FDA Rejects Ropeginterferon alfa-2b for Polycythemia Vera

The drug development process is not always easy or quick. It consists of drug discovery, preclinical and clinical research, and FDA approval. However, sometimes developers are sent back to the drawing board or asked for more information. According to Fierce Pharma, this recently occurred for biopharmaceutical company PharmaEssentia. Although their drug ropeginterferon alfa-2b is sold in the European Union as Besremi, the FDA has rejected the approval request for use in the United States. Before the treatment can become available for patients with polycythemia vera, the FDA has requested additional information.

Ropeginterferon alfa-2b

According to AOP Orphan, ropeginterferon alfa-2b is:

a novel, long-acting, mono-pegylated proline interferon with improved pharmacokinetic properties offering improved tolerability and convenience. Ropeginterferon alfa-2b has Orphan Drug designation in the European Union and the United States of America.

In short, ropeginterferon alfa-2b binds to certain interferon-alpha and beta receptors. As a result, the bone marrow stops producing as many red blood cells. The treatment is subcutaneously administered and comes in pre-filled syringes.

Although the FDA rejected the drug’s approval, the organization did not ask for any additional clinical data. However, the FDA did ask PharmaEssentia to provide additional information and instructions around usage: both how patients would be positively impacted and how doctors or other healthcare workers could effectively administer ropeginterferon alfa-2b. Should PharmaEssentia be able to provide this, it may allow for approval at some point in the future.

Polycythemia Vera (PCV)

JAK2 or TET2 gene mutations cause polycythemia vera, a rare but slow-growing blood cancer. Normally, our bodies regulate the amount of white blood cells, red blood cells, and platelets. However, these gene mutations cause the bone marrow to overproduce red blood cells. At the same time, both white blood cells and platelets are also being overproduced. As a result, people with polycythemia vera have thick blood, which can cause strokes, abnormal bleeding, blood clots, and heart attacks. Polycythemia vera is most common between ages 50-70. It also occurs more frequently in males than females.

Symptoms may not appear in early stages of PCV. However, when they do, symptoms include:

  • Headache
  • Dizziness
  • Itchiness, particularly after warm water exposure
  • Painful joint swelling, particularly in the big toe
  • Excessive sweating
  • Abdominal pain and bloating
  • Spleen enlargement
  • Double vision
  • Shortness of breath which worsens upon laying down
  • Facial redness
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.

Share this post

Share on facebook
Share on google
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email