Aspaveli 1080 mg solution for infusion

Spain
Brand name Aspaveli 1080 mg solution for infusion
Form solution for infusion
Active substance / Dosage
PEGCETACOPLAN · 54 mg
Prescription type Hospital Diagnosis
ATC code
L04A L04AJ L04AJ03
Registration number 1211595001
Manufacturer Swedish Orphan Biovitrum Ab (Publ)
Aspaveli 1080 mg solution for infusion solution for infusion

Table of Contents

Package leaflet: Information for the user

Introduction

Package leaflet: information for the user

ASPAVELI 1080mg infusion solution

pegcetacoplan

This medicinal product is subject to additional monitoring, which will allow for faster detection of new safety information. You can help by reporting any adverse reactions you may experience. Section 4, towards the end, includes information on how to report these adverse reactions.

Read the entire leaflet carefully before you start using this medicine, because it contains important information for you.

  • Keep this leaflet, as you may need to read it again.
  • If you have any questions, consult your doctor, pharmacist, or nurse.
  • This medicine has been prescribed for you only, and you should not give it to others, even if they have the same symptoms as you, since it may harm them.
  • If you experience any adverse reactions, consult your doctor, pharmacist, or nurse, even if they are adverse reactions not listed in this leaflet. See section 4.

Contents of the leaflet

  1. What ASPAVELI is and what it is used for
  2. What you need to know before using ASPAVELI
  3. How to use ASPAVELI
  4. Possible adverse reactions
  5. How to store ASPAVELI
  6. Contents of the pack and other information

1. What ASPAVELI is and what it is used for

What ASPAVELI is

ASPAVELI is a medicine that contains the active substance pegcetacoplan. Pegcetacoplan has been designed to bind to complement protein C3, which is part of the body's defense system known as the "complement system".

What ASPAVELI is used for

Paroxysmal nocturnal hemoglobinuria (PNH)

ASPAVELI is used to treat adult patients with a disease called paroxysmal nocturnal hemoglobinuria (PNH) who have anemia due to this condition.

In patients with PNH, the "complement system" is overactive and attacks their red blood cells, which can lead to low blood counts (anemia), fatigue, impaired physical functioning, pain, abdominal pain, dark urine, shortness of breath, difficulty swallowing, erectile dysfunction, and blood clots. By binding to and blocking protein C3, this medicine can prevent the complement system from attacking red blood cells, thereby controlling the disease symptoms. This medicine has been shown to increase red blood cell counts (reduce anemia), which may improve these symptoms.

C3 glomerulopathy (C3G) and primary immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN)

ASPAVELI is used to treat adult and adolescent patients (aged 12 to 17 years) with diseases called complement-mediated C3 glomerulopathy (C3G) or primary immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN). For these conditions, ASPAVELI is used in combination with a renin-angiotensin system inhibitor (RAS inhibitor), unless the use of a RAS inhibitor is considered inappropriate.

Glomerulonephritis is a kidney disease characterized by inflammation in this organ. C3G and primary IC-MPGN are types of glomerulonephritis. In patients with C3G or primary IC-MPGN, the "complement system" is overactive, and when this system is not properly regulated, damage can occur in the glomerulus—a network of tiny blood vessels in the kidney that filters blood. Over time, this impairs the kidneys' ability to remove waste from the blood. If not eliminated, these waste products accumulate in the body and may lead to inflammation, kidney damage, and kidney failure. This can result in blood in the urine (hematuria), excess protein in the urine (proteinuria), reduced kidney function (measured by glomerular filtration rate [GFR]), elevated blood creatinine levels, fatigue, and swelling (edema) of the hands, feet, or ankles. This medicine has been shown to reduce the amount of protein in the urine and may help stabilize kidney function.

2. What you need to know before using ASPAVELI

Do not use ASPAVELI

  • if you are allergic to pegcetacoplan or to any of the other ingredients of this medicine (listed in section 6).
  • if you have an infection caused by so-called encapsulated bacteria.
  • if you are not vaccinated against Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae.

