US FDA awards Supplemental Orphan Drug Designation to SurVaxM to now include treatment of Malignant Glioma

Orphan Tree
  • The U.S. Food and Drug Administration (FDA) has granted supplemental orphan-drug designation (ODD) to SurVaxM for use in patients with malignant gliomas.
  • New orphan designation broadens the patient population for potential use of SurVaxM to include forms of adult and pediatric malignant gliomas other than glioblastoma.
  • ODD expands market exclusivity of SurVaxM beyond its previous designation of adult glioblastoma.

Buffalo, N.Y., May 30, 2024  —   MimiVax Inc, a clinical-stage biotechnology company developing immunotherapeutics for cancer and autoimmune diseases, today announced that the United States Food and Drug Administration (FDA) has awarded a supplemental orphan drug designation to MimiVax’s SurVaxM vaccine to now include malignant glioma. Currently, SurVaxM vaccine is being studied as a treatment for newly diagnosed glioblastoma (nGBM) in a phase 2b randomized clinical study (the SURVIVE trial) which will assess the efficacy of SurVaxM in a large patient population with nGBM. This supplemental orphan drug designation greatly expands the potential for SurVaxM’s use in other forms of pediatric and adult malignant gliomas. An orphan drug designation plays a critical role in encouraging the development of treatments for rare diseases, ultimately improving the lives of patients who might otherwise have few or no treatment options.

Gliomas are brain tumors that are initiated from glial cells that and support nerve cells in the brain and spinal cord. Gliomas comprise about 80% of all malignant primary brain tumors and include such tumors as astrocytomas, oligodendrogliomas and ependymomas.

Receiving orphan drug designations for SurVaxM emphasizes the critical demand for novel and enhanced therapeutic options for people living with malignant gliomas,” said Michael Ciesielski, Ph.D., CEO of MimiVax. “We hope that this designation may help to advance SurVaxM’s application for important indications beyond adult GBM including for pediatric brain cancers.”

Orphan designation is granted by the FDA to advance the evaluation and development of new therapies intended to treat a rare disease or condition that generally affects fewer than 200,000 individuals each year in the United States. Under the Orphan Drug Act, regulatory agencies may offer certain benefits to expedite the approval process for orphan drugs, recognizing the urgent need for treatments for rare diseases. This can include accelerated review timelines and flexibility in clinical trial design, tax credits for qualified clinical trials, FDA user-fee benefits, and seven years of market exclusivity in the United States after new drug approval.

About MimiVax, Inc

MimiVax, Inc. is a Delaware-incorporated, clinical-stage biopharmaceutical company focused on developing immunotherapies for cancer treatment. MimiVax has conducted clinical trials to evaluate the safety and efficacy of SurVaxM in various types of cancer, including glioblastoma (a type of brain cancer), multiple myeloma, neuro-endocrine tumors (NET) and pediatric relapsed high grade glioma, medulloblastoma, ependymoma and diffuse intrinsic pontine glioma (DIPG). These trials have shown promising signs of immune response and potential clinical benefit. For more information on MimiVax, visit or email [email protected].

About SurVaxM

SurVaxM  is an experimental cancer vaccine developed by MimiVax. It is designed to target a protein called survivin, which is found in many cancer cell types. Survivin plays a role in promoting cancer cell survival, tumor growth, and resistance to cell death. SurVaxM is delivered subcutaneously where it stimulates a patient’s immune response against tumor cells with the intent to prevent future disease recurrence. While still in the experimental stages, SurVaxM holds potential as a new treatment option for patients with survivin-expressing forms of cancer.