On May 12, 2021 Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) reported that five abstracts have been accepted for presentation at the 26th annual European Hematology Association (EHA) (Free EHA Whitepaper) 2021 virtual Congress, taking place from June 9 through June 17, 2021 (Press release, Alexion, MAY 12, 2021, View Source [SID1234579764]). During the meeting, data will be presented further supporting the long-term safety of ULTOMIRIS (ravulizumab-cwvz) over two years, including reduction of thrombotic (blood clot) risk, in complement inhibitor-naïve and complement inhibitor-experienced patients with PNH, and reaffirming the critical role of complete C5 complement inhibition in treating this rare disease.
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Also being presented are new Phase 3 interim analysis data that evaluate the efficacy and safety of ULTOMIRIS in pediatric patients as well as the previously announced topline Phase 3 results for the weekly subcutaneous formulation of ULTOMIRIS in adults with PNH.
"Our data presentations at this year’s EHA (Free EHA Whitepaper) Congress underscore the clinical value of ULTOMIRIS – the standard of care for adults living with PNH – and continue to build on its well-established safety and efficacy profile," said John Orloff, M.D., Executive Vice President and Head of Research and Development at Alexion. "These data also demonstrate further progress in our efforts to continue innovating for patients – supporting the use of ULTOMIRIS in children and adolescents and via a convenient, self-administered subcutaneous formulation."
The U.S. Food and Drug Administration (FDA) granted priority review for ULTOMIRIS in children and adolescents with PNH and has set a Prescription Drug User Fee Act (PDUFA) target action date of June 7, 2021. The interim analysis that will be presented at EHA (Free EHA Whitepaper) is from the largest study of pediatric PNH patients to date.
The accepted abstracts are listed below and are now available on the EHA (Free EHA Whitepaper) website. All e-poster presentations will be made available on the virtual congress platform on June 11, 2021 at 09:00 CEST (3:00 a.m. EDT) and will be available throughout the duration of the Congress.
Oral Presentation
Ravulizumab Reduces the Risk of Thrombosis in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria and High Disease Activity: 2-Year Data from A Phase 3, Open-Label Study. Oral presentation ID #S301. The pre-recorded oral presentation will be published on the virtual Congress platform on June 11, 2021 at 09:00 CEST (3:00 a.m. EDT), with a live Q&A session on June 14, 2021. The session begins at 11:00 CEST (5:00 a.m. EDT).
ePoster Presentations
Pharmacokinetics, Pharmacodynamics, Efficacy and Safety of Ravulizumab In Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria: Interim Analysis of A Phase 3, Open-Label Study. ePoster presentation, abstract ID #EP590.
Safety of Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria: Study 301/302 2-Year Results. ePoster presentation, abstract ID #EP594.
Ravulizumab and Eculizumab Reduce Transfusions in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria: Evidence from Three Real-World Databases: TriNetX US EMR, TriNetX US Claims and KOMODO Health. ePoster presentation, abstract ID #EP1337.
Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients with PNH Previously Treated with Eculizumab: Results from A Phase 3 Randomized, Open-Label Study. ePoster presentation, abstract ID #EP586.
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH is a serious ultra-rare blood disorder with devastating consequences. It is characterized by the destruction of red blood cells, which is also referred to as hemolysis. PNH occurs when the complement system—a part of the body’s immune system—over-responds, leading the body to attack its own red blood cells. PNH often goes unrecognized, with delays in diagnosis from one to more than five years. Patients with PNH may experience a range of symptoms, such as fatigue, difficulty swallowing, shortness of breath, abdominal pain, erectile dysfunction, dark-colored urine and anemia. The most devastating consequence of chronic hemolysis is the formation of blood clots, which can occur in blood vessels throughout the body, damage vital organs, and potentially lead to premature death. The prognosis of PNH can be poor in many cases, so a timely and accurate diagnosis—in addition to appropriate treatment—is critical to improving patient outcomes.
