Alexion data at 2026 AAN Annual Meeting reflects industry-leading portfolio and commitment to enhancing care across rare diseases

PUBLISHED 10 April 2026

New data from PREVAIL Phase III trial will add to the clinical evidence supporting
investigational self-administered C5 inhibitor in adults with gMG

Qualitative sub-study of KOMET Phase III trial and real-world evidence will further
demonstrate impact of KOSELUGO^® on symptoms in adults with NF1-PN, building on
established efficacy profile across age groups

Alexion, AstraZeneca Rare Disease, will deliver 20 presentations, including five oral presentations across generalized myasthenia gravis (gMG), neurofibromatosis type 1 (NF1) plexiform neurofibromas (PN) and neuromyelitis optica spectrum disorder (NMOSD) at the American Academy of Neurology (AAN) Annual Meeting in Chicago, Illinois, April 18 to 22, 2026.

Key presentations will include new results from the PREVAIL Phase III trial evaluating novel dual-binding nanobody gefurulimab in adults with anti-acetylcholine receptor (AChR) antibody-positive (Ab+) gMG as well as a new sub-study from the KOMET Phase III trial evaluating KOSELUGO^® (selumetinib) in adults with NF1 who have symptomatic, inoperable PN. An oral presentation will share new transcriptomics data from the CHAMPION-NMOSD Phase III trial, and additional presentations will highlight real-world evidence, including radiological outcomes, on the approved use of ULTOMIRIS^® (ravulizumab-cwvz) in NMOSD.

Christophe Hotermans, Senior Vice President, Head of Global Medical Affairs, Alexion, said: "At AAN 2026, we will have our broadest set of neurology data to date, with presentations showcasing our rare disease portfolio and pipeline innovation across gMG, NMOSD and NF1-PN. New data from the PREVAIL Phase III trial will reinforce C5 inhibition in gMG, with investigational once-weekly self-administered gefurulimab showing rapid, sustained improvements through 26 weeks and new insights from the KOMET Phase III trial will highlight KOSELUGO's impact in adults with NF1-PN. These findings reflect Alexion's determination to advance innovations that make a meaningful impact for patients."

New data highlights gefurulimab potential as an effective, self-administered treatment option for gMG

An oral presentation from the global PREVAIL Phase III trial will share new results evaluating gefurulimab as a self-administered, once-weekly subcutaneous treatment option for adults with gMG. New data will show statistically significant and clinically meaningful improvement in the secondary endpoint of change in Myasthenia Gravis Composite at Week 26 compared to placebo (treatment difference, -3.1 [0.69]; P<0.0001), expanding on data previously presented at the 2025 Myasthenia Gravis Foundation of America (MGFA) Scientific Session during the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting.

Oral presentations spotlight meaningful symptom improvement with KOSELUGO, including reductions in pain in adult NF1-PN

A qualitative sub-study conducted with participants from KOMET, the largest and only placebo-controlled global Phase III trial in adults with NF1-PN, will show that patients on KOSELUGO reported noticeable decreases in pain, tingling, PN size and improvement in sleep, fatigue and body movements that were sustained or further improved over the course of the trial. Finally, an oral presentation of retrospective US claims data over three years will demonstrate lasting reductions in prescription pain medication utilization among adults with NF1-PN taking KOSELUGO.

