What is ARALAST NP?

ARALAST NP is a medicine made from human Alpha ₁-Proteinase Inhibitor (Alpha ₁-PI), which is also known as alpha ₁-antitrypsin (AAT). It is used to treat adults with lung disease (emphysema) caused by severe Alpha ₁ antitrypsin (AAT) deficiency.

• It is not known how increasing AAT levels with ARALAST NP or other Alpha ₁-PI products impacts worsening lung function or emphysema.
• The long-term effects of AAT with ARALAST NP have not been studied.
• ARALAST NP is not for use in lung disease other than severe Alpha ₁-PI deficiency.

ARALAST NP IMPORTANT SAFETY INFORMATION

Who should not use ARALAST NP?

Do not use ARALAST NP if you have IgA deficiency with antibodies to IgA due to the risk of severe allergic reaction.

What is the most important information I need to know about ARALAST NP?

• Severe allergic reactions can occur with ARALAST NP. Symptoms of severe allergic reactions include:
  • Hives
  • Itching
  • Chest tightness
  • Shortness of breath
  • Wheezing
  • Faintness
  • Low blood pressure
  • Anaphylaxis

If you have any of these symptoms, stop using the product and call your doctor right away or go to the emergency department.

• ARALAST NP is made from human plasma and may contain infectious agents, such as viruses. Talk to your doctor if you have questions about how the product is made.

What are the most common side effects of ARALAST NP?

• The most common side effects are: headache, muscle and/or bone pain, infusion site bruising, upset stomach, and runny nose.

These are not all the possible side effects of ARALAST NP. Tell your doctor about any side effect that bothers you or doesn’t go away.

What should I tell my doctor before using ARALAST NP?

Before starting ARALAST NP, tell your doctor if you have IgA deficiency with antibodies to IgA.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1‑800‑FDA‑1088.

For additional safety information, click for Full Prescribing Information and discuss with your doctor.

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What is CINRYZE^®?

CINRYZE^® (C1 esterase inhibitor [human]) is an injectable prescription medicine that is used to help prevent swelling and/or painful attacks in children (6 years of age and older), teenagers, and adults with Hereditary Angioedema (HAE).

CINRYZE^® IMPORTANT SAFETY INFORMATION

You should not use CINRYZE^® if you have had life-threatening immediate hypersensitivity reactions, including anaphylaxis, to the product.

Tell your healthcare provider about all of your medical conditions, including if you have an indwelling catheter/access device in one of your veins; have a history of blood clots, heart disease, or stroke; or are taking birth control pills or androgens. Also tell your healthcare provider if you are pregnant or planning to become pregnant, or are breastfeeding or planning to breastfeed. It is not known if CINRYZE^® can harm your unborn baby, or if CINRYZE^® passes into your milk and if it can harm your baby.

Allergic reactions may occur with CINRYZE^®. Call your healthcare provider or get emergency support services right away if you have any of the following symptoms: wheezing, difficulty breathing, chest tightness, turning blue (look at lips and gums), fast heartbeat, swelling of the face, faintness, rash, hives.

Serious blood clots may occur with CINRYZE^®. Call your healthcare provider or get emergency support services right away if you have any of the following symptoms: pain and/or swelling of an arm or leg with warmth over the affected area, discoloration of an arm or leg, unexplained shortness of breath, chest pain or discomfort that worsens on deep breathing, unexplained rapid heart rate, numbness or weakness on one side of the body.

Because CINRYZE^® is made from human blood, it may carry a risk of transmitting infectious agents, e.g., viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent.

The most common side effects seen with CINRYZE^® were headache, nausea, rash, and vomiting. These are not all the possible side effects of CINRYZE^®. Tell your healthcare provider about any side effect that bothers you or that does not go away. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit w‍ww‍.fda‍.gov‍/medwatch, or call 1‑800‑FDA‑1088.

For additional safety information, please click here for Full Prescribing Information and discuss with your doctor.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1‑800‑FDA‑1088.

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What is ELAPRASE^® (idursulfase)?

ELAPRASE is a prescription medicine for patients with Hunter syndrome.

ELAPRASE has been shown to improve walking ability in patients 5 yrs and older.

