CASGEVY 4 - 13 × 10⁶ cells/mL dispersion for infusion

Introduction

Package Leaflet: Information for the Patientor Caregiver

Casgevy4‑13×106cells/ml dispersion for infusion

exagamglogén autotemcel (CD34+ cells)

This medicinal product is subject to additional monitoring, which will allow for quicker identification of new safety information. You can help by reporting any side effects you may get. The last section of the package leaflet contains information on how to report side effects.

Read all of this leaflet carefully before you are giventhis medicine, because it contains important information for you.

• Keep this leaflet, you may need to read it again.
• If you have any further questions, ask your doctor or nurse.
• If you get any side effects, talk to your doctor or nurse. This includes any possible side effects not listed in this leaflet. See section 4.

Contents of the pack

1. What is Casgevy and what is it used for
2. What you need to know before you are given Casgevy
3. How Casgevy is prepared and administered
4. Possible side effects
5. Storage of Casgevy
6. Contents of the pack and further information

1. What is Casgevy and what is it used for

What is Casgevy

Casgevy is a gene therapy medicine that contains the active substance exagamglogén autotemcel.

Casgevy is specifically prepared for you using your own blood stem cells. Blood stem cells can become other blood cells, such as red blood cells, white blood cells, and platelets. These cells are taken from your blood, then genetically modified, and given back to you as a transplant in a hospital.

What Casgevy is used for

Casgevy is used to treat:

• People aged 12 years and olderwith beta-thalassemia who need regular blood transfusions (transfusion-dependent thalassemia, TDT). People with TDT do not make enough haemoglobin, a blood protein that carries oxygen around the body, due to a defect in a gene. This causes anaemia and they need regular blood transfusions.
• People aged 12 years and olderwith sickle cell anaemia (SCA) who have frequent painful crises (called vaso-occlusive crises or VOC). Patients with SCA have a different form of haemoglobin (sickle haemoglobin or HbS) than other people due to a defect in a gene. HbS leads to abnormal red blood cells that are sickle-shaped and stick together, which can block blood vessels and cause VOC.

How Casgevy works

Casgevy works by increasing the production of a special type of haemoglobin called fetal haemoglobin (HbF). Having more HbF improves the production and function of red blood cells. Therefore, people with TDT may not need blood transfusions and people with sickle cell anaemia may not have VOC.

2. What you need to know before you are given Casgevy

You must not be given Casgevy

• exagamglogén autotemcel or any of the other components of this medicine (listed in section 6);
• any of the components in the medicines that will be given to you to prepare you for treatment with Casgevy (see section 3).

If any of these apply to you, or if you are unsure, tell your doctor immediately. You will not be given the treatment if you are allergic to any of these medicines.

Warnings and precautions

Talk to your doctor or nurse before you start treatment with Casgevy.

Before treatment with Casgevy:

• You will be given two other types of medicinesbefore you are given Casgevy. For more information on these medicines, see section 3.

• Mobilisation medicineto move the blood stem cells from the bone marrow into the bloodstream, so they can be collected to make Casgevy. This step takes 2 to 6 days.
• You will be given a conditioning medicineshortly before you are given Casgevy. This creates space in the bone marrow for new blood cells to grow after treatment with Casgevy.

• Your doctor will discuss the possible impact of the conditioning medicine on fertility. See “Fertility in men and women” below.
• In people with SCA, it may be more difficult to move the blood stem cells from the bone marrow and collect them, compared to people with TDT. Therefore, in people with SCA, more mobilisations and collections may be needed than in people with TDT.

After treatment with Casgevy:

• You will have fewer blood cells for a while, until Casgevy settles in your bone marrow. This includes:

• Low levels of platelets (cells that help blood to clot). Low platelet levels can cause bleeding.
• Tell your doctor immediatelyif you have any of these signs of low platelet levels: severe headache, unusual bruising, prolonged bleeding, or bleeding without injury, such as nosebleeds, bleeding gums, blood in urine, stools, or vomit, or coughing up blood.
• Low levels of neutrophils (a type of white blood cell that normally prevents infections). Low neutrophil levels can make it more likely that you will get infections.

Tell your doctor immediatelyif you have any of these signs of low white blood cell levels: fever, chills, or other signs of infection, such as sore throat, cough, or shortness of breath, pain or burning when urinating, or frequent urination, or diarrhoea.

