Drug updated on 3/28/2024
| Dosage Form | Injection: (intravenous; 10 mg/2 mL, 50 mg/10 mL, 100 mg/20 mL) |
| Drug Class | Interferon gamma blocking antibodies |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- For the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy.
Summary
- Emapalumab-lzsg (Gamifant) is a human anti-interferon-γ antibody used for the treatment of primary hemophagocytic lymphohistiocytosis (HLH), particularly in patients with refractory, recurrent or progressive disease or intolerance to conventional HLH therapy.
- The document reviewed was a Randomized Controlled Trial that investigated the efficacy and safety of emapalumab-lzsg (Gamifant) when administered with dexamethasone in both previously treated and untreated patients who were 18 years old or younger suffering from primary HLH.
- A total of 34 patients participated in this trial; 27 had received prior treatment while seven were previously untreated. All participants could enter into a long-term follow-up study until one year after allogeneic hematopoietic stem-cell transplantation, or until one year after their last dose if transplantation was not performed.
- In terms of effectiveness, it was found that approximately 63% of the previously treated group responded positively to Gamifant as did around 65% percent among those who received an infusion - significantly higher than the prespecified null hypothesis rate at which response would be considered successful at just over half (40%).
- Regarding safety considerations, no organ toxicity associated with Gamifant use was reported during this trial period but severe infections developed in ten individuals undergoing treatment; only one patient discontinued due to disseminated histoplasmosis infection.
- Overall survival rates post-trial showed promising results: about three-quarters – specifically, 74%–of those formerly on other treatments survived as did roughly similar proportion—71%, among all recipients indicating potential benefits outweighing risks involved for most users given its targeted therapeutic action against primary HLH condition.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Gamifant (emapalumab-lzsg) Prescribing Information. | 2022 | Sobi Inc., Waltham, MA |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| Emapalumab in children with primary hemophagocytic lymphohistiocytosis. | 34Subjects F: 53% M: 47% | 2020 | The New England Journal of Medicine |
Sex Distribution:
F:53%
M:47%
34Subjects
Year:
2020
Source:The New England Journal of Medicine
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Emapalumab-lzsg (Gamifant). | 2023 | https://www.aetna.com/cpb/medical/data/900_999/0948.html |