Warnings and precautions

Talk to your doctor, pharmacist, or nurse before starting to use ASPAVELI.

Symptoms of infections

Before starting to use ASPAVELI, inform your doctor if you have any infection.

Since this medicine targets the complement system, which is part of the body's defenses against infections, using this medicine increases the risk of infections, including those caused by so-called encapsulated bacteria such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae. These are serious infections affecting the nose, throat, and lungs or the tissue covering the brain, and they may spread throughout the blood and body.

Talk to your doctor before starting to use ASPAVELI to ensure you receive vaccinations against Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae if you have not previously received them. Even if you have received these vaccines in the past, you may still need additional doses before starting this medicine. These vaccines should be administered at least 2 weeks before starting treatment. If you cannot be vaccinated at least 2 weeks in advance, your doctor will prescribe antibiotics to reduce the risk of infection for 2 weeks after vaccination. After vaccination, your doctor may monitor you more closely for signs of infection.

Symptoms of infection

If you experience any of the following symptoms, you must inform your doctor immediately:

  • headache and fever
  • fever and rash
  • fever with or without chills or shivering
  • shortness of breath
  • rapid pulse
  • sweaty skin
  • headache with neck or back stiffness
  • headache with nausea (feeling sick) or vomiting
  • eyes sensitive to light
  • muscle aches with flu-like symptoms
  • confusion
  • severe pain or discomfort

Make sure your vaccinations are up to date. You should also be aware that vaccines reduce the risk of serious infections but do not prevent all serious infections. According to national recommendations, your doctor may consider additional preventive measures, such as antibacterial medications, to prevent infections.

Allergic reactions

Allergic reactions may occur in some patients. In case of a severe allergic reaction, stop the ASPAVELI infusion and seek immediate medical help. A severe allergic reaction may present as difficulty breathing, chest pain or tightness, and/or dizziness/fainting, intense skin itching or raised bumps on the skin, swelling of the face, lips, tongue, and/or throat, which may cause difficulty swallowing or fainting.

Injection site reactions

Injection site reactions have been observed with the use of ASPAVELI. Before self-administration, you must receive adequate training on the correct injection technique.

Laboratory monitoring for PNH

During treatment with ASPAVELI, your doctor will perform periodic laboratory tests, including blood levels of lactate dehydrogenase (LDH) and kidney function tests, and may adjust your dose if necessary.

Effects on laboratory tests

The use of silica-based reagents in coagulation tests should be avoided, as they may cause an artificial prolongation of activated partial thromboplastin time (aPTT).

Children and adolescents

Do not administer this medicine to children under 18 years of age with PNH, as there is no data on safety and efficacy in this group.

Do not administer this medicine to children under 12 years of age with C3G or primary IC-MPGN, as there is no data on safety and efficacy in this group.

Other medicines and ASPAVELI

Inform your doctor or pharmacist if you are taking, have recently taken, or might need to take any other medicines.

Pregnancy, breastfeeding, and fertility

Women of childbearing potential

The effects of the medicine on the fetus are unknown. Effective contraceptive methods are recommended during treatment and for 8 weeks after treatment in women who could become pregnant. Consult your doctor before using this medicine.

Pregnancy/breastfeeding

ASPAVELI is not recommended during pregnancy and breastfeeding. If you are pregnant or breastfeeding, think you may be pregnant, or plan to become pregnant, consult your doctor before using this medicine.

Driving and using machines

The influence of this medicine on the ability to drive and use machines is negligible or none.

ASPAVELI contains sorbitol

Sorbitol is a source of fructose. If your doctor has told you that you have an intolerance to certain sugars, or if you have been diagnosed with hereditary fructose intolerance (HFI), a rare genetic disorder in which patients cannot break down fructose, consult your doctor before taking this medicine.

ASPAVELI contains sodium

This medicine contains less than 1 mmol of sodium (23 mg) per dose; i.e., essentially "sodium-free".

3. How to use ASPAVELI

Follow exactly the administration instructions for this medicine given by your doctor. If in doubt, consult your doctor again.