• Your doctor will monitor your blood cell levels and give you any necessary treatment. The doctor will tell you when your platelets and neutrophils are back to safe levels.

• Your doctor will monitor your blood cell levels and overall health to help researchers learn about the long-term effects of Casgevy.

• In some patients, haemoglobin levels may be lower than normally expected for their age and sex.

• After treatment with Casgevy, there is a theoretical risk of blood cancers (myelodysplasia, leukaemia, or lymphoma), although this has not been seen in studies with Casgevy. Your doctor will follow you up at least once a year for 15 years to check for any signs of blood cancers.

• Components of Casgevy called dimethyl sulfoxide (DMSO), dextran 40, and Cas9 can cause severe allergic reactions. Your doctor or nurse will monitor you for signs and symptoms of an allergic reaction, both during and after treatment with Casgevy. See section 2 “Casgevy contains sodium and dimethyl sulfoxide (DMSO)”.

• Casgevy is tested for infectious microbes, but there is still a small risk of infection. Your doctor or nurse will monitor you for signs and symptoms of infections and give you any necessary treatment.

• After treatment with Casgevy, you must not donateblood, organs, tissues, or cells.

• Casgevy is made from your own cells and is only given to you. Information about cell-based medicines must be kept for 30 years in the hospital where you receive treatment. The information they keep will include your name, the product name, and the batch numbers of Casgevy that you have received.

If treatment with Casgevy cannot be completed or fails

If Casgevy cannot be given after the conditioning medicine, or if the modified blood stem cells do not settle in your body, your doctor may decide to give you an injection into a vein that contains your rescue cells (your own original, unmodified blood stem cells) that were collected and stored before you started treatment (see section 3). If you are given the rescue cells, you will not get any benefit from the treatment and will still need treatment for TDT or SCA.

Children under 12 years

Casgevy must not be given to children under 12 years. It is not known if Casgevy is safe and effective in these children.

Other medicines and Casgevy

Tell your doctor or nurse if you are taking, have recently taken, or might take any other medicines.

Do not take medicines that remove iron from your body(iron chelators like deferoxamine, deferiprone, or deferasirox) for at least 7 days before you are given the conditioning medicine. Your doctor will tell you if you can start taking these medicines after treatment with Casgevy and when to take them.

Do not take other medicines for sickle cell anaemia(such as hydroxyurea/hydroxycarbamide, crizanlizumab, or voxelotor) for at least 8 weeks before you are given the mobilisation and conditioning medicines. Your doctor will tell you if you should start taking these medicines after treatment with Casgevy and when to take them.

Live vaccinesmust not be given in the 6 weeks before the conditioning medicine used to prepare you for treatment with Casgevy, or after treatment while your immune system (the body’s defence system) is recovering. Talk to your doctor if you need to have any vaccinations.

Pregnancy

This treatment must not be given during pregnancydue to the possible effects of the conditioning medicine. The effects of Casgevy in pregnant women are not known. Talk to your doctor about the possibility of becoming pregnant after receiving Casgevy.

If you are pregnant or think you may be pregnant after treatment with Casgevy, tell your doctor immediately.

If you are a woman who can become pregnant, a pregnancy test will be performedbefore you start the mobilisation and conditioning medicines to check that you are not pregnant.

Contraception in men and women

If you are a woman who can become pregnant, or a man who can father a child, you must use an effective method of contraceptionfrom the start of mobilisation treatment and for at least 6 monthsafter receiving Casgevy. Talk to your doctor about suitable methods of contraception.

Breast-feeding

Breast-feeding must be stopped during conditioningdue to the possible effects of the conditioning medicine. It is not known if the components of Casgevy can pass into breast milk. Your doctor will discuss the benefits of breast-feeding for your baby against the possible risks of the treatment.

Fertility in men and women

You may not be able to become pregnant or father a child after receiving the conditioning medicine. You must discuss your options with your doctor before treatment.These may include storing reproductive material (e.g. eggs, sperm) for use in the future.

Driving and using machines

The mobilisation medicine and the conditioning medicines used before treatment with Casgevy can cause dizziness and fatigue. If you feel dizzy, tired, or unwell, do not drive, use machines, or take part in activities that need you to be alert.