Alexion presentations during the 2026 AAN Annual Meeting

Lead Author	Abstract Title	Presentation Details
gMG
Gwathmey, K.	Efficacy and Safety of Subcutaneous Self-Administered Gefurulimab in Generalized Myasthenia Gravis: Primary Results From the Phase 3, Randomized, Double-Blind, Placebo-Controlled PREVAIL Study	Oral Presentation 006 Abstract #1879 April 20, 2026 02:00 PM CT
Juel, V.	MG-ADL Subdomain Score Changes With Eculizumab or Ravulizumab: An Analysis of the Global MG SPOTLIGHT Registry (Encore)	Poster Presentation Abstract #1774 April 20, 2026 08:00 - 09:00 AM CT
Berling, E.	Validation of ME&MGTM Digital Biomarkers in Monitoring Generalized Myasthenia Gravis Symptoms: The DOMYA Study*	Poster Presentation Abstract #2859 April 21, 2026 05:00 - 06:00 PM CT
Barnett-Tapia, C.	ME&MGTM Digital Biomarkers Correlate with Equivalent MG-ADL and MG-QoL15r Sub-items Scores*	Poster Presentation Abstract #2851 April 21, 2026 05:00 - 06:00 PM CT
Yungher, B.	The Phase 4 OCTAGON Study Investigating Oral Corticosteroid Tapering in Adult Patients With Generalized Myasthenia Gravis (gMG) Treated With Ravulizumab: Trial in Progress	Poster Presentation Abstract #2146 April 21, 2026 05:00 - 06:00 PM CT
Nowak, R.	Concomitant Immunosuppressive Therapy Use With Ravulizumab: Analysis of the Global MG SPOTLIGHT Registry (Encore)	Poster Presentation Abstract #1761 April 21, 2026 05:00 - 06:00 PM CT
Shugars, C.	Patient Satisfaction With Ravulizumab Treatment for Generalized Myasthenia Gravis (gMG) in the United States (US)	Poster Presentation Abstract #1868 April 22, 2026 11:45 AM - 12:45 PM CT
Snook, R.	Impact on Healthcare Resource Utilization in Early Initiators of Ravulizumab or Efgartigimod for Treatment of Generalized Myasthenia Gravis in the USA (Encore)	Poster Presentation Abstract #1694 April 22, 2026 11:45 AM - 12:45 PM CT
Mahler, J.	Real-World Biologics in Generalized Myasthenia Gravis: Evidence for Reduced Hospitalization Rates (Encore)	Poster Presentation Abstract #3312 April 22, 2026 11:45 AM - 12:45 PM CT
NF1
Lyons, G.	Effect of Selumetinib Treatment on Long-Term Prescription Pain Medication Utilization in Adults: A Retrospective Study of a US Claims Database	Oral Presentation 006 Abstract #4173 April 20, 2026 02:00 PM CT
Swampillai, A.	Adult Patients' Experiences with Selumetinib Treatment Versus Placebo for Neurofibromatosis Type 1-Plexiform Neurofibroma Associated Symptoms and Their Impacts: A Qualitative Sub-Study of a Phase 3, Placebo-Controlled Trial (KOMET)	Oral Presentation 007 Abstract #3807 April 20, 2026 02:12 PM CT
NMOSD
Pittock, S.	Gene Expression Signatures Associated With Prior Rituximab Treatment in Patients With Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD) in the CHAMPION-NMOSD Trial	Oral Presentation 007 Abstract #2164 April 19, 2026 02:12 PM CT
Sotirchos, E.	Real-World Clinical Outcomes With Eculizumab and Ravulizumab in Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD): Results From the Global NMO SPOTLIGHT Registry (Encore)	Oral Presentation 009 Abstract #3238 April 19, 2026 02:36 PM CT
Wruble, M.	The Predictive Value of Billing Codes and Treatment Data to Identify Relapse Hospitalizations among Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) (Encore)	Poster Presentation Abstract #4999 April 19, 2026 08:00 - 09:00 AM CT
Sato, W.	A real-world study of the effectiveness and safety of ravulizumab in AQP4-Ab+ NMOSD patients with suboptimal response to satralizumab in Japan - interim analysis (Encore)	Poster Presentation Abstract #3426 April 19, 2026 05:00 - 06:00 PM CT
Bernitsas, E.	Financial and Mental Health Burden on Caregivers of Patients With Neuromyelitis Optica Spectrum Disorder (NMOSD) in the United States	Poster Presentation Abstract #2135 April 19, 2026 05:00 - 06:00 PM CT
Okuda, D.	Clinical and Radiological Outcomes in People with Aquaporin-4 Antibody Positive Neuromyelitis Optica Spectrum Disorder Following Ravulizumab Treatment (AMAZE)	Poster Presentation Abstract # 4722 April 20, 2026 08:00 - 09:00 AM CT
Conway, D.	Latent Class Analysis (LCA) of Treatment Preferences Among Adults With Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD) in the United States	Poster Presentation Abstract # 2092 April 20, 2026 05:00 - 06:00 PM CT
Bhattacharyya, S.	Quantifying the Lifetime Health and Societal Benefits From Earlier Diagnosis and Access to Approved Targeted Immunotherapies in Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD) in the United States	Poster Presentation Abstract #2080 April 21, 2026 08:00 - 09:00 AM CT
Pittock, S.	Long-term Efficacy and Safety of Ravulizumab in Anti-aquaporin-4 Antibody-positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD): Final Analysis of the Phase 3 CHAMPION-NMOSD Trial (Encore)	Poster Presentation Abstract #1923 April 21, 2026 08:00 - 09:00 AM CT

