Ravulizumab

So the mechanism of action is basically inhibit terminal complement, uh, inhibition and preventing, uh, MAC formation.

The approved agents include eculizumab, um, and ravulizumab, uh, which are monoclonal antibodies.

Uh, zalucoplant is a small macrocyclic peptide.

Uh, we have, uh, two other, uh, agents in late stage, uh, development and potentially, uh, can be available soon.

Uh, because of their specificity, uh, targeted therapies, uh, offer fairly rapid, uh, clinical improvement and, uh, generally a more favorable safety profile although, uh, there are certain concerns we need to be discussing, which I will do later.

For eculizumab, uh, it is a, uh, IV, uh, infusion, uh, given every two weeks, and the patient undergo a initial, uh, dosing, um, or loading interval, which consists of, uh, biweekly doses, and then they get, uh, every two weeks infusion.

For ravulizumab, it's also IV.

Um, the patient undergoing the initial dose, and then the first maintenance dose start at two weeks, and then thereafter they get, uh, maintenance dosing every eight weeks or so.

Eculizumab is administered intravenously using an indication-specific induction and maintenance schedule.

Ravulizumab is a longer-acting C5 inhibitor that permits less frequent maintenance dosing.

The precise regimen depends on the selected agent, patient weight, indication, and prescribing information.

The treatment goal is resolution of active TMA, recovery or stabilization of kidney function, control of hemolysis, and prevention of further organ injury.

Again, we had three medications.

Uh, eculizumab was the first one out, then ravulizumab, and then, uh, zalukapam.

Um, so for, uh, ECU and RAVU, uh, they are IgG.

Zalukapam is actually a small peptide, um, molecule.

Um, the first two, ECU and RAVU, are given in, uh, IV formulation, and zalukapam is given sub-Q.

Um, eculizumab is, uh, every two weeks.

Um, ravulizumab is, uh, every eight weeks.

And then zalukapam is daily, um, dosing.

I'd had my meningitis shots.

Ultomiris was becoming the standard treatment, but it hadn't quite been put in place, so I started on Soliris.

It's kind of amazing.

After my first loading dose of Soliris, all these wonky labs zipped into the normal zone.

If you're gonna have a absurdly rare disease, it's nice to have one that has treatment.

And we have a, an abundance of treatments that, as I understand it, not everyone responds to different treatments, and I didn't.

I was only a partial responder to the Soliris, Ultomiris treatment.

My hematologist has been wonderful.