Neihulizumab for GvHD Receives Fast Track Designation

In a recent press release, biotech company AltruBio Inc. shared that its therapy neihulizumab (AbGn-168H) received Fast Track designation for the treatment of patients with steroid refractory acute graft-versus-host disease (GvHD). Steroid refractory refers to a condition which does not or has not previously responded to steroid treatment.


For years, AltruBio has been working to develop unique therapeutic options to fill unmet patient needs. In this case, GvHD can be difficult to treat if the steroids or other treatment options are not working. Although treatments do exist, many patients are still left without options, which makes neihulizumab a potentially attractive option.

AltruBio describes neihulizumab as:

a first-in-class antibody targeting a variety of autoimmune and immune-mediated inflammatory diseases.

This immune checkpoint regulator targets PSGL-1 and reduces the burden of overactive T-cells, a type of immune cell. So far, 170 patients have received neihulizumab. At the moment, researchers are evaluating neihulizumab for GvHD in a Phase 1b clinical trial.

The drug received Fast Track designation. This status is meant for the development and review of drugs designed to fill an unmet need, which the FDA defines as:

providing a therapy where none exists or providing a therapy which may be potentially better than available therapy.

To fill an unmet need, a drug must show increased efficacy over current treatments, reduce the burden of prior treatments, or improve patient outcomes. Beyond Fast Track designation, neihulizumab also received Orphan Drug designation. This status is for drugs designed to treat those with rare diseases (affecting 200,000 or less Americans).

Graft-versus-Host Disease (GvHD)

There are two forms (acute and chronic) of graft-versus-host disease (GvHD), a complication which may occur after allogeneic stem cell or bone marrow transplants. This occurs when the donor’s T-cells view the host’s cells as foreign and attack the recipient’s body. Chronic GvHD typically occurs over 3 months following transplant and may last a lifetime. Symptoms of chronic GvHD include:

  • Dry eyes or mouth
  • Appetite loss
    • This may also be associated with difficulty eating, or new food and temperature sensitivities.
  • Painful mouth and throat ulcers
  • Skin rash
  • Hard, brittle nails
  • Hair and nail loss
  • Gum disease and/or tooth decay
  • Nausea and vomiting
  • Difficulty breathing
  • Chronic cough
  • Unintended weight loss
  • Jaundice (yellowing of the skin and eyes)

Alternately, acute GvHD typically occurs within the first 6 months following a transplant. While chronic GvHD can affect multiple organs, the acute version usually impacts the skin, liver, and gastrointestinal tract. Symptoms include:

  • Nausea and vomiting
  • Diarrhea
  • Abdominal pain
  • Jaundice
  • Itchy skin or skin rashes
    • This often occurs on the feet.
  • Appetite loss
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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