Casgevy contains sodium and dimethyl sulfoxide (DMSO)

This medicine contains approximately 5.3-70 mg of sodium (the main component of cooking/table salt) in each vial. This is equivalent to 0.3-4% of the maximum recommended daily intake of sodium for an adult. The total number of vials that make up a dose varies depending on the patient.

This medicine contains approximately 50 mg of DMSO per ml. See section 2 “Warnings and precautions”.

3. How Casgevy is prepared and administered

Casgevy is given once only.

Casgevy can only be given in a authorised treatment centre (a specialised hospital) by doctors with experience in stem cell transplants and in treating patients with blood disorders such as TDT and SCA.

STEP 1: Before treatment with Casgevy, your doctor will give you a mobilisation medicine. This medicine moves the blood stem cells from the bone marrow into the bloodstream. The cells are then collected in a machine that separates the different blood cells (this is called apheresis). This whole process may be repeated more than once. Each collection step takes about one week.

“Rescue cells”are also collected and stored in the hospital. These are your own blood stem cells and are kept unmodified in case there is a problem with the treatment process. See above in section 2, “If treatment with Casgevy cannot be completed or fails”.

STEP 2: Your blood stem cells will be sent to the manufacturing site where they will be used to make Casgevy. It may take up to 6 months from the time your cells are collected until Casgevy is made and tested before it is sent back to your doctor.

STEP 3: Shortly before the stem cell transplant, your doctor will give you a conditioning medicinein the hospital. This step takes 2 to 6 days and will prepare you for the treatment by clearing the cells from the bone marrow, so they can be replaced by the modified Casgevy cells. After you receive this medicine, your blood cell levels will drop to very low levels (see section 4). You will stay in the hospital until after the infusion of Casgevy.

STEP 4: You will be given one or more vials of Casgevy as an injection into a vein through a central venous catheter. Central venous catheters are thin, flexible tubes that a doctor inserts into a large vein to access the bloodstream. The risks of central venous catheters are infections and blood clots. Your doctor and nurses will monitor you for any complications of the central venous catheter. Giving all the injections may take several hours. Once you have received Casgevy, you will stay in the hospital so your healthcare team can closely monitor your recovery. This may take about 2 months, but the time may vary. A doctor will decide when you can go home.

4. Possible Adverse Effects

Like all medicines, this medicine can cause adverse effects, although not all people suffer from them. Consult your doctor or nurse about possible adverse effects.

Some adverse effects are related to the mobilization medicine and the conditioning medicine. You should also read the instructions for these medicines.

The following serious adverse effects may appear in the first few days or weeks after treatment, but they can also appear much later.

• Pain in the upper right part of the abdomen below the ribs, yellowish coloration of the eyes or skin, rapid weight gain, swelling of arms, legs, and abdomen, and difficulty breathing.
• These may be signs of a serious liver conditioncalled veno-occlusive disease.
• Severe headache, abnormal bruising, prolonged bleeding or bleeding without injury such as nosebleeds, bleeding gums, blood in urine, stools, or vomit, or coughing up blood.
• These may be signs of thrombocytopenia, low platelet levels,which can reduce the blood's ability to clot and may lead to bleeding.
• Fever, chills, or infections.
• These may be signs of neutropenia, low levels of a type of white blood cellcalled neutrophils that fight infections.

Tell your doctor immediatelyif you experience any of the above adverse effects.

Other Adverse Effects of Mobilization Medicine and Cell Collection

Very Common(may affect more than 1 in 10 people)

• nausea
• vomiting
• headache
• stomach pain
• bone or muscle pain

Common(may affect up to 1 in 10 people)

• pulmonary condition with symptoms such as sudden chest pain, fever, difficulty breathing, and signs of fluid accumulation in the lungs that can be seen on a chest X-ray, which occurs in sickle cell anemia (acute chest syndrome)
• painful crisis of sickle cell anemia (sickle cell anemia with crisis)
• fever
• high white blood cell levels (leukocytosis)
• diarrhea
• mouth and throat pain
• numbness of the mouth
• joint pain
• general pain
• feeling of fatigue
• low potassium levels in the blood (hypokalemia)
• low magnesium levels in the blood (hypomagnesemia)
• high phosphate levels in the blood (hyperphosphatemia)

