Business Details
20 Years
experience
Your Trusted Partner for Pediatric Rare Disease Therapies
Asklepion Pharmaceuticals is a biopharmaceutical company dedicated to addressing critical unmet medical needs in pediatric rare diseases. Founded in 2006, we focus on discovering, developing, and commercializing life-saving therapies for children with limited or no treatment options. Our work is driven by a commitment to scientific rigor, ethical standards, and collaboration with universities, pharmaceutical partners, and financial assistance programs to ensure FDA-approved treatments reach patients.We achieved a significant milestone with FDA approval for an oral therapy targeting bile acid synthesis defects, reflecting our expertise in navigating complex regulatory pathways. Our team in Baltimore, Maryland, combines decades of experience in drug development to advance a pipeline focused on rare and critical pediatric conditions, prioritizing accessibility and quality of life improvements for patients and families.
Services
IV Citrulline
Acute Lung Injury Treatment
Rare Disease Therapies
Pediatric Clinical Trials
Bile Acid Synthesis Defects
Orphan Drug Development
Details
- Year established: 2006
Highlights and features
- We focus exclusively on life-saving treatments for rare pediatric diseases
- Our team has secured FDA approval for critical therapies
- We partner with leading universities and pharmaceutical companies
- We received the NORD Rare Impact Award for our contributions
- We prioritize ethical standards and accessibility in drug development
Services
IV Citrulline
IV Citrulline is an investigational treatment for preventing acute lung injury complications in pediatric heart surgery patients. Our Phase III clinical trial demonstrated its potential to improve post-operative outcomes. The therapy received Special Protocol Assessment from the FDA and patent protection through 2036.
Rare Disease Therapies
We develop targeted therapies for rare pediatric conditions with no existing treatment options. Our pipeline focuses on critical diseases requiring urgent medical intervention. Our scientific team applies advanced biotechnology to address unmet clinical needs in small patient populations.
Clinical Trials
Our clinical trial programs evaluate novel treatments for rare pediatric diseases through rigorous, FDA-regulated studies. We collaborate with leading medical institutions to conduct trials for conditions like acute lung injury and metabolic disorders. Our research follows strict ethical guidelines and scientific standards.
Orphan Drug Development
We specialize in orphan drug development for diseases affecting fewer than 200,000 patients. Our team navigates complex regulatory pathways to bring therapies to market. We maintain partnerships to ensure commercialization and accessibility of approved treatments for rare conditions.
Questions
What does Asklepion Pharmaceuticals specialize in?
We specialize in developing treatments for rare pediatric diseases with limited therapeutic options, focusing on life-saving therapies for children.
What clinical trials are currently active?
We are conducting Phase III trials for IV Citrulline to prevent acute lung injury sequelae in pediatric cardiac surgery patients.
Has Asklepion received regulatory approvals?
Yes, we have FDA approval for an oral treatment targeting bile acid synthesis defects and hold orphan drug designations.
How does Asklepion collaborate with other organizations?
We partner with universities, pharmaceutical companies, and financial assistance programs to advance drug development and accessibility.
What recognition has Asklepion received?
We received the 2016 NORD Rare Impact Award for our contributions to rare disease research and development.
