Voydeya (danicopan)
An approved treatment for Paroxysmal Nocturnal Hemoglobinuria.
The same compound appears under different names depending on the context. Here is how to identify Danicopan wherever you encounter it, plus the key facts at a glance.
- Generic name
- Danicopan
- Brand name
- Voydeya
- Development codes
- ALXN2040, ACH-4471
- Drug class
- Complement factor D inhibitor
- Manufacturer
- Alexion, AstraZeneca Rare Disease (originally Achillion Pharmaceuticals)
- How it's taken
- The starting dose is 150 mg by mouth 3 times a day, with or without food, at about the same times each day, always together with ongoing ravulizumab or eculizumab.
A pill taken 3 times a day, approved by the FDA on March 29, 2024 as an add-on to ravulizumab (Ultomiris) or eculizumab (Soliris) for extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria. Extravascular hemolysis is red blood cell destruction outside the blood vessels, mainly in the spleen and liver, that can leave some people on a C5 inhibitor anemic. It is not approved for use on its own.
Where Danicopan fits
A first-in-class factor D inhibitor, approved only as an add-on to ravulizumab or eculizumab for people with PNH who still have extravascular hemolysis. AstraZeneca estimates that about 10% to 20% of people with PNH on a C5 inhibitor have clinically significant extravascular hemolysis.
How Danicopan works
C5 inhibitors such as ravulizumab and eculizumab stop PNH red blood cells from being burst open inside blood vessels. The surviving cells can still get coated with fragments of a complement protein called C3, which marks them for removal by the spleen and liver, a process called extravascular hemolysis. Danicopan blocks factor D, an enzyme the alternative complement pathway needs to build the C3 convertase that produces those fragments. In patients on ravulizumab or eculizumab, adding it cut the share of circulating PNH red cells coated with C3 fragments by more than half, while the C5 inhibitor kept control of red cell destruction inside blood vessels[1].
Mechanism: Oral complement factor D inhibitor that blocks the alternative complement pathway, reducing the C3 fragments that tag PNH red blood cells for removal in the spleen and liver
Side effects and safety
- Liver enzyme increases. ALT rose in 14% of patients in the main trial. Liver tests before starting and periodically; treatment may be paused or stopped for significant rises.
- High cholesterol. Raises total and LDL cholesterol. Cholesterol is checked periodically and cholesterol-lowering medicine started if needed.
- Hemolysis after stopping. Close monitoring for at least 2 weeks after the last dose. Keep taking ravulizumab or eculizumab if Voydeya is stopped.
- Drug interactions. Raises levels of some other medicines (BCRP and P-gp substrates). With Voydeya, the rosuvastatin dose should not exceed 10 mg a day.
- Meningococcal and pneumococcal vaccination at least 2 weeks before the first dose
- Liver tests before starting and periodically during treatment
- Cholesterol checks periodically
- Watch for hemolysis for at least 2 weeks after stopping
- Carry the Patient Safety Card during treatment and for 1 week after the last dose
Voydeya carries a boxed warning for serious infections caused by encapsulated bacteria, including Neisseria meningitidis (meningococcus), Streptococcus pneumoniae and Haemophilus influenzae type B. These infections can become life-threatening or fatal quickly, even in vaccinated people. Vaccines against meningococcus and pneumococcus must be completed or updated at least 2 weeks before the first dose; if treatment cannot wait, antibiotics are given and the vaccines are given as soon as possible. Because of this risk, Voydeya is available only through a restricted program called the VOYDEYA REMS, and patients carry a Patient Safety Card during treatment and for 1 week after the last dose. In the main trial, side effects on Voydeya vs placebo (both added to a C5 inhibitor) included headache (11% vs 10%), vomiting (7% vs 0%), fever (7% vs 0%), a rise in the liver enzyme ALT (5% vs 3%), high blood pressure (5% vs 3%) and pain in the arms or legs (5% vs 0%). Overall, 14% of patients on Voydeya had ALT increases, so liver tests are checked before and during treatment. It also raises total and LDL cholesterol, and some patients needed cholesterol-lowering medicine[1].
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Danicopan
The starting dose is 150 mg by mouth 3 times a day, with or without food, at about the same times each day, always together with ongoing ravulizumab or eculizumab. The dose can be raised to 200 mg 3 times a day if hemoglobin has not risen by more than 2 g/dL after 4 weeks, if a transfusion was needed in the previous 4 weeks, or based on the doctor's judgment. A missed dose is taken when remembered unless the next dose is due within 3 hours, in which case it is skipped. Voydeya should be avoided with severe liver impairment (Child-Pugh C)[1].
Availability and cost
Only available as the brand-name product.
Help paying for Voydeya
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
Alexion OneSource CoPay Program: eligible commercially insured patients enrolled in OneSource may pay as little as $0. Not for Medicare (Parts B and D), Medicare Advantage, Medigap, Medicaid, VA, DoD or TRICARE.
For: private insurance · source - Other support
30-day free trial of Voydeya for eligible patients who have not tried it before for extravascular hemolysis.