In patients 16 months to 5 yrs old, ELAPRASE did not show improvement in disease-related symptoms or long term clinical result; however, treatment with ELAPRASE has reduced spleen size similarly to patients 5 yrs and older.

It is not known if ELAPRASE is safe and effective in children under 16 months old.

ELAPRASE IMPORTANT SAFETY INFORMATION

You or your child should be closely watched during and after ELAPRASE treatment and you should confirm with your healthcare team in advance of treatment that it is prepared to manage serious allergic reactions, including anaphylactic reactions. Tell your healthcare team immediately if any signs of an allergic reaction happen. Those signs may include breathing problems, low blood pressure, rash, hives, itching, flushing, fever and/or headache.

When serious allergic reactions happened during clinical trials, later ELAPRASE treatments were managed with allergy-controlling drugs before or during treatment, a slower rate of ELAPRASE treatment, and/or early discontinuation of treatment.

Children with serious genetic mutations may be at risk for allergic reactions, serious side effects and antibody development. In a clinical study of children 7 years and younger, patients with certain types of genetic mutations experienced a higher number of allergic reactions, serious side effects, and development of an immune response to treatment. This immune response may interfere with the effectiveness of ELAPRASE. Talk to your healthcare team about whether you or your child may be at risk.

If you or your child has breathing problems, other respiratory illness, heart problems, or susceptibility to fluid overload, you or your child may be at higher risk of fluid overload during ELAPRASE treatment. Your healthcare team should be advised of those problems before treatment and you should confirm with your healthcare team in advance of treatment that it is appropriately trained to watch for signs of fluid overload and provide the necessary medical support. Patients at risk for fluid overload may require longer observation time.

What are possible side effects of ELAPRASE?

The most common side effects of ELAPRASE include:

• In patients aged 5 and older:
  • Headache
  • Itching
  • Muscle and bone pain
  • Hives
  • Diarrhea
  • Cough

• In patients aged 7 and older:
  • Fever
  • Rash
  • Vomiting
  • Hives

The most common side effects needing medical attention were allergic reactions, and included rash, hives, itching, flushing, fever, and headache. Tell your healthcare team immediately if any signs of an allergic reaction happen. These are not all the possible side effects of ELAPRASE.

For additional safety information, please click here for Full Prescribing Information, including Boxed Warning for Risk of Anaphylaxis, and discuss with your doctor.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1‑800‑FDA‑1088.

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FIRAZYR IMPORTANT SAFETY INFORMATION

Laryngeal attacks can become life threatening. If you have an HAE attack of the throat (laryngeal attack), inject FIRAZYR and then go to the nearest hospital emergency room right away.

The most common side effects of FIRAZYR include:

• redness, bruising, swelling, warmth, burning, itching, irritation, hives, numbness, pressure, or pain at the injection site
• fever
• too much of an enzyme called transaminase in your blood
• dizziness
• nausea
• headache
• rash

These are not all of the possible side effects of FIRAZYR. Tell your healthcare provider if you have any side effect that bothers you or that does not go away. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1‑800‑FDA‑1088.

Tell your healthcare provider if you have any other medical conditions, if you are breastfeeding or plan to breastfeed, or if you are pregnant or planning to become pregnant. FIRAZYR has not been evaluated in pregnant or nursing women. You and your healthcare provider will decide if FIRAZYR is right for you.

If your symptoms continue or come back, you may repeat your FIRAZYR injection at least 6 hours apart. Do not use more than 3 doses of FIRAZYR in a 24-hour period.

Tiredness, drowsiness, and dizziness have been reported following the use of FIRAZYR. If this occurs, do not drive a car, use machinery, or do anything that needs you to be alert.

Please see the full Prescribing Information, including Patient Information.

Indication

FIRAZYR is a medicine used to treat acute attacks of hereditary angioedema (HAE) in adults 18 years of age and older.

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What is GATTEX?

GATTEX® (teduglutide) for subcutaneous injection is a prescription medicine used in adults and children 1 year of age and older with Short Bowel Syndrome (SBS) who need additional nutrition or fluids from intravenous (IV) feeding (parenteral support). It is not known if GATTEX is safe and effective in children under 1 year of age.

