PU-H71 receives orphan drug designation for myelofibrosis

Micrograph showing myelofibrosis
The US Food and Drug Administration (FDA) has granted orphan drug designation to PU-H71 to treat patients with myelofibrosis. The drug specifically targets the epichaperome, a network of high-molecular- weight complexes found in multiple diseases, including cancer and neurologic disorders. These complexes enhance cellular survival, irrespective of tissue of origin or genetic background. According to research published in Nature Reviews Cancer, Pu-H71 interferes with the epichaperome function in diseases and does not affect normal cells. PU-H71 is being evaluated in a phase 1b trial in myelofibrosis and advanced metastatic breast cancer. [Read Article]

Ivosidenib active in R/R IDH1-mutated AML patients

Poster session at ASCO 2018
Photo by © ASCO/Scott Morgan 2018
The investigational drug ivosidenib, an inhibitor of the mutant IDH1 enzyme, achieved complete remission (CR) rates of 32% and an overall response rate of 42% in relapsed/refractory (R/R) patients with acute myeloid leukemia (AML) and IDH1 mutation, according to investigators. In addition, overall survival (OS) in patients who achieved CR more than doubled compared with those in the overall study population. Fewer patients with CR had febrile neutropenia and infectious complications, and 25% of patients with CR were able to clear the IDH1 clone. [Read Article]

FDA grants pembrolizumab accelerated approval for PMBCL

Pembrolizumab (Keytruda)
Photo courtesy of Merck
The US Food and Drug Administration (FDA) granted accelerated approval to the anti-PD-1 therapy pembrolizumab (Keytruda) for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL). The indication also includes patients who have relapsed after 2 or more prior lines of therapy. Pembrolizumab had received priority review for PMBCL late last year and also has orphan drug designation and breakthrough therapy designation for this indication. The FDA based its approval on data from the KEYNOTE-170 trial. [Read Article]