ARTICLE
The first pharmaceutical therapy for the rare disease tenosynovial giant cell tumors (TGCT) won FDA approval. Pexidartinib (Turalio) was OK’d for adults with symptomatic TGCT associated with severe morbidity or functional limitation and not responsive to surgery. The approval came with the requirement of a boxed warning about a risk of serious and potentially fatal liver disease. During the randomized trial, adverse events occurred almost four times as often in the pexidartinib group, and serious adverse events occurred more than six times as often (13% vs 2%). Additionally, 38% of patients in the pexidartinib group required dose reductions or interruptions as compared with 10% of the placebo group, and 13% of patients randomized to pexidartinib stopped treatment versus 0% in the placebo group. Source: https://www.medpagetoday.com/hematologyoncology/othercancers/81403