GATTEX IMPORTANT SAFETY INFORMATION

What is the most important information I should know about GATTEX?

GATTEX may cause serious side effects, including:

Making abnormal cells grow faster

GATTEX can make abnormal cells that are already in your body grow faster. There is an increased risk that abnormal cells could become cancer. If you get cancer of the bowel (intestines), liver, gallbladder or pancreas while using GATTEX, your healthcare provider should stop GATTEX. If you get other types of cancers, you and your healthcare provider should discuss the risks and benefits of using GATTEX.

Polyps in the colon (large intestine)

Polyps are growths on the inside of the colon. Your healthcare provider will have your colon checked for polyps within 6 months before starting GATTEX and have any polyps removed. Children and adolescents will be checked for blood in the stool before they start using GATTEX. To keep using GATTEX, your healthcare provider should have your colon checked for new polyps at the end of 1 year of using GATTEX. If no polyp is found, your healthcare provider should check you for polyps as needed and at least every 5 years and have any new polyps removed. If cancer is found in a polyp, your healthcare provider should stop GATTEX.

Blockage of the bowel (intestines)

A bowel blockage keeps food, fluids, and gas from moving through the bowels in the normal way. Tell your healthcare provider right away if you have any of these symptoms of a bowel or stomal blockage:

• trouble having a bowel movement or passing gas
• stomach area (abdomen) pain or swelling
• nausea
• vomiting
• swelling and blockage of your stoma opening, if you have a stoma

If a blockage is found, your healthcare provider may temporarily stop GATTEX.

Swelling (inflammation) or blockage of your gallbladder or pancreas

Your healthcare provider will do tests to check your gallbladder and pancreas within 6 months before starting GATTEX and at least every 6 months while you are using GATTEX. Tell your healthcare provider right away if you get:

• stomach area (abdomen) pain and tenderness
• chills
• fever
• a change in your stools
• nausea
• vomiting
• dark urine
• yellowing of your skin or the whites of your eyes

Fluid overload

Your healthcare provider will check you for too much fluid in your body. Too much fluid in your body may lead to heart failure, especially if you have heart problems. Tell your healthcare provider if you get swelling in your feet and ankles, you gain weight very quickly (water weight), or you have trouble breathing.

The most common side effects of GATTEX in adults include:

• stomach area (abdomen) pain or swelling
• nausea
• cold or flu symptoms
• skin reaction where the injection was given
• vomiting
• swelling of the hands or feet
• allergic reactions

The side effects of GATTEX in children and adolescents are similar to those seen in adults.

Tell your healthcare provider if you have any side effect that bothers you or that does not go away.

What should I tell my healthcare provider before using GATTEX?

Tell your healthcare provider about all your medical conditions, including if you or your child:

• have cancer or a history of cancer
• have or had polyps anywhere in your bowel (intestines) or rectum
• have heart problems
• have high blood pressure
• have problems with your gallbladder, pancreas, kidneys
• are pregnant or planning to become pregnant. It is not known if GATTEX will harm your unborn baby. Tell your healthcare provider right away if you become pregnant while using GATTEX.
• are breastfeeding or plan to breastfeed. It is not known if GATTEX passes into your breast milk. You should not breastfeed during treatment with GATTEX. Talk to your healthcare provider about the best way to feed your baby while using GATTEX.

Tell your healthcare providers about all the medicines you take, including prescription or over-the-counter medicines, vitamins, and herbal supplements. Using GATTEX with certain other medicines may affect each other causing side effects. Your other healthcare providers may need to change the dose of any oral medicines (medicines taken by mouth) you take while using GATTEX. Tell the healthcare provider who gives you GATTEX if you will be taking a new oral medicine.

Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1‑800‑FDA‑1088.

For additional safety information, click here for full Prescribing Information and Medication Guide, and discuss any questions with your doctor.

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What is GLASSIA?

GLASSIA is a medicine containing human Alpha ₁-Proteinase Inhibitor (Alpha ₁-PI) that is used to treat adults with lung disease (emphysema) because of severe Alpha ₁-antitrypsin (Alpha ₁) deficiency. GLASSIA is not meant to be used as a therapy for lung disease other than severe Alpha ₁ deficiency. Effects of GLASSIA on worsening lung function and emphysema progression have not been proven in clinical trials. Long-term effects of Alpha ₁ replacement and maintenance therapy have not been studied.

