Issue: July 25, 2018
June 13, 2018
1 min read

FDA grants orphan drug designation to PU-H71 for myelofibrosis

Issue: July 25, 2018
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Photo of Srdan Verstovsek
Srdan Verstovsek

The FDA granted orphan drug designation to PU-H71 for the treatment of myelofibrosis.

PU-H71 (Samus Therapeutics) is a novel agent that targets the epichaperome, a foundational protein complex present in cancer, neurological disorders and other diseases.

A phase 1b study is underway to assess the safety, tolerability, pharmacokinetics and preliminary efficacy of PU-H71 for patients with myelofibrosis who are taking concomitant ruxolitinib (Jakafi, Incyte).

“Targeting the epichaperome offers an exciting new avenue for treating myelofibrosis and related diseases,” Srdan Verstovsek, MD, PhD, chief of the section for myeloproliferative neoplasms in the leukemia department at The University of Texas MD Anderson Cancer Center, lead clinical research adviser for the phase 1b myelofibrosis study and a HemOnc Today Editorial Board Member, said in a Samus Therapeutics-issued press release. “In myelofibrosis, the epichaperome plays a central role in optimizing the JAK-STAT pathway, allowing JAK2 to form dimers that evade inhibition with a JAK2 inhibitor such as ruxolitinib.

“By inhibiting epichaperome function and breaking this mechanism, we believe PU-H71 can increase anticancer activity of JAK2 inhibitors,” Verstovsek added. “I look forward to seeing how the combination of these therapies can affect outcomes in patients for whom this resistance is associated with poor prognoses.”

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.