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FDA Grants Orphan Drug Status to Novel Chemotherapy Agent for Multiple Myeloma

By: Sarah Campen, PharmD
Posted: Wednesday, February 21, 2018

On February 7, 2018, the U.S. Food and Drug Administration granted Orphan Drug status to the chemotherapy agent PT-112 for the treatment of multiple myeloma. Orphan drug status is expected to expedite the clinical development and regulatory review of the drug.

PT-112 is a first-in-class platinum-pyrophosphate conjugate that triggers cancer cell death, which releases particles that recruit and activate T-cells against tumors. It has demonstrated an affinity for specific compartments within the bone, indicating PT-112 may be effective in treating hematologic malignancies.

An abstract and poster session presented at the 2017 American Society of Hematology (ASH) Annual Meeting (Abstract 1797) reported that PT-112 reduced cancer cell activity in myeloma mouse models. The agent also demonstrated potent synergy in in vitro systems with standard-of-care agents bortezomib and lenalidomide. P. Leif Bergsagel, MD, of the Mayo Clinic, Scottsdale, Arizona, and colleagues concluded the findings “provide a strong rationale” for study in patients with multiple myeloma.

Dr. Bergsagel and colleagues have begun enrolling the first cohort of patients into the phase I/II study of PT-112 as a single agent in relapsed or refractory multiple myeloma and are currently recruiting more participants. They aim to assess the safety and toxicity of this agent in humans and determine the optimal dose to further evaluate its safety, effectiveness, and tolerability profile.



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