Other Adverse Effects of Conditioning Medicine

• Very Common(may affect more than 1 in 10 people)
• fever during a period of low neutrophil count (a type of white blood cell) (febrile neutropenia)
• low red blood cell levels (anemia)
• low lymphocyte levels, a type of white blood cell (lymphopenia)
• low white blood cell levels (leukopenia)
• low potassium levels in the blood (hypokalemia)
• high phosphate levels in the blood (hyperphosphatemia)
• low magnesium levels in the blood (hypomagnesemia)
• low phosphate levels in the blood (hypophosphatemia)
• fluid retention
• headache
• fever
• feeling of fatigue
• nasal bleeding
• nausea
• vomiting
• stomach pain
• inflammation of the stomach lining
• constipation
• diarrhea
• mouth and throat pain
• inflammation of the mucous membranes, such as the gums (mucositis)
• decreased appetite
• weight loss
• bone or muscle pain
• dry skin
• skin peeling
• skin and nail color changes
• small blood spots under the skin
• rash
• hair loss (alopecia)
• high bilirubin levels in the blood, a breakdown product of red blood cells, which can cause yellowish coloration of the skin and eyes (hyperbilirubinemia)
• increased levels of a liver enzyme in the blood (alanine aminotransferase)

Common(may affect up to 1 in 10 people)

• set of symptoms similar to those of pneumonia, such as fever, chills, cough, and respiratory problems, which occur without signs of lung infection (idiopathic pneumonia syndrome)
• inability of the lungs to supply oxygen to the blood (respiratory failure)
• difficulty breathing
• blood infection (sepsis)
• blood infection caused by the bacteria Klebsiella (Klebsiella sepsis)
• lung infection (pneumonia)
• mouth infection caused by a fungus (oral candidiasis)
• infection of the hair follicles (folliculitis)
• increased heart rate (tachycardia)
• increased levels of liver enzymes in the blood (aspartate aminotransferase, gamma-glutamyltransferase)
• low blood pressure (hypotension)
• low oxygen levels in the blood (hypoxia)
• enlargement of the liver
• enlargement of the spleen
• nerve damage in the arms or legs that causes pain or numbness, burning, and tingling (peripheral neuropathy)
• problems with the nerves that cause pain or numbness, burning sensation, and tingling (peripheral sensory neuropathy)
• nerve pain
• problems with taste
• blurred vision
• dry eyes
• hot flashes
• cough
• indigestion
• reflux disease in which stomach acid rises above the stomach into the esophagus or digestive tube (gastroesophageal reflux disease)
• gum bleeding (gingival bleeding)
• throat pain
• difficulty swallowing
• inflammation of the large intestine that causes pain and diarrhea (colitis)
• inflammation of the digestive tube (esophagitis)
• blood in vomit
• rectal bleeding
• inflammation of the stomach and intestine
• mouth ulcers
• general pain
• pain while urinating
• blood in urine
• absence of menstruation
• bleeding between menstrual periods
• irregular menstruation
• pain in the vulva and vagina
• premature menopause
• weight gain
• bruising
• itching
• redness of the skin
• cuts or scrapes on the skin
• low levels of all types of blood cells (pancytopenia)
• low levels of reticulocytes, a type of immature red blood cell (reticulocytopenia)
• bleeding in a part of the brain related to balance and coordination (cerebellar hemorrhage)
• abnormal accumulation of fluid surrounding the brain and spinal cord (hydrocephalus)
• low levels of albumin, a blood protein (hypoalbuminemia)
• low levels of calcium in the blood (hypocalcemia)
• joint pain
• longer blood clotting time
• higher levels of an inflammation indicator (C-reactive protein)
• longer time for transplanted cells to start growing and producing normal blood cells (graft delay)

Other Adverse Effects of Casgevy

• Common(may affect up to 1 in 10 people)
• immune system disorder (hemophagocytic lymphohistiocytosis) in which types of white blood cells (histiocytes and lymphocytes) accumulate in organs, causing excessive inflammation and tissue destruction. Symptoms may include fever that is not due to an infection and does not respond to antibiotics, enlargement of the liver and/or spleen, skin rashes, respiratory problems, easy bruising, low blood pressure, kidney abnormalities, and heart problems
• difficulty breathing, which may require oxygen to help breathe, sometimes with chest pain, fever, chills, or cough (acute respiratory distress syndrome)
• set of symptoms similar to those of pneumonia, such as fever, chills, cough, and respiratory problems, which occur without signs of lung infection (idiopathic pneumonia syndrome)
• increased heart rate (tachycardia)
• low red blood cell levels (anemia)
• low white blood cell levels (leukopenia)
• low calcium levels in the blood (hypocalcemia)
• headache
• feeling of numbness, tingling, pinching (paresthesia)
• nasal bleeding
• rash
• small blood spots under the skin
• fever
• chills
• longer time for transplanted cells to start growing and producing normal blood cells (graft delay)
• events such as chills and increased heart rate when Casgevy is administered (infusion-related reactions)

Tell your doctor or nurseif you experience any of these adverse effects. Tell your doctor or nurse immediatelyif any of these adverse effects worsen.