GLASSIA IMPORTANT SAFETY INFORMATION

What is the most important information I need to know about GLASSIA?

• GLASSIA can cause severe allergic reactions including hives, swelling in the mouth or throat, itching, tightness in the chest, trouble breathing, wheezing, faintness or low blood pressure
• If you will be taking GLASSIA outside a healthcare setting, ask your healthcare provider (HCP) about an epinephrine pen and/or other supportive care for certain severe allergic reactions.

Who should not use GLASSIA?

Do not use GLASSIA if you:

• Have immunoglobulin A (IgA) deficiency with antibodies to IgA
• Have a severe allergic reaction to human Alpha₁-PI products.

What are the possible or reasonably likely side effects of GLASSIA?

If any of the following problems occur contact your healthcare provider (HCP) or call emergency services right away:

• Worsening or flare-up of your chronic obstructive pulmonary disease (COPD)
• Hives, swelling in the mouth or throat, itching, chest tightness, trouble breathing, wheezing, fainting or dizziness. These could be signs of a serious allergic reaction.

The most common side effects that may occur are headache and upper respiratory tract infections

Other possible side effects of GLASSIA include:

• Cough
• Sinus infection
• Chest discomfort
• Dizziness
• Increased liver enzymes
• Shortness of breath
• Nausea
• Fatigue

These are not all the possible side effects. Tell your HCP about any side effect that bothers you or that does not go away.

For additional safety information, click for Information For Patients including Instructions For Use and discuss with your HCP.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

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What is KALBITOR?

KALBITOR^® is a prescription medicine used to treat sudden attacks of hereditary angioedema (HAE) in people 12 years of age and older. KALBITOR is not a cure for HAE.

KALBITOR IMPORTANT SAFETY INFORMATION

What is the most important information I should know about KALBITOR?

Serious allergic reactions may happen in some people who receive KALBITOR. These allergic reactions can be life-threatening and usually happen within 1 hour after receiving KALBITOR.

• KALBITOR should be given to you by a doctor or nurse in a healthcare setting where serious allergic reactions and hereditary angioedema (HAE) can be treated.
• Symptoms of a serious allergic reaction to KALBITOR can be similar to the symptoms of HAE, the condition that you are being treated for. Your doctor or nurse should watch you for any signs of a serious allergic reaction after treatment with KALBITOR.
• Tell your doctor or nurse right away if you have any of these symptoms of a serious allergic reaction during or after treatment with KALBITOR:
  • wheezing, shortness of breath, cough, chest tightness, or trouble breathing
  • dizziness, fainting, fast or weak heartbeat, or feeling nervous
  • reddening of the face, itching, hives, or feeling warm
  • swelling of the throat or tongue, throat tightness, hoarse voice, or trouble swallowing
  • runny nose, nasal congestion, or sneezing

Who should not receive KALBITOR?

Do not receive KALBITOR if you are allergic to KALBITOR. Before receiving KALBITOR, tell your doctor if you have ever had an allergic reaction to KALBITOR.

What else should I tell my doctor before I receive KALBITOR?

Tell your doctor if you are pregnant, plan to become pregnant, breast-feeding, or plan to breast-feed. It is not known if KALBITOR will harm your unborn baby or pass into your breast milk.

Tell your doctor about all the medicines you take, including prescriptions and over-the-counter medicines, vitamins, and herbal supplements.

What are the possible side effects?

Common side effects of KALBITOR include headache, nausea, diarrhea, fever, injection site reactions, such as redness, rash, swelling, itching, or bruising, and stuffy nose. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1‑800‑FDA‑1088.

Talk to your doctor for more information about your medical condition or treatment.

Please see the Full Prescribing Information, including Boxed Warning and Medication Guide.

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What is TAKHZYRO?

TAKHZYRO is a prescription medicine used to prevent attacks of hereditary angioedema (HAE) in people 12 years of age and older.

It is not known if TAKHZYRO is safe and effective in children under 12 years of age.

