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General Description:

Olipudase alfa, identified by CAS number 927883-84-9, is a notable compound with significant therapeutic applications. Olipudase alfa is recombinant human acid sphingomyelinase. It is the first and only enzyme replacement therapy in the world for the treatment of Acid Sphingomyelinase Deficiency (ASMD), also known as Niemann–Pick disease. ASMD is a rare lysosomal storage disease caused by mutations in the SMPD1 gene, leading to a deficiency in acid sphingomyelinase and the abnormal accumulation of the primary ASM substrate, sphingomyelin. Olipudase alfa works to hydrolyze sphingomyelin accumulated in body tissues, such as the lungs, liver, spleen, kidneys, and bone marrow. Olipudase alfa gained its first global approval in Japan on March 28, 2022. It was later approved by the European Commission on June 28, 2022 and by the FDA on August 31, 2022.

Indications:

This drug is primarily indicated for: Olipudase alfa is indicated as an enzyme replacement therapy for the treatment of non-Central Nervous System manifestations of Acid Sphingomyelinase Deficiency (ASMD) in pediatric and adult patients with type A/B or type B. Its use in specific medical scenarios underscores its importance in the therapeutic landscape.

Metabolism:

Olipudase alfa undergoes metabolic processing primarily in: Olipudase alfa is a recombinant human enzyme and is expected to be eliminated via proteolytic degradation into small peptides and amino acids. This metabolic pathway ensures efficient processing of the drug, helping to minimize potential toxicity and side effects.

Absorption:

The absorption characteristics of Olipudase alfa are crucial for its therapeutic efficacy: After administration of 3 mg/kg olipudase alfa once every two weeks in adults with ASMD, the mean (percent coefficient of variation, CV%) maximum concentration (C_max) and area under the concentration-time curve over a dosing interval (AUC_0-τ) at steady state were 30.2 µg/mL (17%) and 607 µg.h/mL (20%), respectively. The drug's ability to rapidly penetrate into cells ensures quick onset of action.

Half-life:

The half-life of Olipudase alfa is an important consideration for its dosing schedule: After administration of 3 mg/kg olipudase alfa once every two weeks in adults with ASMD, the mean terminal half-life (t1/2) ranged from 31.9 to 37.6 hours. This determines the duration of action and helps in formulating effective dosing regimens.

Protein Binding:

Olipudase alfa exhibits a strong affinity for binding with plasma proteins: There is no information available. This property plays a key role in the drug's pharmacokinetics and distribution within the body.

Route of Elimination:

The elimination of Olipudase alfa from the body primarily occurs through: There is no information available. Understanding this pathway is essential for assessing potential drug accumulation and toxicity risks.

Volume of Distribution:

Olipudase alfa is distributed throughout the body with a volume of distribution of: After administration of 3 mg/kg olipudase alfa once every two weeks in adults with ASMD, the estimated mean (CV%) volume of distribution of olipudase alfa is 13.1 L (18%). Olipudase alfa is not expected to cross the blood-brain barrier or modulate the CNS manifestations of the disease. This metric indicates how extensively the drug permeates into body tissues.

Clearance:

The clearance rate of Olipudase alfa is a critical factor in determining its safe and effective dosage: After administration of 3 mg/kg olipudase alfa once every two weeks in adults with ASMD, the mean (CV%) clearance of olipudase alfa is 0.331 L/h (22%). It reflects the efficiency with which the drug is removed from the systemic circulation.

Pharmacodynamics:

Olipudase alfa exerts its therapeutic effects through: Olipudase alfa is an enzyme replacement therapy that works to ameliorate the signs and symptoms of ASMD by reducing the amount of sphingomyelin that accumulates in organs and causes tissue damage in patients with ASMD. It works to reduce the extent of non-neurological manifestations of ASMD, such as splenomegaly and hepatomegaly. The drug's ability to modulate various physiological processes underscores its efficacy in treating specific conditions.

Mechanism of Action:

Olipudase alfa functions by: ASMD is an autosomal recessive genetic disorder caused by different mutations in the _SMPD1_ gene that encodes acid sphingomyelinase. Historically, ASMD has been called Niemann-Pick disease (NPD), with different classifications based on disease onset and severity. NPD type A (NPD-A) refers to the severe early-onset form, infantile neurovisceral ASMD and NPD type B (NPD-B) is referred to as the later-onset, chronic visceral form of ASMD. Chronic neurovisceral ASMD (NPD-A/B) is a phenotype with intermediate severity. ASMD has a broad spectrum of disease severity and neurological and non-neurological manifestations; thus, it is difficult to classify different types of ASMD using the former classification system of ASMD (A, B, A/B). Acid sphingomyelinase typically breaks down metabolically-related lipids such as sphingomyelin in various cell types, such as the monocytes, macrophages, and hepatocytes. The deficiency of acid sphingomyelinase thus leads to the accumulation of these lipids in body tissues, causing progressive cell and tissue damage and impairing organ functioning. Olipudase alfa is recombinant human acid sphingomyelinase that hydrolyzes sphingomyelin (SM), preventing its accumulation in body organs. As an enzyme replacement therapy, it is an exogenous source of acid sphingomyelinase. This mechanism highlights the drug's role in inhibiting or promoting specific biological pathways, contributing to its therapeutic effects.

Toxicity:

Classification:

Olipudase alfa belongs to the None, classified under the direct parent group Peptides. This compound is a part of the Organic Compounds, falling under the Organic Acids superclass, and categorized within the Carboxylic Acids and Derivatives class, specifically within the Amino Acids, Peptides, and Analogues subclass.

Categories:

Olipudase alfa is categorized under the following therapeutic classes: Amino Acids, Peptides, and Proteins, Enzyme Replacement Therapy, Enzymes, Enzymes and Coenzymes, Esterases, Hydrolases, Phosphoric Diester Hydrolases, Proteins, Recombinant Proteins. These classifications highlight the drug's diverse therapeutic applications and its importance in treating various conditions.

Experimental Properties:

Further physical and chemical characteristics of Olipudase alfa include:

• Molecular Weight: 63590.933
• Molecular Formula: C2900H4373N783O791S24