Thrombopoietin Receptor Agonist Market - Growth Drivers and Challenges
Growth Drivers
- Cost reduction and adoption in TPO-RA Biosimilars: Global cost-containment policies and maturing biosimilar frameworks at FDA/EMA are strong demand drivers. The NLM article published in March 2024 states that the biosimilars prices have reduced from 15–35% lower list prices than reference biologics, and are projected to save the amount of $38 billion to $124 billion from 2021 to 2025. These dynamics signal substantial future affordability gains and wider adoption of TPO-RA biosimilars as of 2025.
- Product innovations and strategies by key players: The FDA approved a prefilled, single-use autoinjector of romiplostim in 2023 to enable patient self-administration, focusing on improving treatment adherence and reduce healthcare resource use. Further, the WHO report states that cross-regional partnerships in Asia and Africa for global access to eltrombopag in 2024 to increase availability in low- and middle-income nations. These advancements combined with post-marketing surveillance and lifecycle management initiatives, the TPO-RA market is expected to drive sustained annual growth.
- Rising disease incidence driving TPO-RA: According to the May 2024 NLM report, the incidence of pediatric ITP is estimated at 1 to 6.4 per 100,000 annually, although rates could be higher since the figures cited are only symptomatic hospitalized cases, with the highest being between the ages of 2 to 5 and during adolescence. This rise in disease burden has been followed by a rise in prescriptions of TPO-RA from 2022 to 2025, as indicated in the FDA's Drug Utilization Database (2024). These trends indicate the pivotal role of TPO-RAs in treating thrombocytopenia, with the The thrombopoietin receptor agonist market in the APAC is the fastest-growing region and is poised to hold the market expected to further increase due to better diagnostic rates and increasing treatment guidelines.
Novel Therapies for Treatment of Immune Thrombocytopenia
|
Target |
Drug class |
Mechanism |
Agents |
Development status |
|
Macrophage |
Syk inhibitor |
Decrease in ADCP (inhibition of macrophage phagocytosis) |
Fostamatinib |
Approved (US) |
|
Macrophage |
BTK inhibitor |
Decrease in ADCP (inhibition of macrophage phagocytosis) |
Rilzabrutinib |
Phase 3 (NCT04562766) |
|
Plasma cells |
Proteasome inhibitor |
Inhibits plasma cell production of anti-platelet antibody |
Bortezomib
|
Phase 1 (NCT03013114) |
|
Plasma cells |
Anti-CD38 antibody |
Inhibits plasma cell production of anti-platelet antibody |
Daratumumab |
Phase 2 (NCT04703621) |
|
Antiplatelet antibodies |
FcRn blocker |
Increase clearance of anti-platelet antibody |
Efgartigimod |
Phase 2 (NCT03102593 |
|
Platelet |
Neuraminidase inhibitor |
Decrease in platelet desialylation thus reducing their destruction in the liver |
Oseltamivir |
Phase 2 (NCT01965626) |
|
Classical complement pathway |
C1s inhibitor |
Decrease in CDC (antibody inhibits C1s activity) |
Sutimlimab |
Phase 2 (NCT04669600) |
Source: NLM, April 2022
TPO-RA Regulatory Approvals Indications in the U.S. and EU
|
Drug (Generic) |
FDA-Approved Indications (2022–2025) |
EMA-Approved Indications (2022–2025) |
Key Therapeutic Areas |
|
Avatrombopag |
ITP – Adults with chronic ITP after insufficient response to previous treatment. CLD – Adults with CLD scheduled for a procedure. |
ITP – Primary chronic ITP in adults refractory to other treatments. CLD – Severe thrombocytopenia in adults with CLD scheduled for invasive procedure. |
ITP, CLD |
|
Eltrombopag |
ITP – Adults & pediatric (≥1 yr) with chronic ITP after insufficient response to corticosteroids, immunoglobulins, or splenectomy. SAA – Adults & pediatric (≥2 yrs) in combination with IST, or patients unresponsive to IST. HCV – Thrombocytopenia in chronic hepatitis C to enable interferon-based therapy. |
ITP – Patients (≥1 yr) with primary ITP ≥6 months refractory to other treatments. SAA – Adults with acquired SAA refractory to IST or unsuitable for transplant. HCV – Adults with chronic HCV thrombocytopenia preventing optimal interferon-based therapy. |
ITP, SAA, HCV |
|
Romiplostim |
ITP – Adults & pediatric (≥1 yr) with ITP after insufficient response to corticosteroids, immunoglobulins, or splenectomy. |
ITP – Adults & pediatric (≥1 yr) refractory to other treatments. |
ITP |
|
Lusutrombopag |
CLD – Adults with CLD scheduled for a procedure. |
CLD – Severe thrombocytopenia in adults with CLD undergoing invasive procedure. |
CLD |
Source: Science Direct, May 2022
Challenges
- Drug cost in developing and developed markets: In Canada, the treatment cost reduces patient access to TPO-RA drugs. A complete treatment cycle may exceed $27,118, according to the NLM report in June 2024, and these are out-of-pocket costs for most patients without full insurance. The absence of strong public reimbursement policies, along with the fragmentation of insurance coverage, discourages market penetration. Pricing strategies based on affordability or local production incentives could unlock future growth prospects, particularly for biosimilar or generic TPO-RAs.
Thrombopoietin Receptor Agonist Market Size and Forecast:
|
Base Year |
2025 |
|
Forecast Year |
2026-2035 |
|
CAGR |
7% |
|
Base Year Market Size (2025) |
USD 2.7 billion |
|
Forecast Year Market Size (2035) |
USD 5.1 billion |
|
Regional Scope |
|
