Recently I wrote an article about a clinical study which is an Ravulizumab pregnancy registry in the USA.
Then tried to imagine what a topic like that would look like if discussed on a day TV time panel show a important clinical and ethical topics like this rarely get attention on such programs. I dreamed on…
TV Moderator: Welcome back to our virtual hot seat, folks! Today, we’re diving into a topic that’s equal parts science, hope, and real talk: the Alexion Ravulizumab Pregnancy Registry. Joining us is Len Woodward, a passionate advocate from the aHUS Alliance, to break it all down. So, grab your drink of choice, and let’s get into it!
First Panelist : Alright, everyone -and Len, welcome to the table! We’re talking about the Alexion Ravulizumab Pregnancy Registry today. For those who don’t know, ravulizumab—aka Ultomiris—is a drug for rare blood disorders like Paroxymal Nocturnal Hemoglobinuria and atypical hemolyic uremic syndrome -aka- aHUS. Now, Alexion’s set up this registry to track what happens when pregnant women take it, because let’s face it, we need answers about how it affects moms and babies. Len, you’ve been in the trenches with this stuff—what’s your take?
Len Woodward: Thanks it’s great to be here with you all. As someone who’s worked with the aHUS community for years, I can tell you this pregnancy registry is a big deal. It covers all the conditions where Ravulizumab is indicated as a treatment. Ravulizumab is a lifeline for people with atypical hemolytic uremic syndrome—aHUS—which can wreak havoc on your kidneys and blood. Pregnancy can trigger it, and for women who need this drug, stopping isn’t always an option. The registry’s about gathering real-world data—how does it work for moms, how do the babies fare? It’s not a lab experiment; it’s life happening.
Second Panellist: Okay, Len, I’m intrigued, but I’ve got my sceptical hat on. Ravulizumab’s a C5 inhibitor—sounds like sci-fi to me—meant to calm down an immune system gone rogue. It’s given every eight weeks, which beats the every-two-weeks deal with eculizumab, right? But here’s my thing: the label says “no data” on pregnancy. So, we’re flying blind, and Alexion’s both making the drug and running the registry. Doesn’t that feel a little… cosy?
Len Woodward: Fair question. Yeah, ravulizumab’s an upgrade from eculizumab—longer-lasting, fewer infusions, less hassle. It was through case studies of pregnant women that eculizumab was found to be OK in pregnancy and breast feeding. It is the upgrade changes that are the focus of concern. And you’re right, there’s no formal pregnancy data from trials because pregnant women were excluded. aHUS happens more often post partum. No data was the case for eculizumab too. That’s why this registry matters—it’s filling that gap with real experiences. As for Alexion running it, I get the “cosy vibe” concern. But they’re under pressure from regulators like the FDA to prove long-term safety. Plus, the data’s not just for them—it’s for doctors, patients, and families making tough calls. I’d love more independent eyes on it, though usually there would an independent scientific panel reviewing the registry data including patient advocates on the panel.
Third Panellist: Len I’m all about evidence and protecting people. I read that aHUS hits about 1 in 25,000 pregnancies, and ravulizumab’s already helped some postpartum cases—like a 2021 study where it worked fast. This registry’s tracking outcomes: healthy babies, complications, all that. It is about the babies. What’s the hope here? Are we talking game-changer for women with rare diseases?
Len Woodward: Exactly. The hope is huge. Take a woman with aHUS—pregnancy can flip that switch, and suddenly she’s facing kidney failure or worse. Ravulizumab’s shown it can stop that spiral, like in those postpartum cases you mentioned. The registry’s building a bigger picture of those moms with aHUS already treated going through pregnancy. As well as those few who onset during pregnancy and not post partum. But is mainly about what happens to the baby’s development.
First Panellist: I love that, Len—warrior women getting answers. But let’s talk risks. The drug’s got a warning about meningitis—a nasty bacterial infection—because it messes with your immune system. Pregnant women are already at higher risk. How’s the registry handling that?
Len Woodward: Great point. That meningitis risk is real with C5 inhibitors—ravulizumab, eculizumab, same deal. You need vaccines beforehand, and the registry’s absolutely tracking infections. So far as I know, no meningococcal cases have popped up in the pregnancy data, but it’s small numbers still. They’re watching it closely because, yeah, pregnancy’s already a vulnerable time. It’s about balancing that risk against the chaos of untreated aHUS—clots, organ damage, you name it or switching to eculizumab.
Second Panellist Alright, Len, I’ll bite—I’m warming up to this. But what about the long game? Babies born healthy, great, but what about when they’re 5, 10? And don’t get me started on cost—this isn’t cheap. Who’s footing the bill for these moms?
Len Woodward: You’re hitting the big ones. Long-term? We don’t know yet— registries typically follow up for a year or two, but 10 years out is a gap we need to fill. That’s where advocacy comes in—pushing for extended studies. Cost-wise, it’s a beast. Ravulizumab’s pricey, and it’s on insurance or healthcare systems to cover it. In places like the UK, the NHS negotiates, but in the US? It’s a patchwork. Equity’s the fight—every mom who needs it should get it, not just the lucky ones.
Third Panellist: Len, you’re painting a picture of hope with some grit, and I’m here for it. I saw a stat—87.5% of postpartum aHUS patients in a trial hit a “complete response” with ravulizumab in about a month. That’s fast. How does this registry turn that into something bigger?
Len Woodward: Yeh, that 87.5% from the “311 trial”—it’s gold. It’s why we’re excited. The registry takes that and scales it—more women, more pregnancies, breastfeeding, real-world messiness included. It’s voluntary, so doctors and patients opt in, but every story adds up. It is not just aHUS moms , it is PNH, gGM and NMSOD moms too. Alexion’s got the data, sure, but it’s also for the next mom who’s scared and searching for answers. It’s building a roadmap.
First Panellist: Alright, folks, time to wrap this party. Len, you’ve brought the heat today. The Alexion Ravulizumab Pregnancy Registry—it’s a lifeline, a question mark, and a call to action all in one. For women out there battling rare diseases, it’s saying, “We see you, and we’re learning.” Hot topic or not, ladies?
Second Panelist: Hot indeed . Science with a side of sass—my kind of day.
Third Panelist: Super hot. It’s women’s health, justice, and hope rolled into one
Len Woodward: Thanks, all—I’ll call it scorching. It’s about real lives, and that’s worth shouting about.
First Panelist: There you go, folks—Len Woodward and the crew, signing off. Keep the convo going—tell us what you think below!
Article No. 721
