| Assessment Status |
Pre-submission consultation scheduled |
| HTA ID |
25058 |
| Drug |
Metreleptin |
| Brand |
Myalepta® |
| Indication |
Metreleptin (Myalepta®) is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: • with confirmed congenital generalised LD (Berardinelli-Seip syndrome) or acquired generalised LD (Lawrence syndrome) in adults and children two years of age and above • with confirmed familial partial LD or acquired partial LD (Barraquer-Simons syndrome), in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control. |
| Rapid review commissioned |
17/09/2025 |
| Rapid review completed |
13/10/2025 |
| Rapid review outcome |
A full HTA is recommended to assess the clinical effectiveness and cost effectiveness of metreleptin compared with the current standard of care. |
| Full pharmacoeconomic assessment commissioned by HSE |
29/10/2025 |
