Generic Eltrombopag Expands Access and Flexibility in ITP and Severe Aplastic Anemia

Kanwarpal S. Kahlon, MD

The commercial availability of an AB-rated generic formulation of eltrombopag (Promacta) represents a meaningful development that could help expand access to treatment for patients with immune thrombocytopenia (ITP) and severe aplastic anemia, according to Kanwarpal S. Kahlon, MD.

In May 2025, generic, AB-rated formulations of eltrombopag as both an oral suspension and tablet are became commercially available for patients with severe aplastic anemia who are insufficiently responsive to immunosuppressive therapy; for patients 1 year of age and older with persistent or chronic ITP that is insufficiently responsive to other treatments and who have an increased risk of bleeding; and for the treatment of thrombocytopenia in patients with chronic hepatitis C whose clinical condition prevents or limits initiation/maintenance of interferon-based therapy.1,2

“[The generic formulation] will be very good for patients, especially with these chronic conditions, where we can feel confident that we can get them this treatment. It's a wonderful and burgeoning field. Hopefully, we'll see more and more of these biosimilars over time, and that will just increase the treatment options that we have for patients,” Kahlon explained.

In an interview with OncLive®, Kahlon discussed the potential economic and clinical implications of the availability of generic eltrombopag as both a tablet and oral suspension; his experience with the reference drug, Promacta; and treatment strategies when using eltrombopag for patients with ITP or severe aplastic anemia.

Kahlon serves as an associate clinical professor of medicine at the UCLA School of Medicine.

OncLive: How might the recent commercial availability of the generic formulation of eltrombopag for ITP and aplastic anemia impact treatment access, and which patient populations could benefit from this bioequivalent option?

Kahlon: Anybody for whom there’s an indication for eltrombopag [could receive the generic formulation]—chronic ITP would certainly be one of them, and aplastic anemia would be another. [Those are] two of the major labels for eltrombopag as it currently stands. However, I would say for any other indication [for the reference] medication, this biosimilar will be useful and could be used in those patients.

What are some of the potential economic advantages of this bioequivalent being available through a co-payer program? Could these types of programs help with treatment adherence?

With Promacta, for some patients, it was challenging to get them the medication. I would say more often it was patients with chronic ITP, maybe less so with aplastic anemia. The availability of a biosimilar would hopefully reduce costs to insurers, pharmacies, and patients. With co-pay assistance, we make this more widely available.

We've seen other biosimilars for other targeted therapies. Imatinib [Gleevec] was one of the early ones for chronic myeloid leukemia, and the fact that it [now has a] generic option has made access to it easier, so I expect that it would be the same [for eltrombopag].

Are there some patients who may benefit from this agent being available in both oral suspension and tablet formulations?

The suspension and/or the tablet formulations could be useful in a variety of patient settings. For inpatient settings in the hospital, patients who can't take anything by mouth, or those who may have a nasogastric tube or other feeding tube, it is useful to be able to give a suspension.

Perhaps in pediatric patients, the availability of a suspension will prove to be useful. For anybody else for whom there may be swallowing difficulties, it is challenging when drugs are in capsule form. It's difficult to put capsules or pills in a suspension and administer to patients who can't take [those formulations].

Having these different forms or modes of administration gives you flexibility, and they make it possible to treat almost any patient.

What should be known regarding the safety of generic eltrombopag?

When these generic or biosimilar agents are approved, they look at safety, and it's equivalent to the parent, trademarked medication. There are some patients who do have some adverse effects [AEs], and that varies patient to patient. Some patients don't complain about anything. Some might have some joint pain. I've not encountered liver function test abnormalities or hepatobiliary problems in the patients whom I've treated, either with ITP or aplastic anemia; however, I would not expect the tolerability, which is good for the biosimilar and the generic, to be any different than the parent drug. It's well tolerated, and I expect the biosimilar would be the same.

As eltrombopag continues to be used in clinical practice, what guidance would you offer to colleagues regarding the monitoring and management of potential AEs associated with this agent?

In ITP, we tend to start [doses] low and titrate the drug up. That's a good way to monitor for AEs, by seeing how people are doing at lower doses. If they're tolerating it well, you should feel comfortable increasing the dose; if there are AEs, you can coach patients through them and find other strategies to address [those toxicities].

With aplastic anemia, the dosing is higher, but you're also treating a condition that's slightly different. In my experience, eltrombopagis well tolerated, whether using it for ITP, aplastic anemia, or another indication. Close patient monitoring for AEs [is important], but it is well tolerated.

What is the significance of the availability of this generic formulation of eltrombopag?

It opens up some treatment options, which [were previously difficult] for patients [to access]—potentially due to insurance issues or copay challenges. I’ve heard [costs] across the spectrum. The fact that this is available [as a generic] will hopefully [mean it is] priced competitively and more widely accessible to patients. [It may also be] a treatment option that clinicians feel comfortable and confident prescribing, knowing they’ll be able to [obtain] it for their patients.

With other therapies that have [had] a generic or biosimilar available, we’ve been able to use and access them more easily. I expect the same will apply here.

References

1. Camber Pharmaceuticals Linkedin. Camber Pharmaceuticals is proud to announce the launch of Eltrombopag, the first AB-rated generic alternative. Posted May 14, 2025. Accessed May 14, 2025. https://www.linkedin.com/posts/camber-pharmaceuticals_camber-pharmaceuticals-is-proud-to-announce-activity-7328407491480809473-ug0C
2. Eltrombopag tablets and oral suspension. Camber Pharmaceuticals. Accessed May 14, 2025. https://www.cambereltrombopag.com