New First-in-Class ITP Therapy May Transform Clinical Practice: David Kuter, MD, DPhil

The FDA recently approved rilzabrutinib (Wayrilz; Sanofi) as a first-in-class treatment for immune thrombocytopenia (ITP), a rare autoimmune disorder. The approval was supported by results from the randomized multicenter phase 3 LUNA 3 study (NCT04562766), which evaluated the efficacy and safety of rilzabrutinib vs placebo in adults and adolescents with persistent or chronic ITP.

In an interview with The American Journal of Managed Care^®, David Kuter, MD, DPhil, lead investigator of the LUNA 3 study, discusses how this approval may influence ITP clinical decision-making and highlights key challenges in integrating rilzabrutinib into clinical practice.

Kuter is director of clinical hematology at Massachusetts General Hospital and professor of medicine at Harvard Medical School.

This transcript has been lightly edited; captions were auto-generated.

Transcript

Overall, what does this approval mean for patients with ITP, and how do you see it shaping clinical decision-making going forward?

I think this offers us a new avenue to treat ITP, as does any new medication in this space. Like all new medications in ITP, and there are many examples I can give to you, but I won't belabor the point, we study patients who have bad disease, and we show it works. Then, as time goes by, we tend to use it in patients with earlier disease, where they don't have 10 years of prior disease or have failed prior therapies.

To a small extent, we've already analyzed that in our current studies with this drug, knowing that patients who have earlier disease and less robust disease have a higher response rate than the 23% I mentioned for this on average refractory group.

As we progress, we tend to use these new medications in our sickest of sick patients, but recognizing full well that patients with lesser disease are probably going to have an even better response rate with this medication. Hence, like all the other therapies in ITP, this will gradually go from the more affected patients to those with earlier disease.

At some stage, when more studies are completed, this may be a drug that one might use in the initial treatment of ITP, or at least in the very early stages of treating ITP, which right now is dominated primarily by corticosteroids and [intravenous immunoglobin].

What challenges do you anticipate in integrating rilzabrutinib into routine clinical practice for ITP?

I think, right now, the integration is always an interesting step. It involves both physician education, data from investigators, such as myself, in this space, and also the usual problem we have, and I say this again, tongue in cheek, with insurance companies providing adequate resources to have patients have access to the drug.

I think, right now, this is a good drug with a good treatment profile to it that will be added to our armamentarium. As we do more studies in ITP, we'll have longer-term follow-up. We didn't talk about long-term follow-up with rilzabrutinib because there's a lot of long-term data we didn't discuss, showing that it has a prolonged effect.

I think, right now, that this medication will be well-received by caregivers for 3 reasons. One is that it works increasingly well early in the disease. Number 2, it has very few, if any, [adverse] effects. Number 3, and we didn't talk much about this as well, is the fatigue component of ITP. In some patients, it's a major problem they face. Not so much bleeding or bruising, they just don't feel well. What's striking to us is that this drug made people have an improved quality of life, so I think that may be another point that physicians, once they get more familiar with the drug, will take into consideration.

Probably the last thing that will evolve with time is the publication of our data in this and other studies of long-term follow-up, showing how long the response rates in those who respond to it are maintained and the fact that new adverse events that might appear after 1 or 2 years of drug treatment actually have not appeared in studies that we've done so far with longer-term administration of this drug.