19/05/2026 Cullinan’s CLN‑049 earns FDA Orphan Drug Designation in relapsed/refractory AML FL118 Receives Dual FDA Designations for Osteosarcoma, Advancing Rare Pediatric Cancer Treatment Cullinan’s CLN‑049 earns FDA Orphan Drug Designation in relapsed/refractory AML (Ref) LN-049, a novel FLT3 x CD3 T cell engager, has received Orphan Drug Designation for relapsed/refractory acute myeloid leukemia (AML) CLN-049 is in Phase 1 trials, including multiple ascending dose studies i

13/01/2026 Ipsen's IPN60340 received the FDA Breakthrough Therapy Designation for AML Ipsen's IPN60340 received the FDA Breakthrough Therapy Designation for AML (Ref) The US FDA granted the Breakthrough Therapy Designation to Ipsen's IPN60340 (anti-BTN3A) in combination with venetoclax and azacitidine in first line unfit acute myeloid leukemia. Christelle Huguet, PhD, EVP and Head of R&D, Ipsen: “This Breakthrough Therapy Designation recognizes both the urgent need for new tr

01/12/2025 Cullinan Therapeutics' CLN-049 received the FDA fast track designation for R/R Acute Myeloid Leukemia Astellas Pharma and Pfizer's Type II variation application for perioperative enfortumab vedotin + pembro has been valiated by the EMA Cullinan Therapeutics' CLN-049 received the FDA fast track designation for R/R Acute Myeloid Leukemia (Ref) The US FDA granted the fast track designation to Cullinan Therapeutics' CLN-049 (FLT3 x CD3 bispecific T cell engager) for th

13/11/2025 Kura Oncology and Kyowa Kirin's ziftomenib received FDA approval for certain R/R AML pts (Ref) The US FDA granted full approval to Kura Oncology and Kyowa Kirin's ziftomenib (KOMZIFTI™; menin inhibitor) for adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible NPM1 mutation who have no satisfactory alternative treatment options. Troy Wilson, President and CEO, Kura Oncology: “KOMZIFTI combines compelling efficacy, a favor

05/11/2025 Leukogene Therapeutics' LTI-214 received the FDA orphan drug designation for AML (Ref) The US FDA granted the orphan drug designation to Leukogene Therapeutics' LTI-214 (M2T-CD33; myeloid-targeted immunotherapy) for the treatment of Acute Myeloid Leukemia (AML). Sandeep Gupta, CEO, Leukogene: “We are honored that the FDA has recognized the therapeutic promise of LTI-214 by granting Orphan Drug Designation. AML remains one of the most challenging hematologic cancers

15/09/2025 Daiichi and Merck's raludotatug deruxtecan received the Breakthrough Therapy Designation for Ovarian, Primary Peritoneal, or Fallopian Tube Cancers (Ref) The US FDA granted the Breakthrough Therapy Designation to Daiichi Sankyo and Merck & Co./MSD's raludotatug deruxtecan (R-DXd; CDH6-directed ADC) for the treatment of adult patients with platinum-resistant epithelial ovarian, primary peritoneal or fallopian tube cancers expressing CDH6 who have received prior trea

05/09/2025 CERo Therapeutics' CER-1236 received the FDA Fast Track Designation for AML (Ref) The US FDA granted the Fast Track Designation to CERo Therapeutics' CER-1236 (TIM-4 ligand targeting CER-T therapy) for the treatment of Acute Myeloid Leukemia (AML). Chris Ehrlich, CEO, CERo Therapeutics: “These FDA designations are important additional validation with regard to the urgency of the condition as well as the potential that the agency sees in the existing data as submitt

18/07/2025 ImCheck’s ICT01 received the Orphan Drug Designation for Acute Myeloid Leukemia (Ref) The US FDA granted the Orphan Drug Designation to ImCheck Therapeutics' ICT01 (anti-BTN3A) for the treatment of acute myeloid leukemia Stephan Braun, CMO, ImCheck Therapeutics: "Receiving FDA orphan drug designation for ICT01 is a significant recognition of ICT01’s innovative therapeutic potential to meet the urgent unmet medical needs of AML patients. This important regulatory m