science

Olipudase alfa

Olipudase alfa is a science topic covered in the lgStudy science library. This page brings together a partial reference excerpt, illustrations, worked examples, real-world applications and a short study plan, so you can understand Olipudase alfa rather than just read about it. In short: Olipudase alfa, sold under the brand name Xenpozyme, is a medication used for the treatment of non-central nervous system (CNS) manifestations of acid sphingomyelinase deficiency type A/B or type B. The most common side events include infections, infusion-related reactions, or gastrointestinal complaints (disease signs and symptoms in children).

Key takeaways

Olipudase alfa belongs to science; place it in that map before memorising details.
Learn the definition first, then one example that makes the definition concrete.
Connect Olipudase alfa to a quantity you can measure, compute or draw — that is where exam questions come from.
Reproduce the core statement of Olipudase alfa from memory before moving on to harder problems.

Reference excerpt

Olipudase alfa, sold under the brand name Xenpozyme, is a medication used for the treatment of non-central nervous system (CNS) manifestations of acid sphingomyelinase deficiency type A/B or type B. The most common side events include infections, infusion-related reactions, or gastrointestinal complaints (disease signs and symptoms in children). Historically referred to as Niemann-Pick disease types A (NPD A) and B (NPD B), acid sphingomyelinase deficiency is a genetic disorder. It belongs to the larger family of metabolic disorders called lysosomal storage diseases, in which fats build up within the parts of the body's cells that break down nutrients and other materials. This affects the way cells work and causes them to die, affecting normal functioning of tissues and organs. Acid sphingomyelinase deficiency is seriously debilitating and life-threatening since the build-up of fatty substances can cause brain damage and swelling of organs such as liver and spleen. Xenpozyme is the first acid sphingomyelinase deficiency-specific treatment. The replacement enzyme is produced by a method known as recombinant DNA technology: it is made by cells into which a gene (DNA) has been introduced, that enables them to produce the enzyme. Olipudase alfa was approved for medical use in Japan in March 2022, in the European Union in June 2022, and in the United States in August 2022. The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.

Medical uses Olipudase alfa is indicated as an enzyme replacement therapy for the treatment of non-central nervous system manifestations of acid sphingomyelinase deficiency in people with type A/B or type B. Olipudase alfa is an enzyme replacement therapy, developed to replace patients' deficient or defective enzyme, acid sphingomyelinase, and thereby reduce fat accumulation within cells and relieve some of the symptoms of the disease. Xenpozyme was approved by the European Medicines Agency for the treatment of acid sphingomyelinase deficiency type A/B or type B, and by the US FDA for the treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency.

Society and culture Olipudase alfa is the international nonproprietary name (INN).

Legal status Olipudase alfa was approved for medical use in Japan in March 2022. In May 2022, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Xenpozyme, intended for the treatment of non-central nervous system (CNS) manifestations of acid sphingomyelinase deficiency type A/B or type B. Xenpozyme was reviewed under the accelerated assessment program of the European Medicines Agency (EMA). The applicant for this medicinal product is Genzyme Europe BV. Olipudase alfa was approved for medical use in the European Union in June 2022.

References

Further reading Diaz GA, Jones SA, Scarpa M, Mengel KE, Giugliani R, Guffon N, et al. (August 2021). "One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency". Genet Med. 23 (8): 1543•1550. doi:10.1038/s41436-021-01156-3. PMC 8354848. PMID 33875845. Maines E, Franceschi R, Rizzardi C, Deodato F, Piccoli G, Gragnaniello V, et al. (2022). "Atherogenic lipid profile in patients with Niemann-Pick disease type B: What treatment strategies?". J Clin Lipidol. 16 (2): 143–154. doi:10.1016/j.jacl.2022.01.008. PMID 35181260. S2CID 246974107. Wasserstein MP, Diaz GA, Lachmann RH, Jouvin MH, Nandy I, Ji AJ, et al. (September 2018). "Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months". J Inherit Metab Dis. 41 (5): 829•838. doi:10.1007/s10545-017-0123-6. PMC 6133173. PMID 29305734.

Worked examples

Example 1 — a first encounter with Olipudase alfa

Start with the simplest possible case. Write down what Olipudase alfa claims or describes in one sentence, then invent the smallest concrete situation in which that sentence is true. In science, the smallest case is usually a single object, a single equation or a single measurement. Check that every symbol or term in your sentence has a meaning in that case.

Example 2 — changing one variable

Take the situation from Example 1 and change exactly one quantity: double it, halve it, or set it to zero. Predict what should happen to Olipudase alfa before you calculate. Comparing your prediction with the result is the fastest way to find out whether you understand the idea or only the words.

Example 3 — an exam-style question

Typical questions about Olipudase alfa ask you to (a) state it precisely, (b) apply it to given data, and (c) explain a limitation. Practise writing all three answers in under five minutes; the third part is what separates a full-mark answer from an average one.

Applications of Olipudase alfa

In research: Olipudase alfa appears in science research whenever the underlying quantities have to be modelled precisely. Papers usually cite it as a starting assumption and then explore where it breaks down.
In technology and industry: Engineering practice reuses Olipudase alfa in design rules, simulations and safety margins. Knowing the idea lets you read a specification sheet and understand why the numbers look the way they do.
In the classroom: Olipudase alfa is common in secondary-school and first-year university syllabi. It links to neighbouring topics Orphan drugs, Sanofi, so understanding it makes those chapters shorter.
In everyday life: Look for Olipudase alfa outside the textbook — in sport, cooking, traffic, electronics or the sky above you. An example you found yourself is remembered far longer than one you were given.

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How to study Olipudase alfa in 20 minutes

Read the reference excerpt below once, without taking notes.
Close the page and write down what Olipudase alfa means in your own words.
Compare your version with the excerpt and mark what you missed.
Work through the three examples above with pen and paper.
Explain Olipudase alfa out loud to somebody else — or to Teacher Smith in the lgStudy chat.

Frequently asked questions

What is Olipudase alfa in simple terms?

Why does Olipudase alfa matter?

Because it connects several science ideas at once: it gives you a definition you can apply, a quantity you can calculate, and a way to check whether a result is plausible.

How should I study Olipudase alfa?

Read the excerpt, restate it from memory, then work through the examples and applications listed on this page. The five-step study plan above takes about twenty minutes.

What does this page cover?

It gives you a compact reference excerpt plus original lgStudy explanations, examples, applications and study material on Olipudase alfa.