Iptacopan Granted Breakthrough Therapy Designation for PNH


Have you ever heard of Breakthrough Therapy designation? This designation is granted by the FDA for therapies designed to treat rare, serious, or life-threatening conditions. In mid-December 2020, PMLive reported that the FDA granted this designation to LNP023 (iptacopan), a treatment option for patients with paroxysmal nocturnal hemoglobinuria (PNH).


Developed by Novartis, iptacopan is an orally administered factor B inhibitor. In addition to receiving Breakthrough Therapy designation, iptacopan also received Rare Pediatric Disease and PRIME designations for the treatment of C3 glomerulopathy, and Orphan Drug designation for IgA nephropathy. Outside of C3G and PNH, iptacopan is being developed for use in patients with atypical hemolytic uremic syndrome and membranous nephropathy.

The FDA defines Breakthrough Therapy designation as:

intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for breakthrough therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.

In this case, the preliminary evidence is sourced from two Phase 2 clinical trials. Within these trials, iptacopan was able to reduce proteinuria, or protein levels in the urine, by just under 50%. It also improved kidney function.

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Resulting from PIGA gene mutations, paroxysmal nocturnal hemoglobinuria (PNH) is a spontaneously acquired hematopoietic stem cell (HSC) disorder. PNH affects red and white blood cells, as well as platelets. The gene mutation causes rapidly multiplying PNH cells. When the immune system attacks the stem cells, healthy cells die while PNH cells survive. PNH affects both men and women equally. It typically affects those between ages 35 and 40. Overall survival rate is usually around 10 years, although patients may survive decades in milder cases. Symptoms include:

  • Easy bruising and bleeding
  • Headache
  • Shortness of breath or difficulty breathing
  • Recurring infections and/or flu-like symptoms
  • Dark and/or bloody urine
  • Fatigue
  • Difficulty swallowing
  • High heart rate
  • Abdominal cramps
  • Chest pain
  • Male sexual dysfunction
  • Kidney disease
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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