*Ad Scientiam research study supported by Alexion

INDICATION & IMPORTANT SAFETY INFORMATION for KOSELUGO® (selumetinib) 10mg & 25mg capsules and 5mg & 7.5mg oral granules

INDICATION

KOSELUGO is indicated for the treatment of adult and pediatric patients 1 year of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Left Ventricular Dysfunction. KOSELUGO can cause cardiomyopathy, defined as a decrease in left ventricular ejection fraction (LVEF) ≥10% below baseline. In the pediatric safety pool, Grade 2 LVEF decrease occurred, as well as decreased LVEF of ≥20% resulting in dose interruption and dose reduction. The median time to first occurrence of LVEF decrease was approximately 12 months. In the adult population, Grade 2 LVEF decrease occurred, with decreased LVEF resulting in dose interruption. The median time to first occurrence of LVEF decrease was approximately 4 months. Assess ejection fraction by echocardiogram prior to initiating treatment, every 3 months during the first year of treatment, every 6 months thereafter, and as clinically indicated. Withhold, reduce dose, or permanently discontinue KOSELUGO based on severity of adverse reaction. In patients who interrupt KOSELUGO for decreased LVEF, obtain an echocardiogram or a cardiac MRI every 3 to 6 weeks until resolution. Upon resolution of decreased LVEF, obtain an echocardiogram or a cardiac MRI every 2 to 3 months.

Ocular Toxicity. KOSELUGO can cause ocular toxicity, including retinal vein occlusion (RVO), retinal pigment epithelial detachment (RPED), and blurred vision. In the pediatric safety pool, blurred vision, photophobia, cataracts, ocular hypertension, and retinal tear occurred. Blurred vision resulted in dose interruption. RPED occurred in the pediatric population during treatment with KOSELUGO and resulted in permanent discontinuation. In the adult population, blurred vision and vitreous floaters occurred in patients receiving KOSELUGO. Conduct ophthalmic assessments prior to initiating KOSELUGO, at regular intervals during treatment, and for new or worsening visual changes. Permanently discontinue KOSELUGO in patients with RVO. Withhold KOSELUGO in patients with RPED, conduct ophthalmic assessments every 3 weeks until resolution, and resume KOSELUGO at a reduced dose.

Gastrointestinal Toxicity. KOSELUGO can cause gastrointestinal toxicities, including diarrhea and colitis. In the pediatric safety pool (N=134), diarrhea occurred in 59% of patients, in addition to diarrhea resulting in permanent discontinuation and dose interruption. In the adult population (N=71), diarrhea occurred in 42% of patients who received KOSELUGO, in addition to diarrhea resulting in dose interruption. The median time to first onset of diarrhea was approximately 2 months in the pediatric safety pool and 1 month in the adult population. Advise patients to start an anti-diarrheal agent (eg, loperamide) and to increase fluid intake immediately after the first episode of diarrhea. Withhold, reduce dose, or permanently discontinue KOSELUGO based on severity of adverse reaction.

Skin Toxicity. KOSELUGO can cause severe rashes, including dermatitis acneiform. In the pediatric safety pool (N=134), rash occurred in 68% of patients. The most frequent rashes included dermatitis acneiform (47%) and maculopapular rash (31%). Pruritus, alopecia, and eczema occurred. In the adult population (N=71), rash occurred in 85% of patients who received KOSELUGO. The most frequent rash included dermatitis acneiform (66%). Alopecia and pruritus occurred in patients who received KOSELUGO. Grade 3 rash and rash resulting in dose interruption and dose reduction occurred in both the pediatric safety pool and the adult population. Permanent discontinuation also occurred in the adult population. Monitor for severe skin rashes. Withhold, reduce dose, or permanently discontinue KOSELUGO based on severity of adverse reaction.

