This way forward to proven results in children
XENPOZYME was proven to show improvements
across multiple organs
Clinical trial in children:
- The safety and effectiveness of XENPOZYME were evaluated in children with ASMD type B or type A/B who were <18 years old (7 children from 2 to <12 years old, and 1 child <2 years old) who all received XENPOZYME over 64 weeks in a clinical trial.
In children, XENPOZYME:
After 1 year of treatment, children taking XENPOZYME experienced improvements in the following exploratory endpoints from baseline:
ALT=alanine aminotransferase; ASMD=acid sphingomyelinase deficiency; AST=aspartate aminotransferase; DLco=diffusing capacity of the lungs for carbon monoxide.
AFTER 2 YEARS
XENPOZYME demonstrated sustained and continuous long-term improvements in multiple organs
All children from the clinical trial continued treatment with XENPOZYME in the Long-Term Trial and were treated for 2.5 to 3.2 years.
Long-Term Trial results
- Children continued to experience improvement in lung function, reduction in spleen volume, reduction in liver volume, and increase in platelet count.
- Children continued to experience improvement in height Z-scores when evaluated through 24 months.
- Children experienced improvement in bone age. This was assessed by hand x-ray.
- At the start of the clinical trial in children, the average bone age was delayed by a little over 2 years. In the Long-Term Trial, after 2 years of treatment with XENPOZYME, the average bone age improved by 1 year. This means bone age became closer to chronological age.
The safety of XENPOZYME was studied in 2 clinical trials in children
Safety profile in children
The safety of XENPOZYME was evaluated in the clinical trial over 64 weeks and in the Long-Term Trial for an overall observation period up to 3.2 years.
The most frequently reported side effects in children (incidence ≥20%)
These are not all the possible side effects of XENPOZYME. Tell your doctor about any side effects that you or your child may experience.
Serious anaphylactic reactions were reported in 2 (25%) children treated with XENPOZYME.
Hypersensitivity reactions: In the clinical trials, some children experienced mild, moderate, or severe hypersensitivity reactions with XENPOZYME.
Infusion-associated reactions (IARs): Some children experienced side effects in the clinical trials that may have been associated with the XENPOZYME infusion. These typically occurred between the time of infusion and up to 24 hours after the infusion was complete.
During the infusion, the doctor will monitor for IARs. If you or your child is having a reaction to the infusion, whether mild or severe, tell your doctor or infusion nurse right away. The doctor may slow or stop the infusion and may lower the next dose.
In the clinical trials, the majority of IARs with XENPOZYME were mild to moderate
- No one in the clinical trials stopped receiving XENPOZYME due to side effects.
IMPORTANT SAFETY INFORMATION AND INDICATION
WARNING: SEVERE ALLERGIC REACTIONS
Allergic Reactions Including Anaphylaxis
WARNINGS AND PRECAUTIONS
Allergic Reactions (Including Anaphylaxis) and Infusion-Associated Reactions (IARs)
See Boxed WARNING for more information. Your doctor may decide to give you antihistamine, anti-fever, and/or steroid medications before your infusions. Signs of allergic reactions and infusion-associated reactions (IARs) included hives, itchy skin, skin redness, rash, swelling underneath the skin, tender bumps under the skin, and localized swelling, as well as headache, vomiting, nausea, fever, and diarrhea.
Reactions may occur during and/or after the infusion. Tell your healthcare provider right away if you experience any reactions. Your healthcare provider may slow or stop the infusion or may lower the next dose.
Elevated Transaminases Levels
XENPOZYME may be associated with elevated liver enzymes (known as transaminases) within 24 to 48 hours after infusion. Your doctor should check your liver enzyme levels with a blood test:
- within one month before starting XENPOZYME;
- within 72 hours before any infusion during the dose escalation phase, or before your next scheduled XENPOZYME infusion if you missed a dose.
Based on the results of your blood tests, your doctor may make changes to your dose or infusion schedule. Upon reaching the recommended maintenance dose, your doctor may continue to monitor your liver enzyme levels.
Risk to Unborn Babies
Starting or increasing the dose of XENPOZYME is not recommended in a pregnant female as it may cause harm (birth defects) to the developing baby. If you are pregnant or plan to become pregnant, tell your doctor right away.
If you are a female of reproductive potential, your doctor will verify your pregnancy status before you start treatment with XENPOZYME. You should use effective contraception during XENPOZYME treatment and for 14 days after your last dose if XENPOZYME is discontinued.
- Most frequently reported adverse drug reactions in adults (incidence ≥10%) were headache, cough, diarrhea, low blood pressure, and redness in the eye.
- Most frequently reported adverse drug reactions in pediatric patients (incidence ≥20%) were fever, cough, diarrhea, runny nose, abdominal pain, vomiting, headache, hives, nausea, rash, joint pain, itchy skin, fatigue, and sore throat.
XENPOZYME® (olipudase alfa-rpcp) is indicated for treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients.
Please see full Prescribing Information, including Boxed WARNING, for complete details.