Hematology
Paroxysmal nocturnal hemoglobinuria (PNH): the approved drugs compared for prescribers
Empaveli side by side on what each first fill waits on, what the label requires you to monitor and what an insurer asks to see, with FDA decisions, label changes and open trials for paroxysmal nocturnal hemoglobinuria (PNH).
When to suspect paroxysmal nocturnal hemoglobinuria (PNH) and what to test →The paroxysmal nocturnal hemoglobinuria (PNH) prescribing guide
One page per drug with the dispensing gate, prior authorization documentation, dosing, monitoring and interactions, each line sourced.
The drugs side by side
Each cell is a summary of that drug's prescribing guide, which carries the full detail and a source on every line. Insurer thresholds are one public policy shown as an example; plans differ.
| Empavelipegcetacoplan | |
|---|---|
| Label dated | August 21, 2026 |
| Starting dose | 1,080mg (20mL) SC twice weekly, by a commercial infusion pump with a reservoir of at least 20mL or the single-use EMPAVELI Injector (abdomen only). Pump infusion about 30 minutes with 2 sites or 60 minutes with 1; Injector about 30 to 60 minutes. Supplied in 8-vial cartons, a 28-day supply. |
| Dispensing gate | REMS-certified prescriber, pneumococcal and meningococcal (ACWY and B) vaccination at least 2 weeks before day 1 or antibacterial prophylaxis with vaccines as soon as possible, no unresolved encapsulated bacterial infection, counseling with the Patient Guide and Patient Safety Card, and the pharmacy's documentation of vaccination status with the office |
| Required monitoring | Serious infection with encapsulated bacteria at any time; LDH, which sets the dose (every 3 days above 2 times ULN, then LDH twice weekly for 4 weeks); hemolysis for at least 8 weeks after any discontinuation; boosters on the ACIP schedule |
| Example PA threshold | FEP example: PNH, age 18 or older, documented baseline hemoglobin, no dual therapy with another PA drug for PNH, vaccination including Hib, prescriber REMS enrollment; 12 months, 30 vials per 90 days |
| Support line | ApellisAssist, 1-866-692-7527, Start Form by fax to 1-888-754-1285, Apellis Care Educator self-infusion training, QuickStart supply on insurance delay, copay to $0 for commercial plans; REMS line 1-888-343-7073 |
- Label dated
- August 21, 2026
- Starting dose
- 1,080mg (20mL) SC twice weekly, by a commercial infusion pump with a reservoir of at least 20mL or the single-use EMPAVELI Injector (abdomen only). Pump infusion about 30 minutes with 2 sites or 60 minutes with 1; Injector about 30 to 60 minutes. Supplied in 8-vial cartons, a 28-day supply.
- Dispensing gate
- REMS-certified prescriber, pneumococcal and meningococcal (ACWY and B) vaccination at least 2 weeks before day 1 or antibacterial prophylaxis with vaccines as soon as possible, no unresolved encapsulated bacterial infection, counseling with the Patient Guide and Patient Safety Card, and the pharmacy's documentation of vaccination status with the office
- Required monitoring
- Serious infection with encapsulated bacteria at any time; LDH, which sets the dose (every 3 days above 2 times ULN, then LDH twice weekly for 4 weeks); hemolysis for at least 8 weeks after any discontinuation; boosters on the ACIP schedule
- Example PA threshold
- FEP example: PNH, age 18 or older, documented baseline hemoglobin, no dual therapy with another PA drug for PNH, vaccination including Hib, prescriber REMS enrollment; 12 months, 30 vials per 90 days
- Support line
- ApellisAssist, 1-866-692-7527, Start Form by fax to 1-888-754-1285, Apellis Care Educator self-infusion training, QuickStart supply on insurance delay, copay to $0 for commercial plans; REMS line 1-888-343-7073
FDA decisions for paroxysmal nocturnal hemoglobinuria (PNH)
From the Trial Friend FDA decision calendar, which records each date from the sponsor's or the FDA's own announcement.
No pending or recent decisions on the calendar.
Recent label changes
What changed in each label revision, from the FDA's approval letters and the labels' own revision lists.
- August 2026Empaveli. The kidney use for C3G and IC-MPGN was widened to include reducing the loss of kidney function. The label's revision list names only that section, so the PNH use and dose did not change. The REMS materials were updated the same day. FDA label
- July 2025Empaveli. C3 glomerulopathy and primary IC-MPGN were added as a second use, for adults and children 12 and older. FDA REMS page
- February 2024Empaveli. The FDA's safety labeling change removed the Hib vaccine requirement, leaving pneumococcal and meningococcal vaccination. The REMS followed in October 2024. Some insurer policies still list Hib. FDA REMS page
- October 2023Empaveli. Apellis announced FDA approval of the EMPAVELI Injector, a single-use device worn on the belly, as an option to the infusion pump. Apellis release
- May 2021Empaveli. First approval, for adults with PNH. The FDA approved the EMPAVELI REMS on the same day. FDA REMS page
Open trials
Questions prescribers ask about Paroxysmal nocturnal hemoglobinuria (PNH)
Does Empaveli require a REMS?
Yes. Empaveli requires a certified prescriber under an FDA REMS. Its prescribing guide lists the full dispensing gate and its source.
What monitoring does Empaveli require?
Serious infection with encapsulated bacteria at any time; LDH, which sets the dose (every 3 days above 2 times ULN, then LDH twice weekly for 4 weeks); hemolysis for at least 8 weeks after any discontinuation; boosters on the ACIP schedule.
What do insurers require before covering Empaveli?
This is one public insurer policy, an example rather than every plan. FEP example: PNH, age 18 or older, documented baseline hemoglobin, no dual therapy with another PA drug for PNH, vaccination including Hib, prescriber REMS enrollment; 12 months, 30 vials per 90 days.
Which support program starts Empaveli patients on therapy?
ApellisAssist, 1-866-692-7527, Start Form by fax to 1-888-754-1285, Apellis Care Educator self-infusion training, QuickStart supply on insurance delay, copay to $0 for commercial plans; REMS line 1-888-343-7073.
A reference for licensed professionals, checked on November 7, 2026. Labels and plan policies change; the label on DailyMed and the plan's current policy govern. Not medical advice. For patients: the Paroxysmal nocturnal hemoglobinuria (PNH) page and the first 90 days pages for Empaveli.