By Counterforce Health Team —

Understanding Voydeya (Danicopan): A New Option for PNH with Extravascular Hemolysis

What is Voydeya?

Voydeya is a prescription medicine approved for use in adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are still experiencing ongoing red-blood-cell destruction outside the blood vessels (so-called extravascular hemolysis, or EVH) despite receiving standard therapy. 

In simpler terms: for patients with PNH who are already treated with drugs like Soliris (eculizumab) or Ultomiris (ravulizumab) — but who still have signs of hemolysis (especially outside blood vessels) — Voydeya offers an additional treatment option. 

It is not approved as a standalone (“monotherapy”) treatment. It must be used in addition to those other therapies. 

How Does Voydeya Work?

Voydeya contains the active drug danicopan, which works by inhibiting a component of the immune system called complement Factor D. In PNH, the complement system becomes overactive and attacks the body’s own red blood cells. By inhibiting Factor D, Voydeya helps reduce that complement-mediated destruction of red blood cells outside the vessels (extravascular hemolysis). 

So:

  • Standard treatments (Soliris/Ultomiris) mainly target complement component C5 and address intravascular hemolysis (within blood vessels).
  • But for some patients, even with C5 inhibitors, red blood cell destruction still occurs outside the vessels (EVH). Voydeya addresses that gap by targeting Factor D. 

Who Might Benefit from Voydeya?

Voydeya may be appropriate for adult patients who:

  • Have a confirmed diagnosis of PNH.
  • Are already on a C5 inhibitor (e.g., Soliris/Ultomiris) and still have clinically significant signs of extravascular hemolysis (such as low hemoglobin, ongoing anemia, need for transfusions) despite that therapy. 
  • Need additional help reducing symptoms of anemia, fatigue or red-cell destruction outside the vessels.

It is not currently approved for use in children (safety/efficacy not established) or as monotherapy for PNH. 

How to Take Voydeya

  • The usual starting dose is 150 mg three times a day (TID), about every 8 hours. Each dose comprises one 50 mg tablet + one 100 mg tablet. 
  • Depending on response, the dose may be increased to 200 mg three times a day (TID) — e.g., two 100 mg tablets each dose. 
  • It can be taken with or without food. 
  • Important: If a dose is missed and it's within ~3 hours of the next scheduled dose, the missed dose should be skipped. Do not double-up. 
  • If therapy is discontinued, close monitoring for at least 2 weeks is needed for signs of hemolysis. 

Benefits & What to Expect

With Voydeya added to standard C5 inhibitor therapy, potential benefits include:

  • Increase in hemoglobin levels (helping improve anemia).
  • Potential reduction in the need for red-blood-cell transfusions.
  • Improvement in symptoms associated with anemia (e.g., fatigue).
  • Addressing the residual burden of extravascular hemolysis that standard C5 therapy alone may not fully control.

It’s important to note: results vary by individual, and some patients may respond more strongly than others.

Possible Side Effects & Warnings

Common side effects

  • The most common side effect is headache. 
  • Other reported adverse effects include vomiting, fever, elevated liver enzymes, hypertension, pain in arms/legs, increased bilirubin levels.

Important safety warnings

  • Voydeya affects the immune system and increases the risk of serious infections, particularly from encapsulated bacteria (for example, Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae type B). A Boxed Warning applies. 
  • Because of that risk, patients must be vaccinated against these bacteria before starting Voydeya (unless urgent initiation is required) and may require prophylactic antibiotics.
  • Voydeya may increase liver enzyme levels and increase cholesterol/lipid levels (hyperlipidemia). Liver function and lipids should be monitored. 
  • The drug should not be started in patients with an unresolved serious infection caused by encapsulated bacteria. 

Limitations & Things to Know

  • Voydeya is not a cure for PNH — it is a targeted therapy for a specific subset of patients (those with clinically significant extravascular hemolysis despite C5 inhibitor therapy).
  • It has not been shown to be effective as a monotherapy (i.e., cannot replace C5 inhibitor therapy). 
  • Long-term data (beyond 12-weeks) still continue to be collected; individual responses may vary.
  • Access and cost: Because PNH is a rare disease, and because Voydeya is a new therapy with a restricted distribution program (REMS), patients and caregivers will need to work closely with their hematology team, specialty pharmacy, and insurance. 

The Bottom Line

Voydeya (danicopan) represents an important advance in the treatment of PNH for patients experiencing residual extravascular hemolysis despite standard C5 inhibitor therapy. By targeting a different part of the complement pathway (Factor D), it offers another tool to help improve hemoglobin levels, reduce transfusion burden, and manage symptoms of anemia.

However, it requires careful patient selection, monitoring (especially for infections and liver/lipid safety), and continued use in combination with standard therapy. If you or a loved one have PNH and are dealing with ongoing anemia or hemolysis despite Soliris/Ultomiris, it may be worthwhile to discuss Voydeya with your hematologist to determine if it’s a suitable option.