At least 2 weeks before starting treatment with this medicine, your doctor will review your medical history and may administer one or more vaccines. If you cannot be vaccinated at least 2 weeks before starting treatment with ASPAVELI, your doctor will prescribe antibiotics for 2 weeks after vaccination to reduce the risk of infection.

If you are using this medicine to treat PNH

Dosage for adults:

The recommended initial dose for adults with PNH is 1,080 mg twice weekly. You should take the dose on two days each week, on Day 1 and Day 4 of each treatment week.

If you are switching from another type of PNH medicine, called a C5 inhibitor, to ASPAVELI, you should take ASPAVELI in addition to your current dose of the C5 inhibitor as prescribed for 4 weeks. After 4 weeks, you should stop taking the C5 inhibitor.

Do not change the dose or dosing interval without consulting your doctor. Your doctor may adjust your dose to 1,080 mg every three days (e.g., Day 1, Day 4, Day 7, Day 10, Day 13, and so on), if appropriate. If you think you have missed a dose, speak to your doctor as soon as possible.

If you are using this medicine to treat C3G or primary IC-MPGN

Dosage for adults:

The recommended initial dose for adults with C3G or primary IC-MPGN is 1,080 mg twice weekly. You should take the dose on two days each week, on Day 1 and Day 4 of each treatment week.

Dosage for adolescents:

The recommended initial dose twice weekly for adolescents with C3G or primary IC-MPGN is based on the patient's body weight. Your doctor will calculate your dose based on the following dosing table.

You should take the dose on two days each week, on Day 1 and Day 4 of each treatment week.

Body weight

First dose

(infusion volume)

Second dose

(infusion volume)

Maintenance dose

(infusion volume)

50 kg or more

1,080 mg twice weekly (20 ml)

35 to less than 50 kg

648 mg (12 ml)

810 mg (15 ml)

810 mg twice weekly (15 ml)

30 to less than 35 kg

540 mg (10 ml)

540 mg (10 ml)

648 mg twice weekly (12 ml)

Form and route of administration

ASPAVELI is intended to be administered as a subcutaneous infusion using:

  • an infusion pump or
  • a body administration system.

The initial doses of the medicine will be administered by a healthcare professional at a clinic or treatment centre. If the treatment is working well, your doctor may discuss with you the possibility of self-administering the medicine at home. If this is appropriate, a healthcare professional will train you or your caregiver on how to administer the infusion.

Infusion rate(s)

When using an infusion pump, the infusion time is approximately 30 minutes if two infusion sites are used, or approximately 60 minutes if only one site is used.

When using the body administration system, the infusion time usually ranges between 30 and 60 minutes (depending on the rate at which the medicine flows into the body).

The infusion must be started without delay after loading the medicine into the syringe and must be completed within 2 hours of syringe preparation.

Instructions for use: preparation of the syringe

Step 1

Prepare for infusion

Before starting:

  1. Remove a carton containing a vial from the refrigerator. Keep the vial in the carton at room temperature and allow it to warm for about 30 minutes.

Do not attempt to speed up the warming process by using a microwave or any other heat source.

  1. Find a flat, well-lit work surface, such as a table.
  2. Gather the supplies:
  1. When using an infusion pump (Figure 1):
  1. Syringe pump system with manufacturer’s instructions (not shown)
  2. Compatible syringe

C1. Transfer needle OR

C2. Needle-free transfer device to withdraw product from the vial

  1. Infusion equipment (not shown; varies according to device manufacturer’s instructions)
  2. Infusion tubing and Y-connector (if needed)
  3. Sharps container
  4. Alcohol wipes
  5. Gauze and tape or transparent dressing

OR

ii) When using a body delivery system (Figure 2):

  1. Body delivery system with manufacturer’s instructions (not shown)
  2. Compatible syringe

C1. Transfer needle OR

BIEN

C2. Needle-free transfer device to withdraw product from the vial

  1. Sharps container
  2. Alcohol wipes

Figure 1 Example of supplies (infusion pump)

Medical diagram with alphabetical labels showing a syringe, a vial, containers, pill blisters, and a cylindrical jar on a gray background

Figure 2 Example of supplies (body delivery system)

Dark container F, syringe B with needle C1, cap C2, and three white sachets G on a light gray background

Thoroughly clean the work surface with an alcohol wipe

Wash hands well with soap and water. Dry hands.

