Sept. 28 Quick Takes: FDA approves Amicus’ Pompe disease therapy

FDA Thursday approved late-onset Pompe disease treatment Pombiliti cipaglucosidase alfa plus Opfolda miglustat from Amicus Therapeutics Inc. (NASDAQ:FOLD). Pombiliti will be priced at $650,000 per year for a patient weighing about 70 kilograms, which is “slightly below” the cost of enzyme replacement therapies for Pompe, Amicus said. The product has been approved by EMA and the U.K.’s MHRA.

Recently approved bispecific antibody Rybrevant amivantamab from the Janssen Biotech Inc. unit of Johnson & Johnson (NYSE:JNJ), plus lazertinib, could become the new standard of care in the large first-line EGFR-mutant non-small cell lung cancer (NSCLC) indication, with data from the Phase III MARIPOSA study suggesting a progression-free survival (PFS) benefit over Tagrisso osimertinib. Janssen reported that the combination led to a statistically significant and clinically meaningful increase in PFS, and a trend toward overall survival benefit over market leading EGFR inhibitor Tagrisso in a planned interim analysis. Last year, Tagrisso from AstraZeneca plc (LSE:AZN; NYSE:AZN), which has a broad label for EGFR mutant NSCLC that includes adjuvant therapy, had $5.4 billion in sales...