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We strive today for
your child's tomorrow.
We strive today for
your child's tomorrow.


July 1, 2019

Asklepion Pharmaceuticals concludes enrollment of Phase III trial: L-citrulline for Prevention of Sequelae of Acute Lung Injury in Pediatrics Undergoing Cardiopulmonary Bypass for Heart Defects

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April 26, 2019

Key Patent Coverage for Asklepion Pharmaceuticals’ Investigational Candidate Extended to 2036

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December 19, 2017

Asklepion Pharmaceuticals Announces Special Protocol Assessment (SPA) Agreement with FDA for Phase 3 Program of Intravenous Citrulline

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December 8, 2017

Asklepion Pharmaceuticals Presents Phase 1b/2a Clinical Trial Data for IV-Citrulline at the Pediatric Cardiac Intensive Care Society 13th Annual International Meeting

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January 9, 2017

Asklepion Pharmaceuticals announces receipt of Orphan Drug Designation from the U.S. FDA for the prevention of clinical sequelae of acute lung injury induced by cardiopulmonary bypass in pediatric patients.

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June 9, 2016

Asklepion Pharmaceuticals sells its U.S. rights, titles, and ownership of ursodeoxycholic acid suspension to Retrophin.

May 17, 2016

Asklepion Pharmaceuticals is a recipient of the 2016 NORD Industry Innovation Award. The Rare Impact Awards, presented by the National Organization for Rare Disorders (NORD)®, recognizes the people, organizations, advocates and companies who are making a difference in the fight against rare diseases.

March 18, 2015

Retrophin (Nasdaq: RTRX) exercises its right to buy all rights to Cholbam® (cholic acid) from Asklepion Pharmaceuticals.

March 17, 2015

FDA approves Cholbam® (cholic acid) capsules, the first FDA approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for patients with peroxisomal disorders (including Zellweger spectrum disorders).

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March 17, 2015

FDA grants Asklepion Pharmaceuticals, LLC a Rare Pediatric Disease Priority Review Voucher which entitles the Company to designate a single human drug application or a single biologic application as qualifying for a priority review by the agency.

January 12, 2015

Asklepion Pharmaceuticals, LLC and Retrophin (Nasdaq: RTRX) announce the signing of a definitive agreement under which Retrophin has acquired the exclusive right to purchase from Asklepion, all worldwide rights, titles, and ownership of cholic acid for the treatment of bile acid synthesis defects, if approved by the U.S. Food and Drug Administration (FDA).

April 16, 2014

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommends the authorization of Kolbam® for the ‘treatment of inborn errors of primary bile acid synthesis, in infants from one month of age for continuous lifelong treatment through adulthood, encompassing the following single enzyme defects: Sterol 27-hydroxylase (presenting as cerebrotendinous xanthomatosis, CTX) deficiency; 2- (or α-) methylacyl-CoA racemase (AMACR) deficiency; Cholesterol 7 α-hydroxylase (CYP7A1) deficiency.’

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November 21, 2013

Asklepion Pharmaceuticals announces the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Cholbam® (cholic acid), an investigational medicine, for the treatment of bile acid synthesis disorders due to single enzyme defects and as adjunctive treatment of peroxisomal disorders including Zellweger spectrum disorders in patients who exhibit manifestations of liver disease, steatorrhea or complications from decreased fat soluble vitamin absorption.

April 26, 2012

Asklepion Pharmaceuticals completes enrollment for the CIT-002-02 clinical trial in the surgical repair of a large atrial septal defect and/or a large unrestrictive ventricular septal defect, or a partial or complete atrioventricular septal defect, in pediatric patients. The trial will evaluate the pharmacokinetics and safety of a revised protocol of intravenous L-citrulline versus placebo in children undergoing cardiopulmonary bypass.

January 10, 2011

Asklepion Pharmaceuticals completes a study for the comparative bioavailability of three formulations of cholic acid in healthy male subjects using a multiple dose repeated measures approach in treatment of inborn errors of bile acid synthesis.

August 23, 2010

Asklepion Pharmaceuticals completes an open label, single center, non-randomized study to compare the therapeutic efficacy of cholic acid capsules with that of the currently used formulation of cholic acid capsules used to treat children with inborn errors of bile acid synthesis to assess the therapeutic efficacy and safety and tolerability of cholic acid capsules.

October 23, 2006

Asklepion Pharmaceuticals obtains right to exclusively market URSOFALK™ Suspension (ursodeoxycholic acid) in the United States from Dr. Falk Pharma GmbH.

July 14, 2006

Asklepion Pharmaceuticals obtains exclusive license from Vanderbilt University for intravenous L-citrulline.

March 8, 2006

Asklepion Pharmaceuticals, LLC is founded to give a future to patients who previously had none. The Company transitions a 15-year academic intent into a commercial program to develop cholic acid for inborn errors of bile acid metabolism.