About ULTOMIRIS
ULTOMIRIS (ravulizumab-cwvz) is the first and only long-acting 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 complement cascade over-responds, leading the body to attack its own healthy cells. ULTOMIRIS is administered intravenously every eight weeks or, for pediatric patients less than 20 kg, every four weeks, following a loading dose. ULTOMIRIS is approved in the United States (U.S.), European Union (EU) and Japan as a treatment for adults with paroxysmal nocturnal hemoglobinuria (PNH). It is also approved in the U.S. and Japan for atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) in adult and pediatric (one month of age and older) patients, as well as in the EU for the treatment of adults and children with a body weight of at least 10 kg with aHUS. To learn more about the regulatory status of ULTOMIRIS in the countries that we serve, please visit www.alexion.com.
About SOLIRIS
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. In many countries around the world, SOLIRIS is approved to treat paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), adults with generalized myasthenia gravis (gMG) who are acetylcholine receptor (AchR) antibody positive and/or adults with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive. SOLIRIS is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). To learn more about the regulatory status of SOLIRIS in the countries that we serve, please visit www.alexion.com.
INDICATIONS & IMPORTANT SAFETY INFORMATION for ULTOMIRIS (ravulizumab-cwvz)
INDICATIONS
What is ULTOMIRIS?
ULTOMIRIS is a prescription medicine used to treat:
adults with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH).
adults and children 1 month of age and older with a disease called atypical Hemolytic Uremic Syndrome (aHUS). ULTOMIRIS is not used in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).
It is not known if ULTOMIRIS is safe and effective in children with PNH.
It is not known if ULTOMIRIS is safe and effective in children younger than 1 month of age.
IMPORTANT SAFETY INFORMATION
What is the most important information I should know about ULTOMIRIS?
ULTOMIRIS is a medicine that affects your immune system and can lower the ability of your immune system to fight infections.
ULTOMIRIS increases your chance of getting serious and life-threatening meningococcal infections that may quickly become life-threatening and cause death if not recognized and treated early.
You must receive meningococcal vaccines at least 2 weeks before your first dose of ULTOMIRIS if you are not vaccinated.
If your doctor decided that urgent treatment with ULTOMIRIS is needed, you should receive meningococcal vaccination as soon as possible.
If you have not been vaccinated and ULTOMIRIS therapy must be initiated immediately, you should also receive 2 weeks of antibiotics with your vaccinations.
If you had a meningococcal vaccine in the past, you might need additional vaccination. Your doctor will decide if you need additional vaccination.
Meningococcal vaccines reduce but do not prevent all meningococcal infections. Call your doctor or get emergency medical care right away if you get any of these signs and symptoms of a meningococcal infection: headache with nausea or vomiting, headache and fever, headache with a stiff neck or stiff back, fever, fever and a rash, confusion, muscle aches with flu-like symptoms and eyes sensitive to light.
Your doctor will give you a Patient Safety Card about the risk of meningococcal infection. Carry it with you at all times during treatment and for 8 months after your last ULTOMIRIS dose. It is important to show this card to any doctor or nurse to help them diagnose and treat you quickly.
ULTOMIRIS is only available through a program called the ULTOMIRIS REMS. Before you can receive ULTOMIRIS, your doctor must: enroll in the ULTOMIRIS REMS program; counsel you about the risk of meningococcal infection; give you information and a Patient Safety Card about the symptoms and your risk of meningococcal infection (as discussed above); and make sure that you are vaccinated with a meningococcal vaccine, and if needed, get revaccinated with the meningococcal vaccine. Ask your doctor if you are not sure if you need to be revaccinated.
ULTOMIRIS may also increase the risk of other types of serious infections. Make sure your child receives vaccinations against Streptococcus pneumoniae and Haemophilis influenzae type b (Hib) if treated with ULTOMIRIS. Call your doctor right away if you have any new signs or symptoms of infection.
Who should not receive ULTOMIRIS?
Do not receive ULTOMIRIS if you have a meningococcal infection or have not been vaccinated against meningococcal infection unless your doctor decides that urgent treatment with ULTOMIRIS is needed.
Before you receive ULTOMIRIS, tell your doctor about all of your medical conditions, including if you: have an infection or fever, are pregnant or plan to become pregnant, and are breastfeeding or plan to breastfeed. It is not known if ULTOMIRIS will harm your unborn baby or if it passes into your breast milk. You should not breastfeed during treatment and for 8 months after your final dose of ULTOMIRIS.
