BioMarin Pharmaceuticals has received US approval for its enzyme therapy, Palynziq, to treat the rare brain-threatening phenylketonuria (PKU), a genetic disorder that prevents the breakdown of amino acids. The drug helps with PKU by restoring the patient’s ability to breakdown phenylalanine, where high levels are toxic to the brain, allowing the reduction of its concentration in their blood. This approval is deemed “an important milestone” for adults living with the condition however, it comes with a black box warning for anaphylaxis, which was experienced by 9% of the clinical trial population. Palynziq is also currently under EU review. Please click here to read more on this story from a PharmaTimes article.
AZ have revealed top-line results from a phase 3 trial in lung cancer, stating that their cancer immunotherapy, Imfinzi, delivered a survival benefit when compared to placebo in stage 3 unresectable non-small cell lung cancer (NSCLC). The results showed that patients given Imfinzi after chemoradiation therapy (CRT) extended median progression-free survival (PFS) by 11.2 months compared to placebo. This strengthens the position of the PD-L1 inhibitor, which was approved in February, and may improve its uptake for the indication and breakout into the later, metastatic NCSLC setting. Please click here to read further from BioPharmaDIVE.
A combo of AbbVie/J&J’s Imbruvica and Roche’s Gazyva has been shown to improve progression free-survival (PFS) in chronic lymphocytic leukaemia (CLL), setting it up for the first-line setting. The phase 3 trial (iLLUMINATE) showed that the first-in-class Bruton’s tyrosine kinase (BTK) combo, when compared with the chlorambucil/Gazvya combo which is recommended as a “category 1 treatment”, significantly improved PFS. The data has reportedly been shared with regulators in hope that it will widen the treatment’s indication into the first-line setting. If you would like to read more, please click here for a PharmaTimes article.