- Global study to enroll approximately 270 adults with COVID-19 and severe pneumonia or acute respiratory distress syndrome -
- Company maintains commitment to supplying its medicines to patients for currently approved indications -
“Alexion has been in close contact with physicians and global health authorities in an effort to rapidly evaluate the potential of C5 inhibition in treating patients with severe COVID-19,” said
The decision to begin this trial is based on a) published preclinical data suggesting that inhibition of terminal complement can lower cytokine and chemokine levels and significantly reduce lung inflammation and pathology in animal models of viral pneumoniai, and b) elevated complement biomarkers and promising preliminary clinical evidence from patients who have accessed SOLIRIS® (eculizumab) through our compassionate use program, which suggests that complement inhibition may improve coronaviral-mediated lung injury.
Independent investigators have expressed interest in studying the potential of C5 inhibition in severe COVID-19 pneumonia, and we are aware of several ongoing or planned independent studies and anecdotal results from the use of our C5 inhibitors in patients with COVID-19. While these healthcare professionals continue to aggregate data regarding the potential of terminal complement inhibition in COVID-19 pneumonia from the approximately 100 patients who have been treated so far,
For additional information on Alexion’s ongoing efforts related to COVID-19, please visit: https://alexion.com/our-commitment/covid-19.
About the Phase 3 Study
The Phase 3 open-label, randomized, controlled study is designed to evaluate the safety and efficacy of ULTOMIRIS in approximately 270 adults hospitalized with COVID-19 and severe pneumonia, acute lung injury or acute respiratory distress syndrome (ARDS). Study participants will be randomized 2:1 to receive ULTOMIRIS or best supportive care. The primary endpoint is survival at Day 29. Secondary endpoints will assess the need for mechanical ventilation, oxygenation, duration of ICU stay and hospitalization, and safety, among others.
Patients in the ULTOMIRIS arm will receive a weight-based loading dose of ULTOMIRIS on Day 1 (2400mg for patients weighing 40-60kg, 2700mg for 60-100kg, or 3000mg for ≥100kg). Follow-up dosing on Days 5 and 10 will also be weight-based; patients weighing 40 to 60kg will receive 600mg of ULTOMIRIS and patients weighing 60kg or more will receive 900mg of ULTOMIRIS. On Day 15, all patients will receive 900mg of ULTOMIRIS. All patients will continue to receive medications, therapies, and interventions per standard hospital treatment protocols for the duration of the study. Following the 4-week treatment period, there will be safety follow-up monitoring for three months.
Expanded Access Programs
In recognition of the urgent needs of some patients and in order to streamline the emergency access process,
Access & Supply Considerations
Alexion’s focus has always been on developing transformative medicines for patients with rare and ultra-rare diseases that typically affect several hundred to a few thousand patients worldwide. Like all of our medicines, ULTOMIRIS is a biologic medicine, which are very large complex molecules made up of genetically engineered proteins that are manufactured in living cells through a highly complicated process that requires significant time, expertise and precision. During this global health crisis, we have taken proactive measures that are designed to mitigate the risk of potential supply interruptions, and we strive to maintain sufficient inventory levels to continue serving current and new patients receiving our medicines for approved rare and ultra-rare indications as well as those participating in ongoing clinical trials.
We recognize that, should the role of C5 in treating severe respiratory complications of COVID-19 be demonstrated in a controlled clinical trial, there is the potential for significantly increased demand for our C5 inhibitors. We have taken steps to significantly increase future supply as part of our efforts to prepare for this and other potential scenarios that may arise so that we are ready to support access and the anticipated increased supply demand. In the meantime,
About ULTOMIRIS® (ravulizumab-cwvz)
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. For currently approved indications, ULTOMIRIS is administered intravenously every eight weeks or every four weeks for pediatric patients less than 20 kg, following a loading dose. ULTOMIRIS is approved in
INDICATIONS & IMPORTANT SAFETY INFORMATION FOR ULTOMIRIS® (ravulizumab-cwvz) AND SOLIRIS® (eculizumab)
ULTOMIRIS and SOLIRIS are prescription medicines called monoclonal antibodies. ULTOMIRIS and SOLIRIS are used to treat adults with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). ULTOMIRIS and SOLIRIS are also used to treat adults and children with a disease called atypical Hemolytic Uremic Syndrome (aHUS). Neither ULTOMIRIS nor SOLIRIS is for use in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).
In addition, SOLIRIS is used to treat adults with a disease called generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. SOLIRIS is also used to treat adults with a disease called neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive.
It is not known if ULTOMIRIS is safe and effective in children with PNH or in children younger than one month of age in aHUS. It is also not known if SOLIRIS is safe and effective in children with PNH, gMG, or NMOSD.
