Assegnazione della qualifica di medicinale orfano a Volixibat nella Colangite Biliare Primaria (PBC).

Il medicinale "Volixibat potassio" corrisponde ai criteri di assegnazione della qualifica di medicinale orfano per la seguente indicazione: Trattamento della colangite biliare primaria.

Tale medicinale è iscritto nel registro comunitario dei medicinali orfani sotto il numero EU/3/24/3017.

Sponsor: Mirum Pharmaceuticals International B.V.

Mirum Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to volixibat as a potential treatment for cholestatic pruritus in patients with primary biliary cholangitis (PBC). The regulatory designation is based on the positive interim analysis of the Phase 2b VANTAGE study which showed statistically significant improvement versus placebo in pruritus for patients treated with volixibat. The confirmatory portion of the study is ongoing with completion of enrollment expected in 2026.

Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver.

Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (VISTAS study), and primary biliary cholangitis (VANTAGE study).

In June 2024, Mirum announced positive interim results from the Phase 2b VANTAGE study showing statistically significant improvement in pruritus as well as meaningful reductions in serum bile acids and improvements in fatigue for patients treated with volixibat. No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate.