Xenpozyme

About olipudase alfa-rpcp

• Xenpozyme received Food and Drug Administration (FDA) approval on August 31st, 2022. 
• Xenpozyme is an enzymatic lysosomal sphingomyelin hydrolysis agent used to treat  non-central nervous system symptoms associated with acid sphingomyelinase deficiency (ASMD) in both adult and pediatric patients.
• Olipudase alfa functions as an enzyme replacement therapy designed to alleviate the indications and manifestations of acid sphingomyelinase deficiency (ASMD) by diminishing the accumulation of sphingomyelin in organs. This reduction aims to mitigate tissue damage experienced by individuals with ASMD.
• ASMD, or acid sphingomyelinase deficiency, is an inherited disorder historically referred to as Niemann-Pick disease types A, A/B, and B. 
• This rare genetic condition results from a deficiency of the enzyme acid sphingomyelinase, crucial for metabolizing the lipid sphingomyelin. 
• ASMD, caused by genetic variants in the SMPD1 gene, manifests with multiorgan symptoms affecting the liver, lungs, spleen, blood, and digestive system. 
• It is a progressive disease with symptoms that can worsen over time, impacting various aspects of life.
• Obtaining advice from a healthcare professional is vital for precise diagnosis and proper management.

Strength: 

Xenpozyme is available as an injection of : 20 mg of olipudase alfa-rpcp as a lyophilized powder in a single-dose vial.

Recommended Dosage:

The prescribed dosages of Xenpozyme for both adults and pediatric patients during the dose escalation and maintenance phases are determined according to body weight. This determination is specifically tailored for individuals with a body mass index (BMI).

Follow aseptic technique for Xenpozyme preparation by reconstituting vials based on the calculated dose. After allowing vials to reach room temperature, add 5.1 mL of Sterile Water for Injection, USP to each vial, gently rolling to create a clear 4 mg/mL solution. Inspect for particulate matter and discoloration. Withdraw the required volume, dilute with 0.9% Sodium Chloride Injection, USP, adjusting for weight categories or fixed total volumes. For infusion, prepare a syringe or bag, gently invert for mixing, and refrain from shaking due to the protein nature of the solution. Vials are single-use; discard any unused solution after preparation.

• For adults, the recommended initial dose of Xenpozyme entails administering 0.1 mg/kg through intravenous infusion. In the maintenance phase, adults are advised to receive a standardized dosage of 3 mg/kg via intravenous infusion every two weeks.
• In the case of pediatric patients, the suggested starting dose is 0.03 mg/kg delivered as an intravenous infusion. Likewise, during the maintenance phase, pediatric patients are encouraged to undergo Xenpozyme treatment with a regular dosage of 3 mg/kg via intravenous infusion every two weeks.

Important:

Prior to commencing Xenpozyme treatment, it is essential to conduct certain preparatory measures. These include obtaining baseline transaminase levels (specifically alanine aminotransferase [ALT] and aspartate aminotransferase [AST]) for all patients within the month preceding the initiation of treatment. Additionally, it is crucial to verify the pregnancy status in females of reproductive potential. In anticipation of Xenpozyme administration, the consideration of pretreatment measures becomes important. This may involve the use of antihistamines, antipyretics, and/or corticosteroids to manage potential side effects or reactions.

Prior to commencing Xenpozyme injections, discuss your medical history and current medications with your healthcare provider. Inform your doctor if you smoke, or consume alcohol. Share a comprehensive list of all medications, including prescriptions and supplements, with your healthcare provider and pharmacist when introducing new medications.

Warnings & Precautions

• Before administering Xenpozyme, consider pre-treatment with antihistamines, antipyretics, and/or corticosteroids to mitigate hypersensitivity reactions. Discontinue Xenpozyme immediately and initiate appropriate medical treatment in the event of a severe hypersensitivity reaction, such as anaphylaxis. 
• Immediate access to medical support is essential, and in severe cases, discontinue Xenpozyme and evaluate the risks before considering re-administration. 
• Infusion-associated reactions (IAR) are potential occurrences during the administration of Xenpozyme. Although pre-treatment measures may be employed to mitigate the risk of such reactions, in the event of severe reactions, discontinuation of Xenpozyme is necessary. 
• Xenpozyme injection may lead to elevated transaminase levels, which should be assessed prior to initiation, within 72 hours before certain infusions, and as part of routine clinical management. It is required to monitor the potential dose adjustments. 
• Pregnant individuals should avoid dosage initiation or escalation due to potential fetal malformations, with pregnancy status verification recommended before treatment. 
• Effective contraception is advised for females of reproductive potential during and after treatment discontinuation. 
• Prioritize discussing pre-existing medical conditions with healthcare providers before initiating Xenpozyme and promptly report any adverse reactions.

Common Xenpozyme Side Effects:

• Headache
• Fever
• Itching
• Nausea and vomiting
• Muscle pain
• Fatigue
• Increased CRP levels
• Urticaria (Hives)
• Abdominal pain
• Cough
• Diarrhea
• Rhinitis
• Hypotension
• Arthralgia
• Pruritus
• Ocular hyperemia
• Anaphylactic reaction
• Severe skin reactions

Use in Specific Population

• Due to potential harm to the developing fetus, initiating or increasing Xenpozyme dosage during pregnancy is strongly discouraged based on animal studies. However, the decision to continue or discontinue Xenpozyme maintenance dosing in pregnancy should consider the woman’s need for the medication, the potential drug-related risks to the fetus, and the potential adverse outcomes from untreated maternal acid sphingomyelinase deficiency (ASMD) disease.
• Before initiating Xenpozyme treatment, it’s crucial to inform your doctor about any plans for pregnancy or if you are currently pregnant. 
• If you are of reproductive age, effective contraception is recommended during treatment and for 14 days after the last dose if Xenpozyme is discontinued. 
• If you are breastfeeding or planning to breastfeed, consult your doctor as the transfer of Xenpozyme into breast milk is uncertain, and there is no available data. Discuss the most suitable feeding approach for your baby while using Xenpozyme. 
• The safety and effectiveness of Xenpozyme in treating non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) have been established in pediatric patients from birth. 
• It’s important to have open communication with your doctor, addressing any concerns to ensure a safe and well-informed treatment plan.

Storage and Handling

• It is imperative to refrigerate Xenpozyme and store it at temperatures between 2°C to 8°C (36°F to 46°F) within the original carton to shield it from light. 
• It is recommended to keep the medicine away from direct light. 
• Do not freeze the medicine. 
• Keep the medicine away from pets and children.