TAKHZYRO IMPORTANT SAFETY INFORMATION

TAKHZYRO may cause serious side effects, including allergic reactions. Call your healthcare provider or get emergency help right away if you have any of the following symptoms:

• wheezing
• difficulty breathing
• chest tightness
• fast heartbeat
• faintness
• rash
• hives

The most common side effects seen with TAKHZYRO were injection site reactions (pain, redness, and bruising), upper respiratory infection, and headache.

These are not all the possible side effects of TAKHZYRO. For more information, ask your healthcare provider or pharmacist. You may report side effects to the FDA at 1‑800‑FDA‑1088.

TAKHZYRO has not been studied in pregnant or breastfeeding women. Talk to your healthcare provider about the risk of taking TAKHZYRO if you are pregnant, plan to be pregnant, are breastfeeding, or plan to breastfeed.

Please see full Prescribing Information, including information for patients.

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VPRIV INDICATION

VPRIV^® (velaglucerase alfa) for injection is a prescription medication indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.

VPRIV IMPORTANT SAFETY INFORMATION

Hypersensitivity reactions, including serious allergic reactions (anaphylaxis) have occurred. VPRIV should be administered under the supervision of a healthcare professional. VPRIV is given every other week by intravenous infusion that typically takes up to 60 minutes. Appropriate medical support should be available when VPRIV is administered. The most serious side effects in patients treated with VPRIV were hypersensitivity reactions.

Hypersensitivity reactions were the most commonly observed side effects in patients treated with VPRIV in clinical studies. The most commonly observed symptoms of hypersensitivity reactions were: headache, dizziness, low blood pressure, high blood pressure, nausea, tiredness/weakness, and fever. Hypersensitivity reactions in the clinical trials include any event considered related to and occurring within up to 24 hours of VPRIV infusion, including one case of anaphylaxis. Generally the reactions were mild and, in patients not previously treated, occurred mostly during the first 6 months of treatment and tended to occur less frequently with time. After the drug was approved, additional hypersensitivity reactions of chest discomfort, difficulty breathing, itching and vomiting have been reported. In some cases, vomiting can be serious and require hospitalization and/or stopping the medication.

If anaphylactic or other acute reactions occur, your healthcare provider will immediately discontinue the infusion of VPRIV and initiate the appropriate medical treatment. A hypersensitivity reaction should be treated based on the severity of the reaction. Your healthcare provider may manage a reaction by slowing the infusion rate or treating with medicine such as antihistamines, fever-reducing agents and/or corticosteroids or possibly stopping the medication and then restarting with a longer infusion time. For patients who have had symptoms of hypersensitivity reaction to enzyme replacement therapy, the doctor may consider treating the patient with antihistamines and/or corticosteroids before an infusion to help prevent such a reaction from happening.

The most commonly reported side effects during clinical studies (in ≥10% of patients) were hypersensitivity reactions, headache, dizziness, abdominal pain, nausea, back pain, joint pain, increased time it takes for blood to clot, tiredness/weakness, and fever. In clinical studies, the overall frequency of side effects was generally higher in the patients not previously treated with ERT than in the patients who switched from imiglucerase to VPRIV.

Talk to your doctor if you are pregnant, plan to be pregnant, are breastfeeding, or plan to breastfeed.

The safety and efficacy profiles were similar in pediatric (ages 4 to 17) and adult patients. The safety of VPRIV has not been established in patients under 4 years of age. Side effects more commonly seen in pediatric patients compared to adult patients include (>10% difference): rash, increased time it takes for blood to clot, and fever.

The side effect profile in elderly patients was generally similar to that seen in pediatric and other adult patients. In general, dose selection for an elderly patient should be approached cautiously, considering other existing medical conditions.

As with all therapeutic proteins, there is a potential for developing antibodies to VPRIV. In clinical studies, 1 of 54 (2%) patients who had not previously been treated with ERT, who were then treated with VPRIV, developed antibodies. One additional patient developed antibodies to VPRIV during an extension study. It is unknown if having antibodies to VPRIV is associated with a higher risk of infusion reactions. Patients with an immune response to other enzyme replacement therapies who are switching to VPRIV should continue to be monitored for antibodies to VPRIV.

For additional safety information, please click here for Full Prescribing Information and discuss with your doctor.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1‑800‑FDA‑1088.