Step 2

Check the vial and solution

Remove the vial from the carton. Carefully examine the liquid in the vial. ASPAVELI is a clear, colorless to slightly yellowish liquid. Check for particles or discoloration (Figure 3).

Do not use the vial if:

  • The solution appears cloudy, contains particles, or is dark yellow.
  • The protective flip-off cap is missing or damaged.
  • The expiration date (EXP) on the label has passed.

Figure 3

A hand holds vertically a small transparent glass vial with a gray cap on a light neutral background

Step 3

Prepare and fill the syringe

Remove the protective flip-off cap from the vial to expose the center of the gray rubber stopper (Figure 4). Discard the cap.

Clean the stopper with a new alcohol wipe and allow it to dry.

Option 1: If using a needle-free transfer device (such as a vial adapter), follow the instructions provided by the device manufacturer.

OR

Option 2: If using a transfer needle and syringe, follow these instructions:

  1. Attach a sterile transfer needle to a sterile syringe.
  2. Pull the plunger back to fill the syringe with air, approximately 20 mL

(Figure 5).

  1. Ensure the vial is upright. DO NOT invert the vial. Insert the air-filled syringe with the attached transfer needle through the center of the vial stopper.
  2. The tip of the transfer needle should not enter the solution to avoid bubble formation (Figure 6).
  3. Gently push the air from the syringe into the vial. This will inject air into the vial.
  4. Invert the vial (Figure 7).
  1. With the transfer needle tip in the solution, slowly pull back the plunger to fill the syringe with the prescribed dose of ASPAVELI (Figure 8).
  2. Ensure the prescribed dose has been withdrawn. Any excess volume must be removed.
  3. Remove the filled syringe with the transfer needle from the vial.
  4. Do not recap the transfer needle. Unscrew the needle and dispose of it in a sharps container.

Figure 4

Two hands hold a small glass vial with a metal cap for drug preparation in a black-and-white drawing

Figure 5

Two hands hold a syringe with the needle pointing downward

Figure 6

A hand holds a glass vial while

Figure 7

Two hands hold a glass vial to aspirate liquid with

Figure 8

Two hands hold a syringe connected to a vial, with a gray arrow indicating downward movement for

For product infusion using a body administration system, follow the manufacturer's instructions for the device. Dispose of all used disposable materials, as well as any unused product and the empty vial, as advised by your healthcare professional.

For product infusion using a syringe infusion pump system, follow the steps outlined below.

Step 4

Prepare the syringe infusion pump system and tubing

Gather the infusion pump components and follow the device manufacturer's instructions to prepare the pump and tubing.

Step 5

Prepare the infusion site(s)

  1. Select an area on the abdominal region (except for a five-centimeter area around the navel), thighs, hips, or arms for infusion (Figure 9).
  2. Use different site(s) from those used for your last infusion. If multiple infusion sites are used, they should be at least 7.5 cm apart. Rotate infusion sites with each infusion (Figure 10).
  3. Avoid the following areas for infusion:
  1. Do not perform infusion in areas where the skin is tender, bruised, red, or hardened.
  2. Avoid tattoos, scars, and stretch marks.
  1. Clean the skin at each infusion site with a new alcohol wipe, starting from the center and moving outward in a circular motion (Figure 11).
  2. Allow the skin to dry.

Figure 9

Human silhouette with gray circles indicating injection sites on the arm, abdomen, hip, and thigh muscle, with corresponding labels

Figure 10

Two adhesive patches applied on the skin with a line indicating a minimum distance of 7.5 cm between the two application sites

Figure 11

A hand holds a cotton swab over

Step 6

Insert and secure the infusion needle(s)

  1. Pinch the skin with your thumb and index finger around the infusion site (where you intend to place the needle). Insert the needle into the skin (Figure 12). Follow the device manufacturer's instructions regarding needle angle.
  2. Secure the needle(s) using sterile gauze and tape or a transparent dressing placed over each infusion site (Figure 13).

