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

去纤苷 is not distributed through mainland China's retail pharmacy network. Uwell Cloud can help arrange a purchase channel for this medicine, sourced and dispatched from Hong Kong; clinical administration support in China can be coordinated on request. Availability and eligibility for any specific patient are determined by clinical review.

Regulatory Approvals

• FDA — Approved (2016) — Sources
• EMA — Approved (2013) — Sources

Pivotal Clinical Evidence

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

Sources

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