Apraglutide for Acute GvHD Earns Orphan Drug Status

In some cases, serious complications occur following allogeneic stem cell or bone marrow transplants. For example, in patients with Graft-versus-host disease (GvHD), the donor cells may recognize patient cells as foreign, prompting an immune response against those cells. Ultimately, this causes a variety of health issues. Biopharmaceutical company VectivBio Holding AG (“VectivBio”) is aiming to address gastrointestinal damage in GvHD with apraglutide, a GLP-2 analogue. In a news release from late June 2021, VectivBio shared that apraglutide received Orphan Drug designation from the FDA for the treatment of Acute GvHD (aGvHD).


According to VectivBio’s CMO, Dr. Omar Khwaja, MD, PhD, gastrointestinal damage is one of the leading causes of mortality associated with aGvHD. Because of this, a new therapeutic option is needed to improve patient outcomes. Thus, VectivBio developed apraglutide. This long-lasting analogue peptide of GLP-2 works to regenerate gastrointestinal tissue and function, as well as reduce inflammation. As a result, preclinical and clinical studies highlight the therapy’s potential to improve quality of life (QOL) and lower mortality rates. Outside of aGvHD, VectivBio is also exploring apraglutide as a potential therapy for patients with short bowel syndrome (SBS). Altogether, VectivBio hopes to initiate a Phase 2 clinical trial analyzing apraglutide for aGvHD before mid-2022.

The recent Orphan Drug designation opens VectivBio up to a variety of incentives. This program was designed to expedite the development of drugs or biologics designed to treat patients with rare diseases. Overall, the FDA defines “rare diseases” as those affecting under 200,000 Americans. As a result of this designation, VectivBio will receive tax credits, fee waivers, increased regulatory assistance, and 7 years of market exclusivity if/when apraglutide is approved.

Graft-versus-Host Disease (GvHD)

When patients receive an allogeneic transplant, it means the transplanted cells or bone marrow come from a donor. While this can be beneficial, one potential complication includes Graft-versus-Host disease (GvHD). When donor cells see the recipient as “foreign,” the cells prompt an immune response, causing a host of symptoms. Acute GvHD (aGvHD) typically occurs within 6 months following a transplant. Normally, this form affects the liver, skin, and gastrointestinal tract. Alternately, chronic GvHD (cGvHD) occurs over 3 months following a transplant, and symptoms could last for a lifetime. cGvHD may affect one, or multiple, organs.

Symptoms of aGvHD include:

  • Jaundice (yellowing of the skin and eyes)
  • Abdominal pain
  • Nausea and vomiting
  • Diarrhea
  • Appetite loss
  • Pruritus (extreme itching)
  • Skin rashes or redness, particularly blistering or flaky skin on the palms, soles, and trunk

Next, symptoms of cGvHD depend on which part of the body is affected. These symptoms may include:

  • Unintended weight loss
  • Painful mouth/throat ulcers
  • Fatigue
  • Shortness of breath/difficulty breathing
  • Chronic cough
  • Muscle and joint weakness, stiffness, and pain
  • Joint pain and stiffness
  • Appetite loss
  • Nausea and vomiting
  • Abdominal pain or swelling
  • Jaundice
  • Diarrhea
  • Severely dry eyes or mouth
  • Skin rashes
  • Difficulty eating
  • Temperature intolerance
  • Hard, brittle nails (and/or nail loss)
  • Premature hair loss
  • Vaginal or penile itching, dryness, or pain
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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