Increased Creatine Phosphokinase (CPK). KOSELUGO can cause increased CPK, myalgia, and rhabdomyolysis. In the pediatric safety pool (N=134), increased CPK, based on laboratory data, occurred in 73% of patients, including Grade 3 or 4. In the adult population (N=71), increased CPK, based on laboratory data, occurred in 70% of patients who received KOSELUGO, including Grade 3 or 4. Increased CPK resulted in dose interruption and dose reduction in both the pediatric safety pool and adult population. Increased CPK concurrent with myalgia occurred in both populations, including one patient who permanently discontinued KOSELUGO for myalgia in the pediatric safety pool. Obtain serum CPK prior to initiating KOSELUGO, periodically during treatment, and as clinically indicated. If increased CPK occurs, evaluate for rhabdomyolysis or other causes. Withhold, reduce dose, or permanently discontinue KOSELUGO based on severity of adverse reaction.

Increased Levels of Vitamin E and Risk of Bleeding (KOSELUGO Capsules). KOSELUGO capsules contain vitamin E, which can inhibit platelet aggregation and antagonize vitamin K-dependent clotting factors. Supplemental vitamin E is not recommended if daily vitamin E intake (including the amount of vitamin E in KOSELUGO and supplement) will exceed the recommended or safe limits due to increased risk of bleeding. An increased risk of bleeding may occur in patients who are co-administered vitamin-K antagonists or anti-platelet antagonists with KOSELUGO capsules. Monitor for bleeding in these patients and increase international normalized ratio (INR) monitoring in patients taking a vitamin-K antagonist. Perform anticoagulant assessments more frequently and adjust the dose of vitamin-K antagonists or anti-platelet agents as appropriate. KOSELUGO oral granules do not contain vitamin E.

Embryo-Fetal Toxicity. KOSELUGO can cause fetal harm when administered during pregnancy. In animal studies, administration of selumetinib to mice during organogenesis caused reduced fetal weight, adverse structural defects, and effects on embryo-fetal survival at approximate exposures >5 times the human exposure at the clinical dose of 25 mg/m² twice daily. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with KOSELUGO and for 1 week after the last dose.

ADVERSE REACTIONS

Common adverse reactions ≥40% in pediatric patients include vomiting, diarrhea, increased CPK, dry skin, paronychia, nausea, dermatitis acneiform, and pyrexia.

Common adverse reactions ≥40% in adult patients include rash (all), dermatitis acneiform, and diarrhea.

DRUG INTERACTIONS

Effect of Other Drugs on KOSELUGO

Concomitant use of KOSELUGO with a strong or moderate CYP3A4 inhibitor or fluconazole increased selumetinib plasma concentrations, which may increase the risk of adverse reactions. Avoid coadministration with KOSELUGO. If coadministration cannot be avoided, reduce KOSELUGO dosage.

Concomitant use of KOSELUGO with a strong or moderate CYP3A4 inducer decreased selumetinib plasma concentrations, which may reduce KOSELUGO efficacy. Avoid concomitant use with KOSELUGO.

SPECIAL POPULATIONS

Pregnancy & Lactation. Verify the pregnancy status of patients of reproductive potential prior to initiating KOSELUGO. Due to the potential for adverse reactions in a breastfed child, advise patients not to breastfeed during treatment with KOSELUGO and for 1 week after the last dose.

To report SUSPECTED ADVERSE REACTIONS, contact AstraZeneca at 1-800-236-9933 or at https://us-aereporting.astrazeneca.com or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full Prescribing Information for KOSELUGO.

INDICATIONS & IMPORTANT SAFETY INFORMATION for ULTOMIRIS^® (ravulizumab-cwvz)

INDICATIONS

Generalized Myasthenia Gravis (gMG)

ULTOMIRIS is indicated for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.

Neuromyelitis Optica Spectrum Disorder (NMOSD)

ULTOMIRIS is indicated for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive.

