Recently, the U.S. Food and Drug Administration (FDA) granted special designation status to treatments for myelodysplastic syndromes and advanced triple-negative breast cancer.
Breakthrough Therapy Designation for Venetoclax in Combination With Azacitidine for Myelodysplastic Syndromes
Venetoclax in combination with azacitidine has been granted Breakthrough Therapy designation by the FDA for the treatment of adult patients with previously untreated intermediate-, high-, and very high–risk myelodysplastic syndromes (MDS) based on the revised International Prognostic Scoring System.
This designation was granted based on interim results from the M15-531 study (ClinicalTrials.gov identifier NCT02942290), a phase Ib, open-label, nonrandomized, multicenter, dose-finding study evaluating venetoclax in combination with azacitidine in treatment-naive patients with higher-risk MDS comprising a dose-escalation portion and a safety expansion portion.
The primary objectives of the study are to assess the safety profile and pharmacokinetics and determine the recommended phase II dose and dosing schedule of the combination. The response criteria specified in the M15-531 study are based on the modified International Working Group 2006 response criteria for MDS.
Fast Track Designation for Trilaciclib Plus Chemotherapy in Advanced Triple-Negative Breast Cancer
The FDA granted Fast Track designation to trilaciclib in combination with chemotherapy for the treatment of locally advanced or metastatic triple-negative breast cancer. Trilaciclib is currently being evaluated in PRESERVE 2, a pivotal phase III, randomized, double-blind, placebo-controlled study (ClinicalTrials.gov identifier NCT04799249) in patients receiving first- or second-line gemcitabine and carboplatin chemotherapy for triple-negative breast cancer.
“Fast Track designation underscores the urgent need for innovative drugs that can significantly improve triple-negative breast cancer outcomes,” said Raj Malik, MD, Chief Medical Officer at G1 Therapeutics. “It provides an important pathway to help expedite the development and regulatory review of trilaciclib in this indication. We look forward to working closely with the FDA as we advance this pivotal program in triple-negative breast cancer and continue to work to unlock the broader potential of this ‘pipeline-in-a-molecule’ compound that we hope will help patients across multiple tumor types.”
