Asklepion Pharmaceuticals
Asklepion Pharmaceuticals is a Baltimore-based biopharmaceutical company dedicated to discovering, developing, and commercializing treatments for rare pediatric diseases. Their mission is to help patients with little to no therapeutic options due to the rarity of their conditions, focusing on life-saving therapies and improving quality of life for children. They have a pipeline that includes clinical trials for conditions such as acute lung injury induced by cardiopulmonary bypass and are engaged in research and development to address unmet medical needs in rare diseases.
Industries
Nr. of Employees
small (1-50)
Patents
Products
Oral therapy for bile acid synthesis disorders
An FDA-approved oral treatment indicated for inborn errors of bile acid synthesis and as adjunctive treatment in certain peroxisomal disorders; developed for pediatric and adult patients with these metabolic conditions.
Intravenous citrulline
Intravenous citrulline in development for acute lung injury induced by cardiopulmonary bypass and acute sickle cell crisis.
Cholbam
Cholbam (cholic acid) is Asklepion Pharmaceutical's first product listed in the U.S. Food and Drug Administration's Novel Drug Summary for 2015.
Oral therapy for bile acid synthesis disorders
An FDA-approved oral treatment indicated for inborn errors of bile acid synthesis and as adjunctive treatment in certain peroxisomal disorders; developed for pediatric and adult patients with these metabolic conditions.
Intravenous citrulline
Intravenous citrulline in development for acute lung injury induced by cardiopulmonary bypass and acute sickle cell crisis.
Cholbam
Cholbam (cholic acid) is Asklepion Pharmaceutical's first product listed in the U.S. Food and Drug Administration's Novel Drug Summary for 2015.
Services
Sponsor-led design and execution of pediatric clinical studies including enrollment, site coordination, safety monitoring, and data collection for Phase 1b/2a and Phase 3 programs.
Regulatory planning and engagement including obtaining orphan-drug designation, securing Special Protocol Assessments, and preparing submissions to regulatory authorities.
Development and clinical evaluation of oral and IV formulations, including comparative bioavailability and PK studies to support dosing and safety assessments.
Sponsor-led design and execution of pediatric clinical studies including enrollment, site coordination, safety monitoring, and data collection for Phase 1b/2a and Phase 3 programs.
Regulatory planning and engagement including obtaining orphan-drug designation, securing Special Protocol Assessments, and preparing submissions to regulatory authorities.
Development and clinical evaluation of oral and IV formulations, including comparative bioavailability and PK studies to support dosing and safety assessments.
Expertise Areas
- Pediatric rare disease drug development
- Clinical trial management (pediatric, Phase I-III)
- Formulation development (IV and oral)
- Clinical pharmacokinetics and bioavailability studies
Key Technologies
- Intravenous formulation development
- Oral formulation development
- Pharmacokinetic (PK) analysis
- Comparative bioavailability studies
News & Updates
Asklepion Pharmaceuticals was awarded a 2016 Rare Impact Award by the National Organization for Rare Disorders (NORD).
Asklepion Pharmaceuticals was awarded a 2016 Rare Impact Award by the National Organization for Rare Disorders (NORD).