The FDA’s ODD status is granted to investigational assets for treating diseases affecting less than 200,000 people in the US.
This designation provides incentives such as tax credits for clinical trials, prescription drug user-fee exemptions, and the potential for seven-year marketing exclusivity following regulatory approval.
Electra Therapeutics chief medical officer Kim‑Hien Dao said: “The orphan drug designation is an important milestone in ELA026’s clinical development in sHLH.
“This designation underscores the high unmet medical need in sHLH and supports our continued efforts to evaluate the effectiveness of ELA026 in depleting the pathogenic immune cells responsible for the cytokine storm and addressing the high early mortality observed in this disease.”
Recently, the company announced findings from its Phase Ib trial of ELA026.
The open-label, single-arm, multi-centre Phase Ib study is designed to evaluate the safety and efficacy of ELA026 in treating sHLH.
It also aims to assess biomarkers and detect an optimal dose regimen for further evaluation in a Phase II/III study.
According to the results from the ongoing trial, ELA026 treatment led to a 100% overall response rate (ORR) by week four and demonstrated improved two-month survival in treatment-naive mHLH patients compared to historical data.
Additionally, the asset showed a strong response rate across various sHLH subtypes and exhibited a favourable safety profile in this patient group.
Furthermore, manageable adverse events were reported in the trial subjects.
In the study, pharmacodynamic and biomarker responses correlated with clinical responses.
The Phase Ib trial has now been expanded to include up to 20 subjects in Cohort 3 and will continue evaluating ELA026 as a frontline therapy for patients with various sHLH subtypes.