IMPORTANT SAFETY INFORMATION

WARNING: SERIOUS MENINGOCOCCAL INFECTIONS ULTOMIRIS, a complement inhibitor, increases the risk of serious infections caused by Neisseria meningitidis [see Warnings and Precautions (5.1)] Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update vaccination for meningococcal bacteria (for serogroups A, C, W, Y, and B) at least 2 weeks prior to the first dose of ULTOMIRIS, unless the risks of delaying ULTOMIRIS therapy outweigh the risk of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against meningococcal bacteria in patients receiving a complement inhibitor. See Warnings and Precautions (5.1) for additional guidance on the management of the risk of serious infections caused by meningococcal bacteria. Patients receiving ULTOMIRIS are at increased risk for invasive disease caused by Neisseria meningitidis, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious meningococcal infections and evaluate immediately if infection is suspected. Because of the risk of serious meningococcal infections, ULTOMIRIS is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called ULTOMIRIS and SOLIRIS REMS [see Warnings and Precautions (5.2)].

CONTRAINDICATIONS

• Initiation in patients with unresolved serious Neisseria meningitidis infection.

WARNINGS AND PRECAUTIONS

Serious Meningococcal Infections

ULTOMIRIS, a complement inhibitor, increases a patient's susceptibility to serious, life-threatening, or fatal infections caused by meningococcal bacteria (septicemia and/or meningitis) in any serogroup, including non-groupable strains. Life-threatening and fatal meningococcal infections have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors.

Revaccinate patients in accordance with ACIP recommendations considering the duration of ULTOMIRIS therapy. Note that ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information. If urgent ULTOMIRIS therapy is indicated in a patient who is not up to date with meningococcal vaccines according to ACIP recommendations, provide antibacterial drug prophylaxis and administer meningococcal vaccines as soon as possible. Various durations and regimens of antibacterial drug prophylaxis have been considered, but the optimal durations and drug regimens for prophylaxis and their efficacy have not been studied in unvaccinated or vaccinated patients receiving complement inhibitors, including ULTOMIRIS. The benefits and risks of treatment with ULTOMIRIS, as well as those associated with antibacterial drug prophylaxis in unvaccinated or vaccinated patients, must be considered against the known risks for serious infections caused by Neisseria meningitidis.

Vaccination does not eliminate the risk of serious meningococcal infections, despite development of antibodies following vaccination.

Closely monitor patients for early signs and symptoms of meningococcal infection and evaluate patients immediately if infection is suspected. Inform patients of these signs and symptoms and instruct patients to seek immediate medical care if they occur. Promptly treat known infections. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider interruption of ULTOMIRIS in patients who are undergoing treatment for serious meningococcal infection depending on the risks of interrupting treatment in the disease being treated.

ULTOMIRIS and SOLIRIS REMS

Due to the risk of serious meningococcal infections, ULTOMIRIS is available only through a restricted program called ULTOMIRIS and SOLIRIS REMS.

Prescribers must enroll in the REMS, counsel patients about the risk of serious meningococcal infection, provide patients with the REMS educational materials, assess patient vaccination status for meningococcal vaccines (against serogroups A, C, W, Y, and B) and vaccinate if needed according to current ACIP recommendations two weeks prior to the first dose of ULTOMIRIS. Antibacterial drug prophylaxis must be prescribed if treatment must be started urgently, and the patient is not up to date with both meningococcal vaccines according to current ACIP recommendations at least two weeks prior to the first dose of ULTOMIRIS. Patients must receive counseling about the need to receive meningococcal vaccines and to take antibiotics as directed, signs and symptoms of meningococcal infection, and be instructed to carry the Patient Safety Card at all times during and for 8 months following ULTOMIRIS treatment.

Further information is available at https://www.UltSolREMS.com or 1-888-765-4747.

Other Infections

Serious infections with Neisseria species (other than Neisseria meningitidis), including disseminated gonococcal infections, have been reported.

ULTOMIRIS blocks terminal complement activation; therefore, patients may have increased susceptibility to infections, especially with encapsulated bacteria, such as infections caused by Neisseria meningitidis but also Streptococcus pneumoniae, Haemophilus influenzae, and to a lesser extent, Neisseria gonorrhoeae. Patients receiving ULTOMIRIS are at increased risk for infections due to these organisms, even if they develop antibodies following vaccination.

Thromboembolic Event Management

The effect of withdrawal of anticoagulant therapy during treatment with ULTOMIRIS has not been established. Treatment should not alter anticoagulant management.

