去纤苷 defibrotide

Drug Class

Oligonucleotide mixture / Antithrombotic agent

Mechanism

Protects endothelial cells, restores thrombohemorrhagic balance by increasing tissue plasminogen activator (t-PA) and reducing plasminogen activator inhibitor-1 (PAI-1), and exhibits anti-inflammatory effects.

Approved Indication

Treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT).

What This Means For Patients

Defitelio (defibrotide sodium) is an intravenous oligonucleotide therapy approved by the FDA and EMA for severe hepatic veno-occlusive disease (VOD) following stem cell transplantation. It helps protect the lining of blood vessels in the liver and restore blood flow. Eligible patients can access this treatment under the special-import pilot policy in the Boao Lecheng pilot zone.

Regulatory Approvals

• FDA — approved (2016) — label
• EMA — approved (2013) — source

Pivotal Clinical Evidence

• NCT00358501 — Richardson et al., Blood 2016 (NCT00358501) — DOI

Sources

• https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/208112s000lbl.pdf
• https://www.ema.europa.eu/en/medicines/human/EPAR/defitelio