FDA approves pair of therapies for rare pediatric cancers: Novartis’ Lutathera and Day One’s Ojemda

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The FDA has signed off on two novel therapies targeting rare pediatric cancers. Novartis’ Lutathera targets aggressive gastroenteropancreatic neuroendocrine tumors (GEP-NETs) in children 12 and up, while Day One Biopharma’s Ojemda (tovorafenib) tackles treatment-resistant BRAF-mutated relapsed or refractory pediatric low-grade glioma (pLGG) with a BRAF fusion or rearrangement, or BRAF V600 mutation

These approvals offer new treatment possibilities for young patients with rare pediatric cancers who previously had limited treatment options. The FDA’s decisions highlight the growing focus on developing targeted therapies for these underserved malignancies.

Lutathera targets rare gastrointestinal cancers in children

Lutathera, a radioligand therapy (RLT) from Novartis, has won an indication for the treatment of pediatric patients 12 years and older with som…

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Prix Galien Awards: The most innovative biotech, pharma, and orphan drugs of 2023

Prix Galien

Prix Galien names 2023 winners in pharma and biotech

In the world of medical innovation, few accolades carry as much weight as the Prix Galien Awards, which highlight the advances in biotech, pharmaceuticals, and other domains. The 2023 winners include Bristol Myers Squibb’s Camzyos (mavacamten) as the best biotechnology product and Lilly’s Mounjaro (tirzepatide) and Novo Nordisk’s Ozempic (semaglutide) sharing the honor for best pharmaceutical product. Finally, Boehringer Ingelheim’s Spevigo (spesolimab) and CSL / uniQure’s Hemgenix were twin winners in the rare/orphan disease category.

Here, we take a closer look at the Prix Galien 2023 winners in pharma, biotech and rare/orphan disease.

Camzyos: A new chapter in cardiomyopathy therapy

First winning FDA approval in April 2022, Camzyos is a novel option for¬†symptomatic NYHA class II-III obstructive hypertroph…

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