Discover CRYSVITA and Redefine XLH TREATMENT

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1 Discover CRYSVITA and Redefine XLH TREATMENT CRYSVITA is a prescription medicine used to treat adults and children 1 year of age and older with X-linked hypophosphatemia (XLH). 10 mg/ml, 20 mg/ml, 30 mg/ml You should not take CRYSVITA if: You take an oral phosphate supplement and a specific form of vitamin D supplement Your phosphorus levels from a blood sample are within or above the normal range for age You have kidney problems Please see Important Safety Information on page 10 and throughout this brochure and enclosed full Prescribing Information.

2 It s time to redefine XLH treatment with CRYSVITA (burosumab-twza). Table of Contents THE ONLY FDA-APPROVED TREATMENT FOR XLH, CRYSVITA TREATS THE UNDERLYING CAUSE OF THE DISEASE. This brochure will help you and your family understand what XLH is and how CRYSVITA may help. You ll learn how CRYSVITA works, how to get started, and tips for preparing you or your child for treatment. Page What is XLH? 4 How do I get XLH? 5 How CRYSVITA is designed to work 6 How do I take CRYSVITA? 7 CRYSVITA clinical studies 8 Important Safety Information 10 UltraCare Patient Support 12 Getting Started with CRYSVITA 13 Your CRYSVITA appointment 14 Patient Stories 15 What is the most important information you should know about CRYSVITA? Some patients developed allergic reactions (rash and hives) while taking CRYSVITA. Your doctor will monitor you for symptoms of an allergic reaction while you are taking CRYSVITA. High levels of phosphorus in the blood have been reported in some patients taking CRYSVITA. This may be related to a risk of high calcium levels in the kidneys. Your doctor will collect samples to monitor your levels. Administration of CRYSVITA may result in reactions at the injection site, such as hives, reddening of the skin, rash, swelling, bruising, pain, severe itching of the skin, and collection of blood outside of a blood vessel (hematoma). 2 3

3 What is XLH? XLH, or X-linked hypophosphatemia, is a rare skeletal condition that can affect the health of bones in children and adults. People with XLH don t have enough of an important mineral called phosphorus in their blood. Having too little phosphorus a condition known as hypophosphatemia can cause bones to become soft and weak over time. Some doctors may refer to XLH by other names such as: Familial Hypophosphatemia Genetic Rickets Hypophosphatemic Vitamin D-Resistant Rickets Vitamin D-Resistant Rickets Common Signs and Symptoms In children, XLH causes rickets, which leads to delayed growth, short stature and bone abnormalities. In adults, XLH causes osteomalacia, which makes these individuals more prone to bone fractures. Who Gets XLH? In most cases, XLH is an inherited disease, which means parents pass XLH down to their children. If you or your child have XLH, other people in your extended family may have it too. If a mother has XLH, all of her children have a 50% chance of inheriting XLH. If a father has XLH, all of his daughters and none of his sons will inherit XLH. Some children are born with XLH even though there is no family history. This occurs in 20-30% of cases and is called a spontaneous case. People who have a spontaneous case of XLH can pass the condition on to their children. Before taking CRYSVITA (burosumab-twza), tell your doctor about all of your medical conditions, including if you: are pregnant, think you may be pregnant, or plan to become pregnant. There is not enough experience to know if CRYSVITA may harm your unborn baby. Report pregnancies to the Ultragenyx Adverse Event reporting line at are breastfeeding or plan to breastfeed. There is not enough experience to know if CRYSVITA passes into your breast milk. Talk with your doctor about the best way to feed your baby while you receive CRYSVITA. 4 5

4 CRYSVITA (burosumab-twza), is a medicine that addresses the underlying cause of XLH How is CRYSVITA designed to work? A protein called fibroblast growth factor 23 (FGF23) controls the balance of phosphorus in the body. People with XLH have too much FGF23, causing them to lose phosphorus through their urine in a process called phosphate wasting. This causes low levels of phosphorus circulating in the bloodstream, called low serum phosphorus. CRYSVITA is an antibody that works by blocking the extra FGF23 so that phosphorus levels can return to normal. How do I take CRYSVITA? A healthcare provider gives CRYSVITA as an injection under the skin, in the upper arm, upper thigh, buttocks or stomach. The injection site should be rotated. Buttocks Stomach Upper Arm Upper Thigh The amount of CRYSVITA you or your child takes (called the dose) is based on body weight. The maximum dose is 90 milligrams. In children, CRYSVITA is given every 2 weeks In adults, CRYSVITA is given every 4 weeks While taking CRYSVITA, tell your doctor if you experience: An allergic reaction such as rash or hives A rash, swelling, bruising or other reaction at the injection site New or worsening restless leg syndrome 6 7

