FDA Grants Orphan Drug Designation to TTAC-0001 for Glioblastoma Multiforme


FDA grants orphan drug designation to PharmAbcine Inc for TTAC-0001, the company’s leading clinical compound, for treatment of GBM.

Yesterday, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to PharmAbcine Inc for TTAC-0001 for treatment of Glioblastoma Multiforme (GBM).

TTAC-0001 is an investigational, highly selective and potent anti-VEGFR2 (KDR/flk-1) mAb in clinical development for rGBM indications. Over-expressed in most malignant tumors, such as gastric, liver, non-small cell lung cancer (NSCLC), ovarian, brain, colorectal, and breast cancers, VEGFR2 is also a signaling pathway that is a key regulator for tumor angiogenesis.1

Support for evaluating TTAC-0001 in GBM, rGBM and Avastin (bevacizumab) refractory GBM include an increased need for understanding the role of VEGF/VEGFR2 in the tumor vessel formation, immune suppressive modulation of tumor microenvironment (TME) and the function of the antagonist molecule to disorganized tumor vessel normalization, immune supportive modulation, and ultimately tumor vessel disruption.1

Dr. Jin-San Yoo, president and chief executive officer of PharmAbcine, Inc., expressed his hope for the drug’s future. “We are very pleased with this Orphan Drug Designation from FDA for TTAC-0001 for GBM treatment since we have been preparing for clinical studies of TTAC-0001 plus KEYTRUDA® (pembrolizumab) combo therapy for recurrent GBM in addition to TTAC-0001 mono therapy for recurrent GBM progressed after bevacizumab treatment. It will accelerate the progress of TTAC-0001 to provide clinical benefit in the treatment of GBM.”1

TTAC-000’s potential also bears promise due to results from Phase 2 of the drug’s clinical trial, which commenced in January 2017 and closed in August 2017 with data still being recorded and collected (for up to a year post completion). The primary outcomes of the study were to measure the efficacy and safety of the drug as a treatment for recurrent GBM.

Among the data collected from the 12 adult participants with diagnosed primary GBM, the most important measures included: progression-free survival (PFS) at the 6-month time point, objective response rate (ORR), disease control rate (DCR), and overall survival (OS).

Participants were split into 3 different study arms. Patients in cohort 1 were treated with with 8mg/kg of TTAC-0001 on day 1, day 8 and day 15 in every 4 weeks of cycle, while patients in the second cohort were treated with 12mg/kg of TTAC-0001 on day 1, day 8 and day 15 in every 4 weeks of cycle, and patients in cohort 3 were treated with 12mg/kg of TTAC-0001 weekly in every 4 weeks of cycle.2

Results are still being collected from the Phase 2 clinical trial, but the FDA’s orphan drug designation for TTAC-0001 is reason for optimism.

For more data from studies pertaining to the rare disease community, follow Rare Disease Report on Facebook and Twitter.


  1. “PharmAbcine Announces FDA Orphan Drug Designation Granted to TTAC-0001 for Glioblastoma Multiforme.” BusinessWire. 2, Apr. 2018
  2. “Trial to Evaluate the Safety of TTAC-0001(Tanibirumab) in Recurrent Glioblastoma.” National Institute of Health 26 Jan. 2018
Recent Videos
Signs and Symptoms of Connective Tissue Disease
How Gene and Cell Therapy Is Developing in Dermatology
Joyce Teng, MD, PhD, discusses how therapeutic advances in fields like epidermolysis bullosa should progress treatment discourse in other rare dermatoses.
The Prospect of Pz-cel in RDEB Treatment, with Peter Marinkovich, MD
Comparing New Therapies for Dystrophic Epidermolysis Bullosa
Reviewing 2023 with FDA Commissioner Robert M. Califf, MD
Dunia Hatabah, MD | Image Credit: HCPLive
Ricky Safer: What Clinicians Need to Know About PSC
Ryan T. Fischer, MD: Long-Term Odevixibat Benefit for Alagille Syndrome
Saeed Mohammad, MD: IBAT Inhibitors for Cholestatic Disease
© 2024 MJH Life Sciences

All rights reserved.