Reporting Adverse Effects

If you experience any type of adverse effect, consult your doctor or nurse, even if it is an adverse effect that is not listed in this leaflet. You can also report them directly through the national reporting system included in Appendix V. By reporting adverse effects, you can contribute to providing more information on the safety of this medicine.

5. Storage of Casgevy

The following information is intended only for doctors and nurses.

Since this medicine will be administered by a qualified doctor or nurse, they are responsible for the correct storage of the medicine before and during its use, as well as its correct disposal.

Keep out of sight and reach of children.

Do not use this medicine after the expiration date shown on the box and on each vial.

Store frozen, at 135 °C or below, for a maximum of two years. Keep the vials in the box until the time of thawing. Thaw the vials one by one. Do not thaw until the time of infusion. Do not re-freeze after thawing. Once thawed, store at room temperature (20 °C to 25 °C) and infuse within 20 minutes.

This medicine contains human blood cells. Unused medicine must be disposed of in accordance with local guidelines for handling human-derived material.

6. Container Contents and Additional Information

Casgevy Composition

• The active ingredient is exagamglogén autotemcel. Each ml of Casgevy contains 4 - 13 × 10^6 cells (blood stem cells).
• The other components are a solution used to preserve frozen cells, which contains sodium, dimethyl sulfoxide (DMSO), and dextran 40. See section 2 "What you need to know before you start receiving Casgevy".

Appearance of the Product and Container Contents

Casgevy is a semi-transparent infusion dispersion. Casgevy is presented in vials of 1.5 ml to 20 ml. Each box contains one or more vials. A box may contain up to 9 vials. The number of vials depends on the dose of each patient. Your dose may consist of several vials and boxes.

Your name and date of birth, as well as the coded information that identifies you as the recipient, are printed on each box and vial.

Marketing Authorization Holder and Manufacturer

Marketing Authorization Holder:

Vertex Pharmaceuticals (Ireland) Limited

Unit 49, Block 5, Northwood Court, Northwood Crescent,

Dublin 9, D09 T665,

Ireland

Tel: +353 (0)1 761 7299

Manufacturer:

Vertex Pharmaceuticals (Europe) Limited

Unit 49, Block 5, Northwood Court, Northwood Crescent,

Dublin 9, D09 T665,

Ireland

Lonza Netherlands B.V.

Urmonderbaan 20 B

6167 RD Geleen

Netherlands

You can request more information about this medicinal product by contacting the local representative of the marketing authorization holder:

Date of Last Revision of this Leaflet:

This medicinal product has been authorized with a "conditional approval". This approval mechanism means that more information is expected to be provided on this medicinal product. The European Medicines Agency will review the new information on this medicinal product at least once a year, and this leaflet will be updated as necessary.

Other Sources of Information

Detailed information on this medicinal product is available on the European Medicines Agency website: https://www.ema.europa.eu.

The European Medicines Agency website can be found in all languages of the European Union/European Economic Area.

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For Healthcare Professionals Only

This information is intended for healthcare professionals only:

Precautions to be Taken Before Handling or Administering the Medicinal Product

Casgevy is intended for autologous use only. Do not take samples, alter, or irradiate the medicinal product. Irradiation could inactivate the medicinal product.

This medicinal product contains human blood cells. Healthcare professionals handling Casgevy must take appropriate precautions (wear gloves, protective clothing, and eye protection) to avoid the possible transmission of infectious diseases.

Casgevy Container and Storage

• Casgevy is shipped to the treatment center in a cryogenic container.
• Confirm the patient identifiers on the medicinal product label and the batch information sheet (LIS).
• Store in the vapor phase of liquid nitrogen at ≤ -135 °C until ready to thaw and administer.

