Gene therapy for Hem B is exciting, but for us it’s ‘hurry up and wait’

Lately, I’ve been reading a great deal of news about Hemgenix (etranacogene dezaparvovec), a one-time gene therapy approved by the U.S. Food and Drug Administration last November for adults with hemophilia B. Hemgenix, which also was approved by the European Commission in February, was found in clinical studies to significantly reduce the annualized bleeding rates of patients with moderate to severe hemophilia B.

My husband, Jared, has severe hemophilia B. Over the years, he’s closely followed the news about gene therapy in hopes that one day it might be made available to people with hemophilia in our country, the Philippines.

An exciting prospect

I can’t help but feel excited about what the future may hold for my husband, knowing that gene therapy is now possible for his hemophilia type.

Since he was a child, Jared has relied on several therapies, including fresh frozen plasma and cryosupernatant infusions. Today, he receives on-demand factor IX infusions at the onset of each bleeding episode.

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Should Jared succeed at obtaining gene therapy, he’d no longer require regular infusions. I can only imagine how liberating it’d be for him to not have to carefully plan his activities based on their likelihood of inducing a bleed or to constantly have to adjust his schedule.

The prospect of him living a more normal life for the rest of his productive years is exciting! But as of now, it isn’t realistic.

Harsh and expensive reality

Realistically speaking, it’s unlikely that the gene therapy will reach the shores of the Philippines anytime soon. Jared tells me that despite factor products being made available in Western countries in the 1970s, it took decades for them to be offered here. And although regularly scheduled prophylactic doses of clotting factor are considered the gold standard for hemophilia treatment in many countries, the Philippines has yet to catch up.

Currently, Hemgenix is available only in the U.S. and the European Union — at a cost of approximately $3.5 million per treatment, making it the world’s most expensive drug. However, according to the journal Nature, CSL Behring, which bought the global rights to market Hemgenix from uniQure in 2021, said in a statement that the steep price tag is justified because it could save the U.S. healthcare system as much as $5.8 million per person treated.

But in the Philippines, where an average family’s annual income is 307,190 Philippine pesos (roughly $5,660), the treatment would remain out of reach for most.

‘A good pair of jeans’

In a moment of humor, Jared compared gene therapy to having a “good pair of jeans” (pun intended). An avid denim enthusiast, Jared observed that the best-fitting jeans often come with a hefty price tag. (He favors a certain brand known to last for generations — sometimes outlasting the original wearer!)

While it’s ideal to buy a good pair of jeans that’ll last a long time, in some cases, it just isn’t feasible — for instance, if it would compromise one’s ability to survive or if it simply isn’t available. In that case, it’s perfectly justifiable to buy a cheap pair of jeans just to get by, even if the long-term cost is higher.

While there’s nothing wrong with hoping, we must also be realistic about our expectations. So we patiently wait. And we cope with ongoing hemophilia maintenance, which includes staying physically active and keeping up with Jared’s on-demand infusions. It’s the best we can do for now, and it works.

Until better opportunities arise, we’ll simply do our best to get by.

Note: Hemophilia News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Hemophilia News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to hemophilia.