame, and the toll of fame on the unstable Morrison. They were only 21-23 years old, in a music industry and culture awash in drugs, but why did only Morrison succumb to the pressure? The book is insightful into the music industry of the 1960s. It’s incomplete in that no other character is truly explored: Manzarek, Densmore and Krieger are barely mentioned. They were together for six intense years: how did they cope with Morrison’s descent into madness? The book also sheds little insight into Morrison’s state of mind, or why he acts as he does other than the drugs or alcohol, but factually states a portrait of a seeming sociopath: the dozens of paternity suits, his wanting his child to be aborted because he simply didn’t want one, his cruelty to his mother when he performed, his promiscuity and debauchery.

It’s hard to reconcile the deep thinker with the raging drunk. Perhaps the book doesn’t go deep enough into Morrison’s psyche, but then, Morrison didn’t seem to let anyone in. He internalizes his pain, which seeps out in frightening rages, and then is dampened by alcohol. One only needs to look at his photo progression in just six years: from sleek, sexy rock star to bloated, bearded drunk. I don’t think the pain was from being a frustrated poet or even a rock star; that’s putting the cart before the horse. His pain was chronic, malignant. Poetry was one expression; rage and cruelty was another. Adoration from the masses was one treatment; alcohol, maybe heroin, was another, and became the final exit.

Morrison helped put the Doors on the map, and he destroyed the Doors by destroying himself. Forty years later, it seems we will never know what drove Morrison to the edge, and then over. The authors skim over any analysis by comparing his angst to being like the Greek god Dionysus, or part of the Beat generation, or expressing himself like the Indian shamans he revered. But this is shallow. Looking back today, Morrison was an emotionally disturbed artist who sought to medicate his pain through alcohol, and expressed himself through rage. Incredibly, he left a legacy of beautiful, mystical music that captured a unique time in America, and the black hole of his inner life. And we are drawn to such people, scared of them, and yet worship them. Despite the incomplete picture the book paints, it was a great read and I could not put it down. Three stars.

Lucky Number 13

Laurie Kelley March 4, 2011

When is the number 13 lucky?

We now have a factor concentrate for people with factor XIII deficiency. CSL Behring, makers of Helixate FS, Monoclate P and Mononine, was granted FDA approval for their factor XIII concentrate Corifact, indicated to treat congenital factor XIII deficiency. This is the first and only U.S. approved treatment for factor XIII-deficiency and the fourth new product that CSL Behring has brought to the U.S. market in the past two years. Corifact is already available for use in 12 countries throughout the world under the trade name Fibrogammin- P.

Factor XIII deficiency, also known as fibrin-stabilizing factor deficiency, is rare, affecting one in two million, with an incidence in the U.S. of approximately 150 people.

Symptoms include bleeding from the umbilical cord after birth, poor wound healing, miscarriages, subcutaneous bleeding, and excessive bleeding in joints and muscles following trauma. Patients lacking the FXIII protein are also at high-risk for intracranial hemorrhage (ICH), bleeding inside the skull that can be life threatening. Studies have shown that between 25- 60% of factor XIII deficient patients will experience an ICH at least once during their lifetime.

This is great news for those with factor XIII deficiency. For more information, please see www.corifact.com and contact your HTC staff.

Great Book I Just Read
Riders on the Storm by John Densmore

“There’s the Beatles, the Stones, and the Doors,” says Paul Rothschild, producer of one of the greatest bands of the sixties. Drummer John Densmore was only 21 when he joined with three other California students to form a band that would soon skyrocket to the top at a time when the US was a nation at war in Vietnam, and with itself. With quiet Robby Krieger on guitar, methodical and rational Ray Manzarek on keyboard and Adonis-look-a-like singer Jim Morrison, the band was like no other. With no bass player, they combined blues, jazz and psychedelic rock, with some Indian strings and often very dark lyrics, about death, murder and rage. And no wonder: Not long after forming, Densmore writes about their iconic lead singer, “I’m in a band with a psychotic.”

The book is captivating. It starts in Paris, 1975, with Densmore visiting Morrison’s grave, four years after his death at age 27 on July 3, 1971 from an overdose. Returning to the hotel, he pours his feelings out in a “letter” to Morrison on the hotel stationery, which then gets interspersed throughout the book with his recollections. His memoir details his time in the band, returning now and then to the ongoing letter, and towards the end, “updates” Morrison on events of the 1980s and 1990s. Interspersed are appropriate lyrics from their songs, so that the book itself becomes quite artsy. The book gives excellent insight into what the 60s were like, what it’s like being in a band on the rise and what it was like to survive the onslaught of Jim Morrison. What’s missing, though, is what “The Lizard King” was really like. We get snapshots, snippets, and stories, but it’s as if Densmore viewed Morrison at arm’s length, even though he intensely shared six years with him. He was in awe of Morrison—who wasn’t?—and scared of him, and rightly so. Though he feels guilty at not being able to help Morrison, we have to remember he was only a young man from a humble Catholic background, ill equipped to cope with sudden stardom, wealth and the phenomenally complex, creative and self-destructive Morrison.

As if to highlight the detachment Densmore had with Morrison, Densmore dedicates the book to John Lennon of the Beatles, one of Densmore’s heroes, and also mentions Lennon’s assassination in his “letter” to Morrison, yet doesn’t mention or maybe doesn’t even know that Lennon was assassinated on Morrison’s birthday, a fact he should know as Morrison and Densmore were born only a week apart.

“We sensed rage and a possible explosion too near the surface to mess with in dealing with you,” Densmore writes in his letter to the now dead Morrison. “It seemed to have a lid on it—Pandora’s box with all the demons that wanted to be released. We never opened the box… we had to deal with your demons seeping out the side.” They seeped out and poisoned everything; the Doors had to cancel a 20-city tour when Morrison was arrested in Miami for indecent exposure at a concert (he was just pardoned this past December!). The charisma, lyrics and stunning voice of Morrison helped make the Doors a success; his addiction, rage and irresponsibility destroyed them. The book was published in 1990 and Densmore writes, “Well, we’re going on 20 years and there’s no end in sight” of the fascination people have with the Doors. On July 3, it will be 40 years since Morrison’s death, and the Doors are almost as popular as ever. I picked this book up and finished it 5 hours later—it’s compelling, honest, shocking and eye opening. A great book about one of my favorite bands and my favorite singer—for no one could sing like Jim Morrison. Three stars.