Cevidoplenib Granted Orphan Drug Status for Immune Thrombocytopenia

The Food and Drug Administration (FDA) has granted Orphan Drug designation to cevidoplenib (SKI-O-703) for the treatment of immune thrombocytopenia (ITP) in patients who have failed to respond or relapsed after prior therapy.

Cevidoplenib is an investigational spleen tyrosine kinase (SYK) inhibitor, designed to inhibit the downstream signaling of B-cell and Fc receptors resulting in increased platelet counts. The designation is supported by data from a randomized, placebo-controlled phase 2 study (ClinicalTrials.gov Identifier: NCT04056195). 

The trial assessed the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of cevidoplenib in 61 patients with persistent and chronic ITP who failed to respond or relapsed after prior therapy, with a platelet count of less than 30,000/µL. Study participants were randomly assigned to receive cevidoplenib 400mg, cevidoplenib 200mg, or placebo twice daily for 12 weeks.

Results showed 63.6% (14/22) of patients who received cevidoplenib 400mg and 46.2% (12/26) of those who received cevidoplenib 200mg achieved platelet response (primary endpoint; defined as platelet counts ≥30,000/µL and doubling the baseline [average of 2 previous counts] without the use of rescue medication) compared with 33.3% (4/12) of patients who received placebo (P =.151 and P =.504, respectively). 

Additionally, 50% of patients in the higher dose group achieved 2 or more consecutive platelet counts of at least 30,000/µL compared with 8.3% of those who received placebo (P =.015). Achievement of platelet counts of at least 50,000/µL was observed in 40.9% of patients receiving high dose cevidoplenib vs 8.3% of the placebo group (P =.055).

The most common adverse events reported were increased alanine transaminase (8.3%), increased aspartate aminotransferase (6.3%) and nausea (4.2%).

Oscotec is currently seeking partners for further development of cevidoplenib. “Obtaining [Orphan Drug designation] is an important milestone in the development of cevidoplenib and ultimately will benefit patients,” said Dr Taeyoung Yoon, CEO/CSO of Oscotec.