Infusion-Related Reactions

Administration of ULTOMIRIS may result in systemic infusion-related reactions, including anaphylaxis and hypersensitivity reactions. In clinical trials, infusion-related reactions occurred in approximately 1 to 7% of patients, including lower back pain, abdominal pain, muscle spasms, drop or elevation in blood pressure, rigors, limb discomfort, drug hypersensitivity (allergic reaction), and dysgeusia (bad taste). These reactions did not require discontinuation of ULTOMIRIS. If signs of cardiovascular instability or respiratory compromise occur, interrupt ULTOMIRIS and institute appropriate supportive measures.

ADVERSE REACTIONS

Adverse Reactions for gMG

Most common adverse reactions in adult patients with gMG (incidence ≥10%) were diarrhea and upper respiratory tract infection. Serious adverse reactions were reported in 20 (23%) of patients treated with ULTOMIRIS and in 14 (16%) patients receiving placebo. The most frequent serious adverse reactions were infections reported in at least 8 (9%) patients treated with ULTOMIRIS and in 4 (4%) patients treated with placebo. Of these infections, one fatal case of COVID-19 pneumonia was identified in a patient treated with ULTOMIRIS and one case of infection led to discontinuation of ULTOMIRIS.

Adverse Reactions for NMOSD

Most common adverse reactions in adult patients with NMOSD (incidence ≥10%) were COVID-19, headache, back pain, arthralgia, and urinary tract infection. Serious adverse reactions were reported in 8 (13.8%) patients with NMOSD receiving ULTOMIRIS.

DRUG INTERACTIONS

Plasma Exchange, Plasmapheresis, and Intravenous Immunoglobulins

Concomitant use of ULTOMIRIS with plasma exchange (PE), plasmapheresis (PP), or intravenous immunoglobulin (IVIg) treatment can reduce serum ravulizumab concentrations and requires a supplemental dose of ULTOMIRIS.

Neonatal Fc Receptor Blockers

Concomitant use of ULTOMIRIS with neonatal Fc receptor (FcRn) blockers (e.g., efgartigimod) may lower systemic exposures and reduce effectiveness of ULTOMIRIS. Closely monitor for reduced effectiveness of ULTOMIRIS.

USE IN SPECIFIC POPULATIONS

Pregnancy Exposure Registry

There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to ULTOMIRIS during pregnancy. Healthcare providers and patients may call 1-833-793-0563 or go to www.UltomirisPregnancyStudy.com to enroll in or to obtain information about the registry.

To report SUSPECTED ADVERSE REACTIONS, contact Alexion Pharmaceuticals, Inc. at 1-844-259-6783 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see accompanying full Prescribing Information for ULTOMIRIS, including Boxed WARNING regarding serious and life-threatening or fatal meningococcal infections.

INDICATIONS & IMPORTANT SAFETY INFORMATION FOR SOLIRIS^® (eculizumab)

INDICATIONS

Generalized Myasthenia Gravis (gMG)

SOLIRIS is indicated for the treatment of generalized myasthenia gravis (gMG) in adult and pediatric patients six years of age and older who are anti-acetylcholine receptor (AChR) antibody positive.

Neuromyelitis Optica Spectrum Disorder (NMOSD)

SOLIRIS is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.

IMPORTANT SAFETY INFORMATION

SERIOUS MENINGOCOCCAL INFECTIONS SOLIRIS, a complement inhibitor, increases the risk of serious infections caused by Neisseria meningitidis [see Warnings and Precautions (5.1)]. Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. • Complete or update vaccination for meningococcal bacteria (for serogroups A, C, W, Y, and B) at least 2 weeks prior to the first dose of SOLIRIS, unless the risks of delaying SOLIRIS therapy outweigh the risk of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against meningococcal bacteria in patients receiving a complement inhibitor. See Warnings and Precautions (5.1) for additional guidance on the management of the risk of serious infections caused by meningococcal bacteria. • Patients receiving SOLIRIS are at increased risk for invasive disease caused by Neisseria meningitidis, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious meningococcal infections and evaluate immediately if infection is suspected. Because of the risk of serious meningococcal infections, SOLIRIS is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called ULTOMIRIS and SOLIRIS REMS [see Warnings and Precautions (5.2)].

CONTRAINDICATIONS

• SOLIRIS is contraindicated for initiation in patients with unresolved serious Neisseria meningitidis infection.

