FDA Grants Orphan Drug Designation (ODD) Status to NeoImmuneTech’s NT-I7 for the treatment of Glioblastoma Multiforme
NeoImmuneTech, Inc. (NIT) announced that the U.S. Food and Drug Administration (FDA) has granted NT-I7 (efineptakin alfa) (rhIL-7-hyFc) Orphan Drug Designation for the treatment of Glioblastoma Multiforme.
Despite decades of research, GBM remains one of the deadliest and hardest-to-treat cancers. More than 13,000 Americans are expected to be diagnosed with GBM in 2022. Standard treatments using surgery, radiation, TMZ and TTFs have failed to date to greatly improve survival, and there is no standard treatment for recurrence, which is inevitable. Also, among the multiple factors impacting the survival of GBM patients, the current treatment options expose patients to a severe and prolonged systemic lymphopenic state known as “treatment-related lymphopenia” (TRL). TRL is associated with shorter survival both in GBM and other solid tumors. With its potential to restore lymphocyte levels and subsequently reverse systemic lymphopenia, NT-I7 may offer promising ways to effectively treat GBM, if confirmed by its development program.
Dr Se Hwan Yang, Ph.D., President and Chief Executive Officer of NeoImmuneTech, Inc. said: “We are excited that the FDA granted NT–I7 an ODD in the treatment of glioblastoma multiforme. This decision adds further credibility to our existing clinical evidence that NT-I7 has the potential to bring a new essential therapy option to people with advanced/metastatic GBM who have undergone prior chemo-radiation therapy. We look forward to continuing our collaboration with FDA, as we explore the benefits of NT-I7 in treating people with GBM in combination with other anti-cancer treatments, including immunotherapies”.
NT-I7 has been studied in several robust phase I and II clinical trials and has demonstrated the potential to amplify T cells across the subsets, boost the immune system, and prolong the anti-tumor response in people with GBM and other solid tumors.[2] Current studies in GBM include study NIT-107 in newly diagnosed GBM patients and study NIT-120 in recurrent GBM patients. Preliminary data from study NIT-107 presented at Society for Immunotherapy of Cancer Annual Meeting (SITC), in November 2021, showed favorable trends of progression free survival (PFS) and overall survival (OS) in NT-I7 treated high-grade gliomas (HGG) patients after chemoradiotherapy.
The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. Receiving ODD may help to expedite and reduce the cost of development, approval, and commercialization of a therapeutic agent (FDA filing fee waive, guaranteed sales period after approval, etc).