IMPORTANT SAFETY INFORMATION
ULTOMIRIS and SOLIRIS are medicines that affect the immune system. These medicines can lower the ability of the immune system to fight infections. Both medicines increase the chance of getting serious and life-threatening meningococcal infections. Meningococcal infections may quickly become life-threatening and cause death if not recognized and treated early.
Meningococcal vaccines must be received at least two weeks before the first dose of ULTOMIRIS or SOLIRIS if the patient has not already had this vaccine. If the patient’s doctor decided that urgent treatment is needed, meningococcal vaccination should be administered as soon as possible. If the patient has not been vaccinated and therapy must be initiated immediately, two weeks of antibiotics should also be administered with the vaccinations. If the patient had a meningococcal vaccine in the past, additional vaccination might be needed before starting ULTOMIRIS or SOLIRIS. Patients should ask their doctor if an additional meningococcal vaccination is needed. Meningococcal vaccines reduce the risk of meningococcal infection but do not prevent all meningococcal infections. Patients should be instructed to call their doctor or get emergency medical care right away if any of these signs and symptoms of a meningococcal infection occur: 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. The doctor will provide a Patient Safety Card about the risk of meningococcal infection. This card must be carried at all times during treatment and for 8 months after the last ULTOMORIS dose or 3 months after the last SOLIRIS dose.
Before a patient can receive ULTOMIRIS or SOLIRIS, their doctor must: enroll in the corresponding ULTOMIRIS REMS or SOLIRIS REMS program; counsel the patient about the risk of meningococcal infection; give the patient information and a Patient Safety Card about the symptoms and risk of meningococcal infection (as discussed above); and make sure that the patient is vaccinated with a meningococcal vaccine.
ULTOMIRIS and SOLIRIS may also increase the risk of other types of serious infections. Patients should talk to their doctor right away if they have any new signs or symptoms of infection.
Patients must not receive ULTOMIRIS or SOLIRIS if they have a meningococcal infection, or if they have not been vaccinated against meningococcal infection, unless their doctor decides that urgent treatment is needed.
Before a patient receives ULTOMIRIS or SOLIRIS, they should tell their doctor about all of their medical conditions, including if they: 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 or SOLIRIS will harm an unborn baby or if these medicines pass into the breast milk.
Patients should tell their doctor about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. ULTOMIRIS and SOLIRIS can affect how other medicines work, causing side effects.
For patients with PNH, the doctor will need to monitor the patient closely for at least 16 weeks after stopping ULTOMIRIS or 8 weeks after stopping SOLIRIS. Stopping treatment with these medicines may cause breakdown of the red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: a drop in red blood cell count, tiredness, blood in the urine, stomach-area (abdomen) pain, shortness of breath, blood clots, trouble swallowing, and erectile dysfunction (ED) in males.
For patients with aHUS, the doctor will need to monitor closely during and for at least 12 months after stopping ULTOMIRIS, or 12 weeks after stopping SOLIRIS, for signs of worsening aHUS symptoms or problems related to abnormal clotting and breakdown of 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.
ULTOMIRIS can cause serious side effects including infusion reactions. Symptoms of an infusion reaction with ULTOMIRIS may include lower back pain, pain with the infusion, feeling faint or discomfort in the arms or legs. Patients should tell their doctor or nurse right away if they develop these symptoms, or any other symptoms during their ULTOMIRIS infusion that may mean they are having a serious infusion reaction, including: chest pain, trouble breathing or shortness of breath, swelling of the 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.
SOLIRIS can cause serious side effects including serious allergic reactions. Serious allergic reactions can happen during the SOLIRIS infusion. Patients should tell their doctor or nurse right away if they get any of these symptoms during the SOLIRIS infusion: chest pain, trouble breathing or shortness of breath, swelling of the face, tongue, or throat, and feeling faint or pass out. If a patient has an allergic reaction to SOLIRIS, the 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 the 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 the 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 the 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 ULTOMIRIS or SOLIRIS. For more information, ask your doctor or pharmacist. Call your doctor right away if you miss an ULTOMIRIS or SOLIRIS infusion or for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
Please refer to the full
ULTOMIRIS Full Prescribing Information and Medication Guide
SOLIRIS Full Prescribing Information and Medication Guide
This press release contains forward-looking statements that involve risks and uncertainties relating to future events and the future performance of
i Gralinski LE, Sheahan TP, Morrison TE, et al. Complement activation contributes to severe acute respiratory syndrome coronavirus pathogenesis. mBio. 2018;9(5). doi:10.1128/mBio.01753-18.
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