WARNINGS AND PRECAUTIONS

Serious Meningococcal Infections

SOLIRIS, a complement inhibitor, increases a patient's susceptibility to serious, life-threatening, or fatal infections caused by meningococcal bacteria (septicemia and/or meningitis) in any serogroup, including non-groupable strains. Life-threatening and fatal meningococcal infections have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors.

Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with SOLIRIS. Note that ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information.

If urgent SOLIRIS therapy is indicated in a patient who is not up to date with meningococcal vaccines according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer meningococcal vaccines as soon as possible. Various durations and regimens of antibacterial drug prophylaxis have been considered, but the optimal durations and drug regimens for prophylaxis and their efficacy have not been studied in unvaccinated or vaccinated patients receiving complement inhibitors, including SOLIRIS. The benefits and risks of treatment with SOLIRIS, as well as those associated with antibacterial drug prophylaxis in unvaccinated or vaccinated patients, must be considered against the known risks for serious infections caused by Neisseria meningitidis.

Vaccination does not eliminate the risk of serious meningococcal infections, despite development of antibodies following vaccination.

Closely monitor patients for early signs and symptoms of meningococcal infection and evaluate patients immediately if infection is suspected. Inform patients of these signs and symptoms and instruct patients to seek immediate medical care if these signs and symptoms occur. Promptly treat known infections. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider interruption of SOLIRIS in patients who are undergoing treatment for serious meningococcal infection, depending on the risks of interrupting treatment in the disease being treated.

ULTOMIRIS and SOLIRIS REMS

Due to the risk of serious meningococcal infections, SOLIRIS is available only through a restricted program called ULTOMIRIS and SOLIRIS REMS.

Prescribers must enroll in the REMS, counsel patients about the risk of serious meningococcal infection, provide patients with REMS educational materials, assess patient vaccination status for meningococcal vaccines (against serogroups A, C, W, Y, and B) and vaccinate if needed according to current ACIP recommendations two weeks prior to the first dose of SOLIRIS. Antibacterial drug prophylaxis must be prescribed if treatment must be started urgently and the patient is not up to date with both meningococcal vaccines according to current ACIP recommendations at least two weeks prior to the first dose of SOLIRIS. Patients must receive counseling about the need to receive meningococcal vaccines and to take antibiotics as directed, the signs and symptoms of meningococcal infection, and be instructed to carry the Patient Safety Card with them at all times during and for 3 months following SOLIRIS treatment.

Further information is available at https://www.UltSolREMS.com or 1-888-765-4747.

Other Infections

Serious infections with Neisseria species (other than Neisseria meningitidis), including disseminated gonococcal infections, have been reported.

SOLIRIS blocks terminal complement activation; therefore, patients may have increased susceptibility to infections, especially with encapsulated bacteria, such as infections with Neisseria meningitidis but also Streptococcus pneumoniae, Haemophilus influenzae, and to a lesser extent, Neisseria gonorrhoeae. Additionally, Aspergillus infections have occurred in immunocompromised and neutropenic patients. Children treated with SOLIRIS may be at increased risk of developing serious infections due to Streptococcus pneumoniae and Haemophilus influenzae type b (Hib). Administer vaccinations for the prevention of Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) infections according to ACIP recommendations. Patients receiving SOLIRIS are at increased risk for infections due to these organisms, even if they develop antibodies following vaccination.

Thrombosis Prevention and Management

The effect of withdrawal of anticoagulant therapy during SOLIRIS treatment has not been established. Therefore, treatment with SOLIRIS should not alter anticoagulant management.

Infusion-Related Reactions

Administration of SOLIRIS may result in infusion-related reactions, including anaphylaxis or other hypersensitivity reactions. In clinical trials, no patients experienced an infusion-related reaction which required discontinuation of SOLIRIS. Interrupt SOLIRIS infusion and institute appropriate supportive measures if signs of cardiovascular instability or respiratory compromise occur.

ADVERSE REACTIONS

Adverse Reactions for gMG

The most frequently reported adverse reaction in the adult gMG placebo-controlled clinical trial (≥10%) was: musculoskeletal pain.

Adverse Reactions for NMOSD

The most frequently reported adverse reactions in the NMOSD placebo-controlled trial (≥10%) were: upper respiratory infection, nasopharyngitis, diarrhea, back pain, dizziness, influenza, arthralgia, pharyngitis, and contusion.

