Afinitor Disperz Approved to Treat TSC-Associated Partial-Onset Seizures

Afinitor Disperz is now the first approved drug therapy for this indication
Afinitor Disperz is now the first approved drug therapy for this indication

Novartis announced that the Food and Drug Administration (FDA) has approved Afinitor Disperz (everolimus tablets for oral suspension) for the adjunctive treatment of patients aged ≥2 years with tuberous sclerosis complex (TSC)-associated partial-onset seizures. 

This approval marks Afinitor Disperz as the first approved treatment for this condition. Afinitor Disperz is already approved to treat TSC-associated subependymal giant cell astrocytoma (SEGA) in patients aged ≥1 year that require therapeutic intervention but cannot be curatively resected.

The approval was based on the Phase 3, randomized, double-blind, placebo-controlled EXIST-3 (EXamining everolimus In a Study of TSC) study which evaluated adjunctive Afinitor Disperz (low exposure: 3–7ng/mL; high exposure: 9–15ng/mL) in patients with treatment-resistant TSC-associated partial-onset seizures, defined as inadequate control of partial-onset seizures despite use of ≥2 sequential regimens of single or combined anti-epileptic drugs. 

In the study, adjunctive treatment with Afinitor Disperz demonstrated a significant benefit by reducing the frequency of treatment-resistant seizures associated with TSC vs placebo. There was a greater median percentage reduction from baseline in seizure frequency among patients randomized to Afinitor Disperz low exposure (29.3%, 95% CI: 18.8, 41.9; P=0.003) as well as high exposure (39.6%, 95% CI: 35.0, 48.7; P<0.001) vs placebo (14.9%, 95% CI: 0.1, 21.7). 

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In addition, seizure response rate (≥50% reduction) was also greater with Afinitor Disperz low exposure (28.2%, 95% CI: 20.3, 37.3) and high exposure (40.0%, 95% CI: 31.5, 49.0; P<0.001) vs placebo (15.1%, 95% CI: 9.2, 22.8). 

Stomatitis, diarrhea, nasopharyngitis, upper respiratory tract infection, and pyrexia were the most common adverse events observed in the Afinitor Disperz arms. 

Afinitor Disperz, a mammalian target of rapamycin (mTOR) inhibitor, is available as 2mg, 3mg, and 5mg strength tablets for oral suspension in 28-count blisters. 

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