5 Clinical Study Results CRYSVITA (burosumab-twza), in Children: Increased and maintained serum phosphorus levels in the normal range Helped heal rickets and correct leg bone abnormalities Supported growth by improving height Study 1 In 26 children (ages 5-12 years) treated with CRYSVITA every 2 weeks: Phosphorus levels increased from below normal (2.4 milligrams/deciliter [mg/dl]) at the start of the study to within the normal range after 40 and 64 weeks (3.3 and 3.4 mg/dl) a 69% (18 of 26) achieved substantial healing of rickets after 40 weeks, which was maintained after 64 weeks Average mean standing height increased by 11% after 64 weeks b Study 2 In 13 children (ages 1-4 years) treated with CRYSVITA every 2 weeks: 100% increased and maintained their phosphorus levels within the normal range (from 2.5 mg/dl at the start of the study to 3.5 mg/dl after 40 weeks) a 100% had substantial healing of rickets after 40 weeks The children also demonstrated improvements in bone abnormalities of the lower limbs. Adverse Events The most common adverse reactions that were seen in children with XLH taking CRYSVITA are: Headache Injection site reaction Vomiting Fever Pain in arms and legs Decreased vitamin D levels Rash Toothache Muscle pain Tooth infection Dizziness The risks and benefits of CRYSVITA have been tested in four clinical trials involving 213 people with XLH (65 children and 148 adults). CRYSVITA in Adults: Increased and maintained serum phosphorus levels in the normal range Helped heal fractures related to osteomalacia Helped heal osteomalacia Study 3 In 134 adults (ages years), 68 were treated with CRYSVITA every 4 weeks and 66 received a placebo (a non-active ingredient) 94% (64 of 68) treated with CRYSVITA had normal blood phosphorus levels after 24 weeks, compared to 8% (5 of 66) who received a placebo c 43% of total fractures were healed in treated adults within 24 weeks, versus 8% of total fractures in the placebo group Through week 24, 6 new fractures appeared in patients receiving CRYSVITA compared to 8 in patients receiving placebo Study 4 In 14 adults (ages 25-52) treated with CRYSVITA every 4 weeks: CRYSVITA demonstrated healing of osteomalacia in 10 of the 14 patients at 48 weeks Bone biopsies of the 10 patients showed a 57% reduction in osteoid volume to bone volume, which is a measurement of mineralized bone. Adverse Events The most common adverse reactions that were seen in adults with XLH taking CRYSVITA are: Back pain Headache Tooth infection Restless leg syndrome Decreased vitamin D levels Dizziness Constipation Phosphorus levels increased in the blood Narrowing of the spaces within the spine is common in adults with XLH and pressure on the spinal cord has been reported in adults taking CRYSVITA. It is not known if taking CRYSVITA worsens the narrowing of the spaces within the spine or the pressure on the spinal cord. a Normal serum phosphorus levels for this group of patients ranged from 3.2 to 6.1 milligrams/deciliter (mg/dl). The normal range of serum phosphorus levels varies by age and gender. b This number is from the LS mean (SE) change of (0.05) (95% confidence interval [CI]: 0.09 to 0.29). LS mean is a statistic that estimates the mean after adjusting for the effect of other variables in the study, and the 95% CI is a range that includes 8 possible values for the change in height. 9 c Normal levels of serum phosphorus levels for this group of patients ranged from 2.5 to 4.5 milligrams/ deciliter (mg/dl). The normal range of serum phosphorus levels varies by age and gender.