DRUG INTERACTIONS

Plasmapheresis, Plasma Exchange, Fresh Frozen Plasma Infusion, or IVIg

Concomitant use of SOLIRIS with plasma exchange (PE), plasmapheresis (PP), fresh frozen plasma infusion (PE/PI), or in patients with gMG on concomitant IVIg treatment can reduce serum eculizumab concentrations and requires a supplemental dose of SOLIRIS.

Neonatal Fc Receptor Blockers

Concomitant use of SOLIRIS with neonatal Fc receptor (FcRn) blockers may lower systemic exposures and reduce effectiveness of SOLIRIS. Closely monitor for reduced effectiveness of SOLIRIS.

To report SUSPECTED ADVERSE REACTIONS contact Alexion Pharmaceuticals, Inc. at 1-844-259-6783 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see accompanying full Prescribing Information for SOLIRIS, including Boxed WARNING regarding serious and life-threatening or fatal meningococcal infections.

Notes

KOSELUGO^® (selumetinib)

KOSELUGO^® (selumetinib) is a kinase inhibitor that blocks specific enzymes (MEK1 and MEK2), which are involved in stimulating cells to grow. In NF1, these enzymes are overactive, causing tumor cells to grow in an unregulated way creating so-called plexiform neurofibromas (PN). By blocking these enzymes, KOSELUGO slows down the growth of tumor cells and, therefore, the PN growth.

KOSELUGO is approved in the US, EU, Japan, China and other countries for the treatment of certain pediatric patients with NF1 who have symptomatic, inoperable PN.

KOSELUGO is approved in the US, EU, Japan and other countries for the treatment of adult patients with NF1 who have symptomatic, inoperable PN, and additional regulatory reviews are ongoing.

KOSELUGO has been granted Orphan Drug Designation in the US, EU, Japan and other countries for the treatment of NF1.

ULTOMIRIS^® (ravulizumab-cwvz)

ULTOMIRIS^® (ravulizumab-cwvz), the longest-acting C5 complement inhibitor, provides immediate, complete and sustained complement inhibition. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body's immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. Following a loading dose, ULTOMIRIS is administered intravenously every eight weeks in adults, or every four or eight weeks in pediatric patients (based on body weight).

ULTOMIRIS is approved in the US, EU, Japan and other countries for the treatment of certain adults with paroxysmal nocturnal hemoglobinuria (PNH) and for certain children with PNH in the US and EU.

ULTOMIRIS is also approved in the US, EU, Japan and other countries for the treatment of certain adults and children with atypical hemolytic uremic syndrome (aHUS).

Additionally, ULTOMIRIS is approved in the US, EU, Japan and other countries for the treatment of certain adults with generalized myasthenia gravis (gMG).

Further, ULTOMIRIS is approved in the US, EU, Japan and other countries for the treatment of certain adults with neuromyelitis optica spectrum disorder (NMOSD).

ULTOMIRIS is being assessed as a treatment for additional indications as part of a broad development program.

SOLIRIS^® (eculizumab)

SOLIRIS^® (eculizumab) is a first-in-class C5 complement inhibitor. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body's immune system. When activated in an uncontrolled manner, the terminal complement cascade over-responds, leading the body to attack its own healthy cells. SOLIRIS is administered intravenously every two weeks, following an introductory dosing period.

SOLIRIS is approved in the US, EU, Japan, China and other countries for the treatment of certain children and adults with paroxysmal nocturnal hemoglobinuria (PNH).

SOLIRIS is also approved in the US, EU, Japan, China and other countries for the treatment of certain children and adults with atypical hemolytic uremic syndrome (aHUS).

Additionally, SOLIRIS is approved in the US, EU, Japan, China and other countries for the treatment of certain adults with generalized myasthenia gravis (gMG), and for certain pediatric patients with gMG in the US, EU, Japan and other countries.

Further, SOLIRIS is approved in the US, EU, Japan, China and other countries for the treatment of certain adults with neuromyelitis optica spectrum disorder (NMOSD).

SOLIRIS is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome.

Alexion

Alexion, AstraZeneca Rare Disease, is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more rare disease patients around the world. It is headquartered in Boston, US. For more information, please visit https://www.alexion.us.

AstraZeneca

AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca's innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit astrazeneca-us.com and follow the Company on Social Media @AstraZeneca.

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