Mirum Pharmaceuticals Receives the US FDA Approval for Ctexli to Treat Cerebrotendinous Xanthomatosis

Shots:The US FDA has approved Ctexli (chenodiol) to treat cerebrotendinous xanthomatosis (CTX) in adults Efficacy was established in crossover withdrawal P-III (RESTORE) trial assessing Ctexli (250mg, PO, TID) vs PBO in pts for 24wks. Study showed reduced levels of plasma cholestanol & urine 23S-pentol in pts compared to PBORef: Prnewswire | Image: MirumRelated…

ByRidhi RastogiFebruary 24, 2025

INSIGHTS+

Know Your Investor: BVF Partners L.P. (September’24 Edition)

Shots: An equity hedge fund manager company, BVF Partners invests in small cap and early-stage biotechnology companies In 2023, BVF participated in five investment rounds and added 20 companies to its portfolio For a curated report on a specific investor or venture capital, reach out to us at connect@pharmashots.com Established in 1993 in San Francisco, California,…

ByPrince GiriSeptember 5, 2024

NEWS

Mirum Pharmaceuticals’ Livmarli (maralixibat) Receives the CHMP’s Positive Opinion for Progressive Familial Intrahepatic Cholestasis (PFIC)

Shots: The CHMP has granted positive opinion to Livmarli (oral solution) for PFIC patients (age: ≥3mos.) along with COMP for maintaining ODD. EC’s decision is anticipated in Q3’24 The opinions were supported by the P-III (MARCH) trial involving 93 patients with PFIC types (PFIC1, PFIC2, PFIC3, PFIC4, PFIC6 & unidentified mutational status), showing decreased…

ByDipanshu DixitMay 31, 2024

Mirum Pharmaceuticals Receives the US FDA Approval for Ctexli to Treat Cerebrotendinous Xanthomatosis

Know Your Investor: BVF Partners L.P. (September’24 Edition)

Mirum Pharmaceuticals’ Livmarli (maralixibat) Receives the CHMP’s Positive Opinion for Progressive Familial Intrahepatic Cholestasis (PFIC)

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