The official page does not say who qualifies. Ask the program. · source - Insurance and case manager help
Alexion OneSource case management helps with insurance and access questions at 1-888-765-4747, Monday to Friday, 8:30 AM to 8 PM ET. Medicare, Medicaid and other government plan members are referred to third-party resources.
The official page does not say who qualifies. Ask the program. · source
Good to know: The patient page and current copay terms (alexiononesource.com/allpay) do not state an annual maximum; the terms say maximums may vary by patient. An older doctors' page (voydeyahcp.com/support-programs, dated 11/2024) lists up to $15,000 per calendar year, so that figure was left out. No Alexion free-drug program for uninsured patients was found on the pages checked. The same phone number is listed in the label for the VOYDEYA REMS.
- From a charity · NORD RareCarePNH Medical Assistance fundOpen
Pays for: Medical and medication costs.
The foundation says: “Accepting new applications and re-enrollments for current year” - From a charity · NORD RareCarePNH Premium Copay Assistance fundOpen
Pays for: Insurance premiums and copays.
The foundation says: “Accepting new applications and re-enrollments for current year” - From a charity · The Assistance FundParoxysmal Nocturnal Hemoglobinuria (PNH) fundOpen
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
The foundation says: “OPEN — Accepting New Patients. TAF is currently accepting new patient enrollments for this program.” - From a charity · TotalAssist (formerly PAN Foundation)Paroxysmal Nocturnal Hemoglobinuria fundOpen
Pays for: Out-of-pocket costs for approved medications, up to $9,500 per year. Requires health insurance (any kind).
- From a charity · Aplastic Anemia & MDS International FoundationPNH Patient Travel Assistance Fund fundApply directly
Pays for: Travel to a PNH specialist or second opinion (patient plus one companion), up to $800 per year.
The foundation says: “Status not shown on page”
Access and eligibility
Approved for adults with PNH who have extravascular hemolysis while taking ravulizumab or eculizumab. It must be used with one of those drugs, not alone. Safety and effectiveness in children have not been established. Avoid with severe liver impairment (Child-Pugh C). It cannot be started during an unresolved serious infection with encapsulated bacteria. Breastfeeding is not advised during treatment and for 3 days after the last dose.
Source: Alexion OneSource, Voydeya financial assistance
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
ALPHA (NCT04469465) enrolled adults with PNH who had been on a stable dose of ravulizumab or eculizumab for at least 6 months and still had clinically significant extravascular hemolysis, defined as hemoglobin at or below 9.5 g/dL with a high reticulocyte (young red cell) count. They were randomized 2:1 to add Voydeya or placebo for 12 weeks. In the planned interim analysis of 63 patients, hemoglobin rose 2.9 g/dL on Voydeya vs 0.5 g/dL on placebo, 59.5% vs 0% gained at least 2 g/dL without a transfusion, 83.3% vs 38.1% avoided transfusions, and FACIT-Fatigue scores (a 0 to 52 fatigue scale where higher is better) improved 8.0 vs 1.9 points[1][4]. Results were published in The Lancet Haematology in 2023[5].
Development history
Danicopan was developed at Achillion Pharmaceuticals under the code ACH-4471, and Alexion completed its acquisition of Achillion on January 28, 2020[6]. Alexion studied it as ALXN2040[4], and AstraZeneca acquired Alexion in 2021[3]. The FDA approved Voydeya on March 29, 2024[2]. AstraZeneca announced the approval on April 1, 2024, describing Voydeya as a first-in-class oral factor D inhibitor that had received FDA Breakthrough Therapy and Orphan Drug designations[3].
Explore Paroxysmal Nocturnal Hemoglobinuria trials
Other Paroxysmal Nocturnal Hemoglobinuria treatments
PNH is treated with complement inhibitors. The C5 inhibitors eculizumab (Soliris), ravulizumab (Ultomiris) and crovalimab (Piasky, approved June 2024) block red cell destruction inside blood vessels. Pegcetacoplan (Empaveli), which binds C3, and iptacopan (Fabhalta, approved December 2023), a factor B inhibitor pill, act earlier in the complement cascade and target red cell destruction both inside and outside blood vessels. Danicopan (Voydeya, approved March 2024) is an add-on pill for extravascular hemolysis in people who stay on eculizumab or ravulizumab.
Common questions about Danicopan
▸What is Danicopan (Voydeya)?
A pill taken 3 times a day, approved by the FDA on March 29, 2024 as an add-on to ravulizumab (Ultomiris) or eculizumab (Soliris) for extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria. Extravascular hemolysis is red blood cell destruction outside the blood vessels, mainly in the spleen and liver, that can leave some people on a C5 inhibitor anemic. It is not approved for use on its own.
▸How does Danicopan work?
C5 inhibitors such as ravulizumab and eculizumab stop PNH red blood cells from being burst open inside blood vessels. The surviving cells can still get coated with fragments of a complement protein called C3, which marks them for removal by the spleen and liver, a process called extravascular hemolysis. Danicopan blocks factor D, an enzyme the alternative complement pathway needs to build the C3 convertase that produces those fragments. In patients on ravulizumab or eculizumab, adding it cut the share of circulating PNH red cells coated with C3 fragments by more than half, while the C5 inhibitor kept control of red cell destruction inside blood vessels[1].
