ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

The study consisted of a 4-week screening period and a 26-week randomized treatment period
(Primary Evaluation Period). After completion of the 26-week Primary Evaluation Period, all
participants had the opportunity to enter the Extension Period, wherein participants will
receive ravulizumab for up to 2 years.

This study is ongoing. The data presented is for the Primary Evaluation Period. The results
for the Extension Period will be reported after study completion.

Inclusion Criteria:

1. Male or female ≥18 years of age.

2. PNH diagnosis confirmed by documented by high-sensitivity flow cytometry.

3. Presence of 1 or more of the following PNH-related signs or symptoms within 3 months
of screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea),
anemia (hemoglobin <10 gram/deciliter), history of a major adverse vascular event
(including thrombosis), dysphagia, or erectile dysfunction; or history of packed red
blood cells (pRBC) transfusion due to PNH.

4. Lactate dehydrogenase (LDH) level ≥1.5 times the upper limit of normal at screening.

5. Documented meningococcal vaccination not more than 3 years prior to, or at the time
of, initiating study treatment.

6. Female participants of childbearing potential must use highly effective contraception
starting at screening and continuing until at least 8 months after the last dose of
ravulizumab.

7. Willing and able to give written informed consent and comply with study visit
schedule.

Exclusion Criteria:

1. Treatment with a complement inhibitor at any time.

2. History of bone marrow transplantation.

3. Body weight <40 kg.

4. Females who are pregnant, breastfeeding, or who have a positive pregnancy test at
screening or Day 1.

5. Participation in another interventional clinical study or use of any experimental
therapy within 30 days before initiation of study drug on Day 1 in this study or
within 5 half-lives of that investigational product, whichever is greater.

6. History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease
that, in the opinion of the investigator or sponsor, would preclude participation.

7. Unstable medical conditions (for example, myocardial ischemia, active gastrointestinal
bleed, severe congestive heart failure, anticipated need for major surgery within 6
months of randomization, coexisting chronic anemia unrelated to PNH).