Figure 12

Two hands preparing a

Figure 13

Stylized drawing of a human torso with a medical device applied to the lower part of

Step 7

Start the infusion

Follow the device manufacturer's instructions to start the infusion.

Begin the infusion without delay after drawing the solution into the syringe.

Step 8

Complete the infusion

Follow the device manufacturer's instructions to complete the infusion.

Step 9

Record the infusion

Record your treatment as directed by your healthcare professional.

Step

10

Clean up

  1. After the infusion is complete, remove each dressing and slowly withdraw each needle. Cover the infusion site with a new dressing.
  2. Disconnect the infusion set from the pump and dispose of it in a sharps container (Figure 14).
  3. Dispose of all used disposable materials, unused product, and the empty vial as instructed by your healthcare professional.
  4. Clean and store the syringe infusion pump system according to the device manufacturer's instructions.

Figure 14

A hand opens the cap of a dark cylindrical vial with a transparent medical device on a light neutral background

If you forget to use ASPAVELI

If you miss a dose, you should receive it as soon as possible; then take the next dose at the scheduled time.

If you interrupt treatment with ASPAVELI for PNH

PNH is a lifelong disease, so you are expected to use this medicine for a long time. If you wish to stop using the medicine, consult your doctor first. If you stop treatment suddenly, you may be at risk of your symptoms worsening.

If your doctor decides to discontinue treatment with this medicine, follow their instructions on how to stop it. Your doctor will monitor you closely for at least 8 weeks after stopping treatment to detect any signs of red blood cell destruction (hemolysis) due to PNH. Symptoms or problems that may occur due to destruction of red blood cells include:

  • fatigue
  • shortness of breath
  • blood in the urine
  • abdominal pain
  • decreased number of red blood cells
  • blood clots (thrombosis)
  • difficulty swallowing
  • erectile dysfunction in men

Contact your doctor if you experience any of these signs or symptoms.

If you interrupt treatment with ASPAVELI for C3G or primary IC-MPGN

C3G and primary IC-MPGN are lifelong disorders, and therefore, you are expected to use this medicine for a long time. If you wish to stop using the medicine, speak with your doctor first.

4. Possible adverse effects

Like all medicines, this medicine can cause adverse effects, although not everyone will experience them.

Your doctor will discuss the possible adverse effects with you and will explain the risks and benefits of ASPAVELI before starting treatment.

The most serious adverse effect is a serious infection.

If you experience any symptoms of infection (see section 2 “Symptoms of infection”), you must inform your doctor immediately.

If you are unsure about any of the following adverse effects, please ask your doctor to explain them to you.

The following are adverse effects reported in patients with PNH:

Very common (may affect more than 1 in 10 people):

  • Infusion site reactions: these include redness, swelling, itching, bruising, pain, or hardening of the skin. These reactions usually resolve within a few days.
  • Infection of the nose, throat, or respiratory tract (upper respiratory tract infection)
  • Diarrhea
  • Destruction of red blood cells (hemolysis)
  • Stomach pain (abdominal pain)
  • Headache
  • Tiredness (fatigue)
  • Fever or elevated temperature (pyrexia)
  • Cough
  • Urinary tract infection
  • Complications related to mandatory vaccinations
  • Pain in the arms and legs (limb pain)
  • Dizziness
  • Joint pain (arthralgia)
  • Back pain

Common (may affect up to 1 in 10 people):

