AKCEA THERAPS — Tegsedi FDA Review (NDA/BLA 211172, 2026)
AKCEA THERAPS · 2026 · FDA Review (Drugs@FDA) · company profile →
Report summary
Tegsedi (inotersen) was FDA-approved for hereditary transthyretin-mediated amyloidosis polyneuropathy, but its use is associated with significant safety concerns. These include severe thrombocytopenia and glomerulonephritis, necessitating a REMS program and rigorous monitoring. Clinical trials (Study 1) showed Tegsedi improved mNIS+7 and Norfolk QoL-DN scores over 66 weeks. However, the drug also carries risks of inflammatory/immune effects, hypersensitivity, and liver injury. Notably, serious liver events, including transplant rejection in stable liver transplant patients and severe abnormalities in non-transplant patients, have been observed, leading to recommended updates in the prescribing information. Uninterpretable platelet counts were also reported.
Key findings — cited to the page
| Platelet count below 75 x 10^9/L | 14% | TEGSEDI (Study 1) · p.16 |
| Nadir platelet count below 75 x 10^9/L (baseline platelet count below 200 x 10^9/L) | 39% | TEGSEDI (Study 1 and extension study) · p.16 |
| Platelet count below 75 x 10^9/L (placebo) | 0% | TEGSEDI (Study 1) · p.16 |
| Severe thrombocytopenia (platelet count below 25 x 10^9/L) | 3% | TEGSEDI · p.17 |
| Glomerulonephritis | 3% | TEGSEDI (Study 1) · p.17 |
| Glomerulonephritis (placebo) | 0% | TEGSEDI (Study 1) · p.17 |
| Urine protein to creatinine ratio (UPCR) greater than 5 times upper limit of normal | 15% | TEGSEDI · p.18 |
| Nadir platelet count below 75 x 10^9/L (baseline platelet count 200 x 10^9/L or higher) | 6% | TEGSEDI (Study 1 and extension study) · p.16 |
| Urine protein to creatinine ratio (UPCR) greater than 5 times upper limit of normal (placebo) | 8% | TEGSEDI · p.18 |
| Increase from baseline in serum creatinine greater than 0.5 mg/dL | 11% | TEGSEDI · p.18 |
| Increase from baseline in serum creatinine greater than 0.5 mg/dL (placebo) | 2% | TEGSEDI · p.18 |
| Carotid artery dissection and stroke | 0.6% | TEGSEDI · p.18 |
Source: AKCEA THERAPS ↗ · License: US Government public domain. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Neurology library →