Preparation Before Administration

• Coordinate the times of thawing and infusion of Casgevy. Confirm the infusion time in advance and adjust the start time of thawing so that Casgevy is available for infusion when the patient is ready, as Casgevy must be administered within 20 minutes of thawing the vial. Thaw and infuse one vial at a time.
• Before thawing, confirm that the patient's identity matches the patient information on the Casgevy vial or vials. Do not thaw the Casgevy vials if the patient-specific label information does not match the intended patient.
• A patient's dose of Casgevy may consist of one or more patient-specific cryopreserved vials. Count all vials and confirm that each vial is within the validity period using the batch information sheet (LIS) provided.
• Gather the necessary materials to thaw and extract the product from the vials. Except for the water bath, these materials are for single use. Gather sufficient materials for each vial to be administered:

• Water bath
• Alcohol swabs
• Vial adapter (for needle-free extraction)
• Stainless steel filter (18 microns)
• Luer Lock syringe (30 ml)
• Sodium chloride 9 mg/ml (0.9%) injectable solution (5-10 ml required for each vial)
• Luer Lock syringe (10 ml) for flushing with sodium chloride solution

Thawing of Casgevy Vials

• When the dose consists of multiple vials, thaw and administer each vial one at a time. While thawing one vial, the remaining vials must remain in cryopreservation at ≤ -135 °C.
• Thaw each vial at 37 °C using a water bath. Ensure the water bath temperature does not exceed 40 °C.
• Thaw each vial by holding the vial neck, gently agitating in a clockwise and counterclockwise direction. This may take between 10 and 15 minutes. Do not leave the vial unattended during thawing.
• Thawing is complete when ice crystals are no longer visible in the vial.
• Once thawed, immediately remove the vial from the water bath.
• The thawed product should have the appearance of a translucent cell dispersion without foreign particles.
• It must be infused within 20 minutes of thawing.
• The thawed medicinal product must not be re-frozen.

Administration of Casgevy

Casgevy is for autologous use only.The patient's identity must match the patient identifiers on the Casgevy vials. Do not infuse Casgevy if the patient-specific label information does not match the intended patient.

A patient's dose may consist of multiple vials.All vials must be administered. The entire volume of each vial provided must be infused. If more than one vial is supplied, administer each vial in its entirety before proceeding to thaw and infuse the next vial.

1. Placement of the vial adapter and filter

• Remove the flip-off cap from the vial stopper; clean the diaphragm with an alcohol swab.
• Remove the stopper cap from the adapter spike.
• With your thumb and index finger of both hands, push the adapter into the vial diaphragm, applying equal pressure until you hear a single click.
• Pull the adapter upwards until you feel it lock.
• Attach the filter to the vial adapter.

1. Extraction of Casgevy from the Vial

• Attach an empty 30 ml syringe to the filter.
• Extract the entire volume of the product from the vial.
• Remove the syringe filled with product from the filter and set it aside.
• Extract 5-10 ml of sodium chloride 9 mg/ml (0.9%) injectable solution in the empty 10 ml syringe.
• Attach the syringe filled with sodium chloride solution to the filter.
• Inject the sodium chloride solution and remove the empty syringe from the filter. Discard the empty syringe.
• Attach the syringe filled with product to the filter.
• Extract the vial contents into the product syringe and then remove the syringe from the filter.
• You may peel off the optional product/patient identification label from the batch information sheet (LIS) and attach it to the syringe.

1. Administration of Casgevy via a Central Venous Catheter

• Casgevy must be administered within 20 minutes of thawing the product.
• Two people should confirm and verify the patient's identity at the bedside before infusion of each vial.
• Casgevy is administered as an intravenous bolus.
• The total volume of Casgevy administered in one hour must not exceed 2.6 ml/kg.
• Do not use an in-line filter during the infusion of Casgevy.
• After administration of each Casgevy vial, flush the main line with sodium chloride 9 mg/ml (0.9%) injectable solution.

Repeat the above steps with each remaining vial.

Measures to be Taken in Case of Accidental Exposure

In case of accidental exposure, local guidelines for handling human-derived materials should be followed. Work surfaces and materials that may have come into contact with Casgevy should be decontaminated with an appropriate disinfectant.

Precautions to be Taken in the Disposal of the Medicinal Product

The unused medicinal product and all materials that have come into contact with Casgevy (solid and liquid waste) should be handled and disposed of as potentially infectious waste in accordance with local guidelines for handling human-derived materials.