This way forward to proven results in children

RESULTS IN CHILDREN

AFTER 1 YEAR

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:

Lung icon with text stating 46% improvement in lung function in 3 patients as measured by DLco

Spleen icon with text stating 47% reduction in spleen volume in 8 patients

Liver icon with text stating 38% reduction in liver volume in 8 patients

Platelets icon with text stating 38% increase in platelet count in 7 patients

Icon of small child with text stating XENPOZYME improved growth in children experiencing growth delay after 1 year of treatment. This was measured by height Z-scored. The average improvement in height Z-scores from baseline was 0.5 in 7 patients.

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.

SAFETY

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%)

Table illustrating the most frequently reported side effects (incidence of 20% or more) in children taking XENPOZYME

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.

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IMPORTANT SAFETY INFORMATION AND INDICATION

WARNING: SEVERE ALLERGIC REACTIONS

Allergic Reactions Including Anaphylaxis
Allergic reactions, including severe reactions that may be serious or life-threatening (known as anaphylaxis), have occurred during and after XENPOZYME treatment. Tell your healthcare provider right away if you develop any reactions, and seek immediate medical care if severe reactions occur. If a severe allergic reaction occurs, your doctor may decide to discontinue XENPOZYME immediately and provide appropriate medical care. Appropriate medical support measures may be administered, and you may require close observation during and after XENPOZYME administration.

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.

ADVERSE REACTIONS

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.

INDICATION
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.