  • Infection of the ear, mouth, or skin
  • Sore throat
  • Lower number of platelets in the blood (thrombocytopenia), which may cause you to bleed or bruise more easily than normal
  • Nausea (feeling sick)
  • Low levels of potassium in the blood (hypokalemia)
  • Nosebleed (epistaxis)
  • Redness of the skin (erythema)
  • Muscle pain (myalgia)
  • Stomach and intestinal infection, which may cause mild to severe symptoms such as nausea, vomiting, cramps, and diarrhea (gastrointestinal infection)
  • Elevated liver function tests
  • Difficulty breathing (dyspnea)
  • Lower number of white blood cells (neutropenia)
  • Worsening of kidney function
  • Urine of different color
  • High blood pressure
  • Muscle spasms
  • Stuffy nose (nasal congestion)
  • Rash
  • Blood infection (sepsis)
  • Viral infection
  • Fungal infection
  • Respiratory tract infection
  • Eye infection
  • Hives
  • COVID-19
  • Bacterial infection
  • Vaginal infection

Uncommon (may affect up to 1 in 100 people):

  • Inflammation of the cervix
  • Groin infection
  • Pus-filled pocket in the nose (nasal abscess)
  • Pneumonia
  • Tuberculosis
  • Yeast infection of the esophagus
  • Pus-filled pocket around the anus (anal abscess)

The following are adverse effects reported in patients with C3G or primary IC-MPGN:

Very common (may affect more than 1 in 10 people):

  • Infusion site reactions
  • Infection of the nose, throat, or respiratory tract (upper respiratory tract infection)
  • Allergic reaction (including skin rash and eczema)
  • Fever or elevated temperature (pyrexia)
  • Headache
  • Diarrhea
  • Nausea (feeling sick)
  • Worsening of kidney function
  • Influenza (flu)

Common (may affect up to 1 in 10 people):

  • Cough

  • Pneumonia

  • Tiredness (fatigue)

    • Opportunistic infections (including shingles and other infections that occur when the immune system is weakened)

  • Urinary tract infection

  • Ear infection

  • Lower number of platelets in the blood (thrombocytopenia)

  • Muscle pain (myalgia)

  • Nosebleed (epistaxis)

  • Lower number of white blood cells (neutropenia)

    • Low levels of potassium in the blood (hypokalemia)
    • Pain in the arms and legs (limb pain)

Reporting of adverse effects

If you experience any adverse effect, talk to your doctor, pharmacist, or nurse, even if it is a possible adverse effect not listed in this leaflet. You can also report them directly through the national reporting system detailed in Appendix V. By reporting adverse effects, you can help provide more information on the safety of this medicine.

5. Storage of ASPAVELI

  • Keep this medicine out of the sight and reach of children.
  • Do not use this medicine after the expiry date stated on the carton after “EXP”. The expiry date is the last day of the month indicated.
  • Store in a refrigerator (between 2 °C and 8 °C).
  • Keep the vial in the original carton to protect it from light.
  • Medicines should not be disposed of via wastewater. Ask your pharmacist how to dispose of medicines and packaging you no longer need. This helps protect the environment.

6. Contents of the pack and other information

Composition of ASPAVELI

The active substance is pegcetacoplan 1,080 mg (54 mg/ml in a 20 ml vial).

The other components are sorbitol (E 420) (see section 2 “ASPAVELI contains sorbitol”), glacial acetic acid, sodium acetate trihydrate (see section 2 “ASPAVELI contains sodium”), sodium hydroxide (see section 2 “ASPAVELI contains sodium”), and water for injections.

Nature of the product and contents of the container

ASPAVELI is a clear, colourless to slightly yellowish solution for subcutaneous infusion (54 mg/ml in each 20 ml vial). Do not use solutions that are cloudy, contain particles, or show discoloration.

Pack sizes

ASPAVELI is available in a pack containing 1 vial or in a multipack containing 1 x 8 vials.

Please note that the pack does not contain alcohol swabs, needles, or any other supplies or equipment.

Only certain pack sizes may be marketed.

Marketing Authorization Holder

Swedish Orphan Biovitrum AB (publ)

SE-112 76 Stockholm

Sweden

Manufacturer

Swedish Orphan Biovitrum AB (publ)

Norra Stationsgatan 93

113 64 Stockholm

Sweden

Date of the most recent revision of this leaflet: MM/YYYY.

Other sources of information

Detailed information on this medicinal product is available on the European Medicines Agency website: https://www.ema.europa.eu. Links to other websites on rare diseases and orphan medicinal products are also provided.