FDA Grants ODD to Cevidoplenib for the Treatment of Immune Thrombocytopenia


The orphan drug designation (ODD) comes after positive results from a phase 2 trial that showed cevidoplenib improved platelet counts by 63.6% and 40.9% in 2 different dose groups.

Patient with thrombocytopenia -- Image credit: Bungon | stock.adobe.com

Image credit: Bungon | stock.adobe.com

About the Trial

Trial Name: Study of Oral SKI-O-703, SYK Inhibitor, in Patients With Persistent and Chronic Immune Thrombocytopenia (ITP)

ClinicalTrials.gov ID: NCT04056195

Sponsor: Oscotec Inc.

Completion Date: January 10, 2023

The FDA has granted cevidoplenib (SKI-O-703; Oscotec Inc.) an orphan drug designation (ODD) for the treatment of patients with immune thrombocytopenia (ITP). The designation comes after results of a phase 2 clinical study that demonstrated efficacy of cevidoplenib in patients with chronic ITP.1

ITP is a disorder which can lead to easy or excessive bruising and bleeding resulting from unusually low levels of platelets. The condition, which was previously known as idiopathic thrombocytopenic purpura, can cause purple bruises and small, red-purple dots that appear rash-like. Current treatment methods for ITP consist of corticosteroid administration, thrombopoietin receptor agonists; however, sustained remission without treatment is an indicator that ITP management is necessary. Cevidoplenib is a selective SYK inhibitor that treats patients with ITP, and currently treats rheumatoid arthritis and other autoimmune conditions.1,2

"Potential partners are drawing attention, not only to the efficacy of cevidoplenib, but also its exceptional safety profile and the convenience of oral dosing," said Taeyoung Yoon, PhD, CEO, CSO of Oscotec, in a press release. "Obtaining [an] ODD is an important milestone in the development of cevidoplenib and ultimately will benefit patients. We will explore every available option including partnership to deliver our drug as quickly as we can to those who suffers from the potentially crippling disease."1

The multicenter, randomized, double-blind, placebo-controlled, parallel-dose phase 2 trial (NCT04056195) evaluated cevidoplenib in patients aged 18 years and older with persistent and chronic ITP who have either failed to respond to or relapsed after a prior therapy. Patients were randomly assigned to receive 200 mg of cevidoplenib per day (100 mg twice-daily, 12 hours apart) plus 2 capsules of placebo (microcrystalline cellulose), 400 mg of cevidoplenib per day (100 mg 4 times per day) without placebo, or 4 capsules of placebo over a 12-week duration.2,3

The study’s duration was 20 weeks—consisting of 4-week screening period, 12-week treatment period, and 4-week follow-up period—for each participant. The primary end point was platelet response (baseline count: < 30,000/µL) and the secondary endpoints included the number of adverse events (AEs), serious AEs (SAEs) and whether or not they contributed to treatment discontinuation; patient quality of life; bleeding score; and both vital sign and electrocardiogram abnormalities.2,3

According to the results, the response rate for patients who received treatment with cevidoplenib at the 400-mg dose was approximately 63.6%, compared with those who received placebo, which was approximately 33.3% (P = .151). In addition, half of the participants on the higher dose had achieved 2 or more consecutive platelet counts that were 30,000/µL or higher, compared with 8.3% of patients who received placebo (P = .015), and approximately 40.9% of patients achieved platelet counts of 50,000/µL or higher compared with the 8.3% on placebo (P = .055).2

"We are elated to see the data that confirms the potential of cevidoplenib becoming a safe and effective medicine for [patients with ITP] who are not responding to the current standard therapies. Not only that, the data indicate that our SYK inhibitor will be at least as efficacious as the competitors in late-stage development,” said Taeyoung in a press release. “With the proven, exceptional safety profile and the convenience of oral dosing, we believe that cevidoplenib could 1 day become a genuinely competitive drug of choice for patients with ITP.”


1. PR Newswire. FDA Grants Orphan Drug Designation to Cevidoplenib for ITP. News release. March 21, 2024. Accessed March 21, 2024. https://prnmedia.prnewswire.com/news-releases/fda-grants-orphan-drug-designation-to-cevidoplenib-for-itp-302095752.html
2. BioSpace. Oscotec Announces Topline Results for Phase 2 Trial of Cevidoplenib. News release. February 24, 2023. March 21, 2024. https://www.biospace.com/article/releases/oscotec-announces-topline-results-for-phase-2-trial-of-cevidoplenib/
3. Study of Oral SKI-O-703, SYK Inhibitor, in Patients With Persistent and Chronic Immune Thrombocytopenia (ITP). ClinicalTrials.gov identifier: NCT04056195. Last updated July 21, 2023. Accessed March 21, 2024. https://clinicaltrials.gov/study/NCT04056195
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