Alpelisib

What it is and how it works

Mechanism of Action Alpelisib is an inhibitor of phosphatidylinositol-3-kinase (PI3K) with inhibitory activity predominantly against PI3Kα. Gain-of-function mutations in the gene encoding the catalytic α-subunit of PI3K (PIK3CA) lead to activation of PI3Kα and Akt-signaling, cellular transformation and the generation of tumors in in vitro and in vivo models. Activating mutations in PIK3CA have been found to induce a spectrum of overgrowths and malformations comprising a wide group of clinically recognizable disorders commonly known as PROS.

Indications

is indicated for the treatment of adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). VIJOICE is a kinase inhibitor indicated for the treatment of adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Dosage and administration (summary)

Recommended Dose: Pediatric patients (2 to less than 18 years of age): 50 mg taken orally once daily with food. Adult patients: 250 mg taken orally once daily with food. Administration: Swallow tablets whole. Do not chew, divide, or crush. For patients who cannot swallow tablets whole, use tablets or oral granules to create a suspension or a mixture. VIJOICE suspension made with water can be administered orally or via a nasogastric or gastric tube. See full prescribing information for preparation and administration instructions.

Contraindications

is contraindicated in patients with severe hypersensitivity to alpelisib or any of its ingredients . Severe hypersensitivity to VIJOICE or to any of its ingredients.

Warnings and precautions

Severe Hypersensitivity : Permanently discontinue VIJOICE. Promptly initiate appropriate treatment. Severe Cutaneous Adverse Reactions (SCARs) : VIJOICE can cause SCARs, including Stevens-Johnson syndrome (SJS), erythema multiforme (EM), toxic epidermal necrolysis (TEN), and drug reaction with eosinophilia and systemic symptoms (DRESS). Interrupt VIJOICE for signs or symptoms of SCARs. Permanently discontinue VIJOICE if SCARs are confirmed.

Pregnancy

Pregnancy Risk Summary Based on animal data and mechanism of action, VIJOICE can cause fetal harm when administered to a pregnant woman . There are no available data in pregnant women to inform the drug-associated risk.

Storage and supply

(alpelisib) 50 mg, 125 mg, and 200 mg film-coated tablets are available in blister packs based on daily dose as described in Table 13. Table 13: VIJOICE Tablets Daily Dose Blister Packs Daily dose Each carton contains Each child-resistant blister pack contains Appearance of tablet NDC 50 mg daily dose One 28-day supply blister pack 28 tablets: 50 m…

Source: Alpelisib (Vijoice) — US FDA prescribing information, 2025-11-04, DailyMed. Summarised and shortened; the full label governs. Iranian registration, indications and labelling may differ.