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Emapalumab FDA approved in adult and paediatric patients with primary hemophagocytic lymphohistiocytosis
By: scienterrific | Last updated: 21 November 2018 | In: US FDA Onc\Haem Approvals, Haematology
Article Keywords
paediatric, emapalumab, Gamifant, haemophagocytic lymphohistiocytosis, HLH, interferon-γ, Novimmune, lymphoma, FDA
On 20 November 2018, the FDA approved the interferon-γ neutralising monoclonal antibody emapalumab (Gamifant®, Novimmune) for the treatment of adult and paediatric (newborn and older) patients with refractory, recurrent, or progressive primary haemophagocytic lymphohistiocytosis (HLH) or in patients intolerant to conventional HLH therapy.
Trial NI-0501-04
The multicentre, open-label, single-arm trial NI-0501-04 (NCT01818492) enrolled 27 paediatric patients with refractory, recurrent, or progressive primary HLH or those who were intolerant to conventional HLH therapy. Dexamethasone was provided as background HLH treatment for emapalumab doses between 5 to 10 mg/m2/day. All patients received prophylaxis for fungal infections, Herpes Zoster, and Pneumocystis jirovecii prior to emapalumab infusion. The primary efficacy endpoint was the overall response rate (ORR) at the end of treatment which was defined as either a complete or partial response or a ≥50% improvement from baseline of ≥3 HLH abnormalities.
Among the 27 paediatric patients, 7 complete and 8 partial responses were recorded and two patients had an HLH improvement resulting in an ORR of 63% (95% CI, 0.42-0.81; P=0.013).
Safety
Common adverse reactions in ≥20% of patients included infections, hypertension, infusion-related reactions, and pyrexia.
Disclaimer
This article is not medical advice. Patients should seek personal assessment by a licenced specialist. Physicians are recommended to read the full publication(s) as cited in the article before making medical decisions. This article does not supersede nor replace the published article(s).
© Copyright 2018 MediPaper Medical Communications Ltd.
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© Copyright 2018 MediPaper Medical Communications Ltd.
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