Drug intelligence / Profile preview

risdiplam + itraconazole

Development stage
Unknown
Lead developer
Roche
Modality
Small Molecules
Administration
Oral
01

Overview

**Risdiplam + itraconazole** is a combination of **risdiplam**, an oral small molecule designed as an SMN2 splicing modifier for the treatment of spinal muscular atrophy (SMA), and **itraconazole**, an oral antifungal agent and potent CYP3A inhibitor. Risdiplam works by modifying the alternative splicing of the SMN2 gene, increasing the production of functional SMN protein in patients with SMA. Itraconazole is commonly prescribed for systemic and superficial fungal infections; it is a strong inhibitor of cytochrome P450 3A enzymes. Clinical studies have shown that co-administration of 200 mg itraconazole (twice daily) with risdiplam (single 6 mg dose) does not result in a clinically meaningful change in risdiplam’s pharmacokinetics (only a minor 11% increase in AUC, 9% decrease in Cmax), indicating that only a low fraction of risdiplam is metabolized by CYP3A. No dose adjustment of risdiplam is required when given with itraconazole. This combination is not a co-formulated product; co-administration is primarily relevant for assessing potential drug-drug interactions in clinical practice and trials[1][2][3][5][6].

Other names
risdiplamitraconazole
02

Targets

ISS-N1 (SMN2 intronic splicing silencer N1)CYP3A7 (Cytochrome P450 3A7)CYP51A1 (Sterol 14α-demethylase)

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