Tell your doctor about all the vaccines you receive and medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements which could affect your treatment.
If you have PNH and you stop receiving ULTOMIRIS, your doctor will need to monitor you closely for at least 16 weeks after you stop ULTOMIRIS. Stopping ULTOMIRIS may cause breakdown of your red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: drop in your red blood cell count, tiredness, blood in your urine, stomach-area (abdomen) pain, shortness of breath, blood clots, trouble swallowing, and erectile dysfunction (ED) in males.
If you have aHUS, your doctor will need to monitor you closely for at least 12 months after stopping treatment for signs of worsening aHUS or problems related to a type of abnormal clotting and breakdown of your red blood cells called thrombotic microangiopathy (TMA). Symptoms or problems that can happen with TMA may include: confusion or loss of consciousness, seizures, chest pain (angina), difficulty breathing and blood clots or stroke.
What are the possible side effects of ULTOMIRIS?
ULTOMIRIS can cause serious side effects including infusion-related reactions. Symptoms of an infusion-related reaction with ULTOMIRIS may include lower back pain, pain with the infusion, feeling faint or discomfort in your arms or legs. Tell your doctor or nurse right away if you develop these symptoms, or any other symptoms during your ULTOMIRIS infusion that may mean you are having a serious infusion reaction, including: chest pain, trouble breathing or shortness of breath, swelling of your face, tongue, or throat, and feel faint or pass out.
The most common side effects of ULTOMIRIS in people treated for PNH are upper respiratory infection and headache.
The most common side effects of ULTOMIRIS in people with aHUS are upper respiratory infection, diarrhea, nausea, vomiting, headache, high blood pressure and fever.
Tell your doctor about any side effect that bothers you or that does not go away. These are not all the possible side effects of ULTOMIRIS. For more information, ask your doctor or pharmacist. Call your doctor right away if you miss an ULTOMIRIS infusion or for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
Please see the accompanying full Prescribing Information and Medication Guide for ULTOMIRIS, including Boxed WARNING regarding serious and life-threatening meningococcal infections/sepsis.
INDICATIONS & IMPORTANT SAFETY INFORMATION FOR SOLIRIS (eculizumab)
INDICATIONS
What is SOLIRIS?
SOLIRIS is a prescription medicine used to treat:
patients with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH) .
adults and children with a disease called atypical Hemolytic Uremic Syndrome (aHUS). SOLIRIS is not for use in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).
adults with a disease called generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive.
adults with a disease called neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive.
It is not known if SOLIRIS is safe and effective in children with PNH, gMG, or NMOSD.
IMPORTANT SAFETY INFORMATION
What is the most important information I should know about SOLIRIS?
SOLIRIS is a medicine that affects your immune system and can lower the ability of your immune system to fight infections.
SOLIRIS increases your chance of getting serious and life-threatening meningococcal infections that may quickly become life-threatening and cause death if not recognized and treated early.
You must receive meningococcal vaccines at least 2 weeks before your first dose of SOLIRIS if you are not vaccinated.
If your doctor decided that urgent treatment with SOLIRIS is needed, you should receive meningococcal vaccination as soon as possible.
If you have not been vaccinated and SOLIRIS therapy must be initiated immediately, you should also receive two weeks of antibiotics with your vaccinations.
If you had a meningococcal vaccine in the past, you might need additional vaccination. Your doctor will decide if you need additional vaccination.
Meningococcal vaccines reduce but do not prevent all meningococcal infections. Call your doctor or get emergency medical care right away if you get any of these signs and symptoms of a meningococcal infection: headache with nausea or vomiting, headache and fever, headache with a stiff neck or stiff back, fever, fever and a rash, confusion, muscle aches with flu-like symptoms, and eyes sensitive to light.
Your doctor will give you a Patient Safety Card about the risk of meningococcal infection. Carry it with you at all times during treatment and for 3 months after your last SOLIRIS dose. It is important to show this card to any doctor or nurse to help them diagnose and treat you quickly.