▸What are the side effects of Danicopan?
Voydeya carries a boxed warning for serious infections caused by encapsulated bacteria, including Neisseria meningitidis (meningococcus), Streptococcus pneumoniae and Haemophilus influenzae type B. These infections can become life-threatening or fatal quickly, even in vaccinated people. Vaccines against meningococcus and pneumococcus must be completed or updated at least 2 weeks before the first dose; if treatment cannot wait, antibiotics are given and the vaccines are given as soon as possible. Because of this risk, Voydeya is available only through a restricted program called the VOYDEYA REMS, and patients carry a Patient Safety Card during treatment and for 1 week after the last dose. In the main trial, side effects on Voydeya vs placebo (both added to a C5 inhibitor) included headache (11% vs 10%), vomiting (7% vs 0%), fever (7% vs 0%), a rise in the liver enzyme ALT (5% vs 3%), high blood pressure (5% vs 3%) and pain in the arms or legs (5% vs 0%). Overall, 14% of patients on Voydeya had ALT increases, so liver tests are checked before and during treatment. It also raises total and LDL cholesterol, and some patients needed cholesterol-lowering medicine[1].
▸How is Danicopan taken?
The starting dose is 150 mg by mouth 3 times a day, with or without food, at about the same times each day, always together with ongoing ravulizumab or eculizumab. The dose can be raised to 200 mg 3 times a day if hemoglobin has not risen by more than 2 g/dL after 4 weeks, if a transfusion was needed in the previous 4 weeks, or based on the doctor's judgment. A missed dose is taken when remembered unless the next dose is due within 3 hours, in which case it is skipped. Voydeya should be avoided with severe liver impairment (Child-Pugh C)[1].
▸Is Danicopan FDA approved?
Yes, Danicopan (Voydeya) is FDA approved (2024) for the treatment of Paroxysmal Nocturnal Hemoglobinuria.
▸Can Voydeya be used alone for PNH?
No. The label limits Voydeya to add-on use with ravulizumab (Ultomiris) or eculizumab (Soliris), because it has not been shown to work as a stand-alone treatment. The C5 inhibitor keeps controlling red cell destruction inside blood vessels, while Voydeya targets extravascular hemolysis. If Voydeya is stopped, the label says to keep the C5 inhibitor going.
▸What is extravascular hemolysis in PNH?
It is the removal of red blood cells outside the blood vessels, mainly by the spleen and liver. On a C5 inhibitor, PNH red cells survive longer but can get coated with complement C3 fragments that mark them for removal, which can leave some people anemic and needing transfusions. AstraZeneca estimates that about 10% to 20% of people with PNH on a C5 inhibitor have clinically significant extravascular hemolysis.
▸What are the side effects of Voydeya?
In the ALPHA trial, side effects on Voydeya vs placebo included headache (11% vs 10%), vomiting (7% vs 0%) and fever (7% vs 0%). ALT, a liver enzyme, rose in 14% of patients, and cholesterol can go up, so both are checked. The label has a boxed warning for serious infections from encapsulated bacteria such as meningococcus, and vaccines are needed before starting.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- U.S. National Library of Medicine, DailyMed. VOYDEYA (danicopan) tablets, for oral use: prescribing information. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=ab8d1cbd-a28b-4d49-82ab-5c7460719ac7
- U.S. Food and Drug Administration · 2024-03-29. NDA 218037 approval letter. https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2024/218037Orig1s000ltr.pdf
- AstraZeneca · 2024-04-01. Voydeya approved in the US as add-on therapy to ravulizumab or eculizumab for treatment of extravascular haemolysis in adults with the rare disease PNH. https://www.astrazeneca.com/media-centre/press-releases/2024/voydeya-approved-in-us.html
- ClinicalTrials.gov. Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) (ALPHA). https://clinicaltrials.gov/study/NCT04469465
- The Lancet Haematology · 2023. Addition of danicopan to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria and clinically significant extravascular haemolysis (ALPHA): a double-blind, randomised, phase 3 trial. https://www.thelancet.com/journals/lanhae/article/PIIS2352-3026(23)00315-0/fulltext
- Alexion Pharmaceuticals (SEC filing) · 2020-01-28. Alexion completes acquisition of Achillion Pharmaceuticals. https://www.sec.gov/Archives/edgar/data/899866/000089986620000005/achillioncloseprfinal2.htm
- U.S. Food and Drug Administration · 2023-12-05. NDA 218276 approval letter (Fabhalta for PNH). https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2023/218276Orig1s000ltr.pdf
- U.S. National Library of Medicine, DailyMed. EMPAVELI (pegcetacoplan) injection: prescribing information. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c23d89e9-b00b-4520-e053-2995a90a95af
- Alexion, AstraZeneca Rare Disease. Voydeya financial assistance. https://alexiononesource.com/voydeya/financial-assistance