FDA approves sacubitril/valsartan for pediatric HF
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Novartis announced that sacubitril/valsartan has been approved by the FDA for the treatment of children aged 1 year and older with symptomatic HF and systemic left ventricular systolic dysfunction.
The drug (Entresto) reduces N-terminal pro-B-type natriuretic peptide, which leads to improved CV outcomes, according to a press release from the company.
“Pediatric heart failure is extremely serious and carries a substantial burden,” Andrea Baer, executive director at Mended Little Hearts, said in the release. “Given the significant unmet need for heart failure treatments that are proven safe and effective in pediatric patients, this approval and the availability of a new treatment option is great news for these children and their families.”
The approval was based on 12-week analysis of the PANORAMA-HF trial, which showed that sacubitril/valsartan reduced NT-proBNP in children with HF from systemic LV systolic dysfunction. Data from the PARADIGM-HF trial were also considered for this approval, which also showed improved outcomes and reduced NT-proBNP in adults.
“The current clinical management of pediatric systolic heart failure includes the use of several medicines based mostly on data from adult studies, and there is limited data in children with heart failure in clinical trials,” David Soergel, MD, global head of cardiovascular, renal and metabolic drug development for Novartis, said in the release. “PANORAMA-HF is ongoing to secure 52-week follow-up data for the full study population.”
Disclosure s : Baer is an employee of Mended Little Hearts. Soergel is an employee of Novartis.
Perspective
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Jason F. Goldberg, MD
The PANORAMA-HF trial showed decreased mortality and hospitalizations in adults with HF.
While the pediatric arm of this study has shown initial promising results, it is often difficult for pediatric HF trials to replicate the significant gains seen in adult populations. It is reassuring that the FDA understands this dilemma and has approved the therapy for children.
I have had multiple children who have had insurance coverage denied for sacubitril/valsartan despite being of adult size. With this approval, I will be able to treat these patients and possibly improve their cardiac outcomes in a similar fashion to that which has been shown in adult trials.
Much of the practice of pediatric HF includes the extrapolation of adult HF therapies to children, often without FDA approval. The preliminary work by Dr. Shaddy and Novartis has helped to secure this important distinction.
Many of us believe that this therapy will allow many patients to avoid long-term milrinone therapy or transplantation.
Though it is yet to be seen if significant benefits exist for younger patients with HF, this FDA expansion will allow for these hypotheses to be tested both within and beyond the pediatric arm of PANORAMA-HF.
One particular population in which we have used this therapy is the cardio-oncology population at St. Jude Children’s Research Hospital. Because children with active malignancies are not transplant candidates, sacubitril/valsartan may be an invaluable tool to overcome acute-chemotherapy-induced cardiomyopathy and avoid mortality.
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