SOLIRIS is only available through a program called the SOLIRIS REMS. Before you can receive SOLIRIS, your doctor must enroll in the SOLIRIS REMS program; counsel you about the risk of meningococcal infection; give you information and a Patient Safety Card about the symptoms and your risk of meningococcal infection (as discussed above); and make sure that you are vaccinated with the meningococcal vaccine and, if needed, get revaccinated with the meningococcal vaccine. Ask your doctor if you are not sure if you need to be revaccinated.
SOLIRIS may also increase the risk of other types of serious infections. Make sure your child receives vaccinations against Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) if treated with SOLIRIS. Certain people may be at risk of serious infections with gonorrhea. Certain fungal infections (Aspergillus) may occur if you take SOLIRIS and have a weak immune system or a low white blood cell count.
Who should not receive SOLIRIS?
Do not receive SOLIRIS if you have a meningococcal infection or have not been vaccinated against meningitis infection unless your doctor decides that urgent treatment with SOLIRIS is needed.
Before you receive SOLIRIS, tell your doctor about all of your medical conditions, including if you: have an infection or fever, are pregnant or plan to become pregnant, and are breastfeeding or plan to breastfeed. It is not known if SOLIRIS will harm your unborn baby or if it passes into your breast milk.
Tell your doctor about all the vaccines you receive and medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements which could affect your treatment. It is important that you have all recommended vaccinations before you start SOLIRIS, receive 2 weeks of antibiotics if you immediately start SOLIRIS, and stay up-to-date with all recommended vaccinations during treatment with SOLIRIS.
If you have PNH, your doctor will need to monitor you closely for at least 8 weeks after stopping SOLIRIS. Stopping treatment with SOLIRIS may cause breakdown of your red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: drop in the number of your red blood cell count, drop in your platelet count, confusion, kidney problems, blood clots, difficulty breathing, and chest pain.
If you have aHUS, your doctor will need to monitor you closely during and for at least 12 weeks after stopping treatment for signs of worsening aHUS symptoms or problems related to abnormal clotting (thrombotic microangiopathy). Symptoms or problems that can happen with abnormal clotting may include: stroke, confusion, seizure, chest pain (angina), difficulty breathing, kidney problems, swelling in arms or legs, and a drop in your platelet count.
What are the possible side effects of SOLIRIS?
SOLIRIS can cause serious side effects including serious infusion-related reactions. Tell your doctor or nurse right away if you get any of these symptoms during your SOLIRIS infusion: chest pain, trouble breathing or shortness of breath, swelling of your face, tongue, or throat, and feel faint or pass out. If you have an infusion-related reaction to SOLIRIS, your doctor may need to infuse SOLIRIS more slowly, or stop SOLIRIS.
The most common side effects in people with PNH treated with SOLIRIS include: headache, pain or swelling of your nose or throat (nasopharyngitis), back pain, and nausea.
The most common side effects in people with aHUS treated with SOLIRIS include: headache, diarrhea, high blood pressure (hypertension), common cold (upper respiratory infection), stomach-area (abdominal) pain, vomiting, pain or swelling of your nose or throat (nasopharyngitis), low red blood cell count (anemia), cough, swelling of legs or feet (peripheral edema), nausea, urinary tract infections, and fever.
The most common side effects in people with gMG treated with SOLIRIS include: muscle and joint (musculoskeletal) pain.
The most common side effects in people with NMOSD treated with SOLIRIS include: common cold (upper respiratory infection), pain or swelling of your nose or throat (nasopharyngitis), diarrhea, back pain, dizziness, flu like symptoms (influenza) including fever, headache, tiredness, cough, sore throat, and body aches, joint pain (arthralgia), throat irritation (pharyngitis), and bruising (contusion).
Tell your doctor about any side effect that bothers you or that does not go away. These are not all the possible side effects of SOLIRIS. For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit MedWatch, or call 1-800-FDA-1088.
Please see the accompanying full Prescribing Information and Medication Guide for SOLIRIS, including Boxed WARNING regarding serious and life-threatening meningococcal infections.