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Mirum Pharmaceuticals to Acquire Bluejay Therapeutics, Strengthening Rare Disease Portfolio

Shots: Mirum Pharmaceuticals has entered into an agreement to acquire Bluejay Therapeutics, gaining global rights to brelovitug with Breakthrough Therapy and PRIME designations, and expanding Mirum’s rare liver disease portfolio As per the deal, Mirum will acquire all Bluejay shares for $250M in cash and $370M in Mirum stock, plus up to $200M in sales-based…

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Mirum Pharmaceuticals

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 PBO Ref: Prnewswire | Image: Mirum Related…

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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…

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