6 IMPORTANT SAFETY INFORMATION You should not take CRYSVITA (burosumab-twza), if: You take an oral phosphate supplement and a specific form of vitamin D supplement Your phosphorus levels from a blood sample are within or above the normal range for age You have kidney problems What is the most important information you should know about CRYSVITA? Some patients developed allergic reactions (rash and hives) while taking CRYSVITA. Your doctor will monitor you for symptoms of an allergic reaction while you are taking CRYSVITA. High levels of phosphorus in the blood have been reported in some patients taking CRYSVITA. This may be related to a risk of high calcium levels in the kidneys. Your doctor will collect samples to monitor your levels. Administration of CRYSVITA may result in reactions at the injection site, such as hives, reddening of the skin, rash, swelling, bruising, pain, severe itching of the skin, and collection of blood outside of a blood vessel (hematoma). What are the possible side effects of CRYSVITA? The most common adverse reactions that were seen in children with XLH are: Headache Injection site reaction Vomiting Fever Pain in arms and legs Decreased vitamin D levels Rash Toothache Muscle pain Tooth infection Dizziness Narrowing of the spaces within the spine is common in adults with XLH and pressure on the spinal cord has been reported in adults taking CRYSVITA. It is not known if taking CRYSVITA worsens the narrowing of the spaces within the spine or the pressure on the spinal cord. Before taking CRYSVITA, tell your doctor about all of your medical conditions, including: If you are pregnant, think you may be pregnant, or plan to become pregnant. There is not enough experience to know if CRYSVITA may harm an unborn baby. Report pregnancies to the Ultragenyx Adverse Event reporting line at If you are breastfeeding or plan to breastfeed. There is not enough experience to know if CRYSVITA passes into breast milk. Talk with your doctor about the best way to feed your baby while you receive CRYSVITA. While taking CRYSVITA, tell your doctor if you experience: An allergic reaction such as rash or hives A rash, swelling, bruising or other reaction at the injection site New or worsening restless leg syndrome These are not all the possible side effects of CRYSVITA. Call your doctor for medical advice about side effects. You may report side effects to the FDA at (800) FDA-1088 or You may also report side effects to Ultragenyx at The most common adverse reactions that were seen in adults with XLH are: Back pain Headache Tooth infection Restless leg syndrome Decreased vitamin D levels Dizziness Constipation Phosphorus levels increased in the blood 10 11

7 Patient Support Ultragenyx is committed to patients with rare diseases, which is why we created UltraCare a suite of services designed to help you access CRYSVITA. Our UltraCare Guides are experienced professionals who are passionate about supporting you at every step. They can help you: Understand your insurance coverage and explain your benefits Determine your eligibility for financial and patient assistance programs Access patient support resources To learn more visit or contact our UltraCare Guides at ; option 1. Getting Started with CRYSVITA Your doctor can help decide if CRYSVITA is right for you or your child. Before the first CRYSVITA treatment, be prepared to answer a few questions about you or your child s recent medical history. Questions the doctor or nurse may ask you: Are you currently taking any medications? Is there a chance you could be pregnant? Are you planning to become pregnant? Are you currently breastfeeding, or do you plan to breastfeed? Do you have any kidney problems? Your answers to these questions may impact your CRYSVITA treatment plan. You should not take CRYSVITA if: You take an oral phosphate supplement and a specific form of vitamin D supplement Your phosphorus levels from a blood sample are within or above the normal range for age You have kidney problems 12 13

8 Your CRYSVITA (burosumab-twza), Appointment Starting any new medication may take some time to get used to. Before receiving CRYSVITA, the healthcare provider may take a blood sample to measure phosphorus levels. Once CRYSVITA treatment begins, they will continue to check phosphorus and if it is not quite right, the doctor may adjust the dose. Your doctor will determine the appropriate plan for you or your child. A healthcare provider administers CRYSVITA. This can be done at a doctor s office or in your home. The injection should take a few seconds and the healthcare provider may also monitor you or your child for a short period of time after receiving treatment. If you or your child is nervous about the injection, ask whether a shot blocker or a numbing cream placed on the skin could help. After your appointment Follow up with your doctor if you have questions or concerns. Be sure to keep your appointments for scheduled CRYSVITA injections. Writing them down, marking appointments on a calendar, or putting them in your phone may help you remember. While taking CRYSVITA, tell your doctor if you experience: An allergic reaction such as rash or hives A rash, swelling, bruising or other reaction at the injection site New or worsening restless leg syndrome

9 MRCP-KRN June Ultragenyx Pharmaceutical Inc. All rights reserved. CRYSVITA is a registered trademark of Kyowa Hakko Kirin Co., Ltd.