Recently, the U.S. Food and Drug Administration (FDA) granted designations to agents for metastatic castration-resistant prostate cancer, recurrent glioblastoma, and pediatric acute myeloid leukemia.
Fast Track Designation for EPI-7386 in Metastatic Castration-Resistant Prostate Cancer
The FDA granted Fast Track designation to EPI-7386, an oral, highly selective N-terminal domain inhibitor of the androgen receptor, for the treatment of adult male patients with metastatic castration-resistant prostate cancer resistant to standard-of-care treatment.
A phase I clinical trial expects to enroll approximately 18 patients with metastatic castration-resistant prostate cancer in the dose-escalation part of the study at selected clinical sites in the United States and Canada, with an additional 10 patients planned to be enrolled in a dose-expansion cohort involving additional clinical sites. The study will evaluate the safety and tolerability of EPI-7386 while additionally characterizing the pharmacokinetic, biologic, and antitumor effects of therapy.
Fast Track Designation for RNL in Glioblastoma
The FDA gave Fast Track designation to an investigational drug—rhenium nanoliposome (RNL)—for the treatment of patients with recurrent glioblastoma. The nanoliposome-encapsulated radioisotope is designed to safely, effectively, and conveniently deliver a high dose of radiation—of up to 25 times greater concentration than currently used external-beam radiation therapy—directly into the brain tumor.
The drug is being evaluated in the multicenter ReSPECT phase I dose-finding clinical trial. Recently, the ReSPECT trials’ data and safety monitoring board approved proceeding to cohort 6 of the trial, which includes increasing both the drug volume and radiation dose to 8.8 mL and 22.3 mCi, respectively.
FDA Granted Pediatric Disease Designation for OXi-4503 in AML
The FDA has designated OXi4503 (combretastatin A1-diphosphate; CA1P) for the treatment of acute myeloid leukemia (AML) due to genetic mutations that disproportionately affect pediatric patients as a drug for a “rare pediatric disease,” as defined in section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360ff[a][3]).
OXi4503 in combination with the standard chemotherapy cytarabine was generally well tolerated by adult patients with AML, and a maximum tolerated dose level of OXi4503 was identified as the recommended dose for further clinical development of this novel two-drug combination. In 26 evaluable patients with AML, there were four complete remissions and one partial remission. The complete responses were associated with > 1-year overall survival times.
The combination therapy exhibited a manageable toxicity and a promising benefit to risk profile in older adults with relapsed AML. Four of the five objective responders were aged 65 or older with adverse cytogenetic features.
About the Rare Pediatric Disease Voucher Program
The FDA grants rare pediatric disease designation for diseases with serious or life-threatening manifestations that primarily affect people aged from birth to 18 years and that affect fewer than 200,000 people in the United States. Under the FDA's Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval of a new drug application or biologics license application for a product for the prevention or treatment of a rare pediatric disease may be eligible for a voucher, which can be redeemed to obtain priority review for any subsequent marketing application and may be sold or transferred.
