Head of Clinical Development, Rare Diseases @
Sr Medical Director, Clinical Research @ Enobia Pharma (acquired by Alexion)
Member, Scientific Advisory Board @ Fibrous Dysplasia Foundation
- Biotechnology executive with proven leadership skills. Started the R&D group at Retrophin, going from a two-member team to a full organization with 20+ members. Among the accomplishments of the team are five pre-IND meetings with FDA, the successful launch of a phase 2 study and a first in human with a novel compound in less than
- Biotechnology executive with proven leadership skills. Started the R&D group at Retrophin, going from a two-member team to a full organization with 20+ members. Among the accomplishments of the team are five pre-IND meetings with FDA, the successful launch of a phase 2 study and a first in human with a novel compound in less than one year.
One more year passed, and we now have three commercial products, 2 INDs, 2 orphan designations, and a phase 1 and a phase 2 studies ongoing.
- Extensive experience in clinical trial development and management, protocol writing and supervision, including phases 1-3 and post-marketing, pharmacokinetics (PK) and drug-drug interaction (DDI) studies for biologicals and small molecules
-Founder and first Medical Director of the Pediatric Bone Diseases Program (Omaha, NE)
-Participated and presented in numerous meetings with regulatory authorities.
- Global Medical lead for the successful BLA submission for Lumizyme for the ERT of Pompe disease (Genzyme Corporation).
-Member of the Eliglustat team, an orally active glucocerebroside synthase inhibitor for the treatment of Gaucher disease Type 1 (Genzyme Corporation).
-Global Medical Lead for Asfotase alfa for the ERT of hypohposphatasia (Enobia/Alexion Pharmaceuticals).
-Experience and deep understanding of pharmacovigilance.
- Experience in development and maintenance of Disease Registries (Pompe Disease registry, Genzyme Corporation).
- Extensive leadership and team building experience.
- Experience in due diligence procedures.
- Experience in reimbursement negotiations and marketing plans for treatment of rare diseases
- Author of eight book chapters in medical text books and over 50 scientific articles.
- Over the years, developed skills in team leadership and conflict management.
Enzyme Replacement Therapy
Chief Medical Officer @ From May 2013 to Present (2 years 8 months) Adjunt Associate Professor of Pediatrics and Orthopedic Surgery @ Founder and Director (until 2007), Pediatric Metabolic Bone Diseases Clinic, Children's Hospital, Omaha, NE From 2002 to Present (13 years) Executive Medical Director, Clinical Research @ From 2012 to May 2013 (1 year) Sr Medical Director, Clinical Research @ Medical director for 12 clinical research studies, involved in pipeline development and interdepartmental activities. From 2010 to 2012 (2 years) Medical Director Clinical Research @ Clinical Research, Lysosomal Storage Diseases. Approval of Lumizyme. From 2007 to 2010 (3 years) Medical Director, Pharmacovigilance @ From 2007 to 2008 (1 year) Fellow, Orthopedic Surgery (Metabolic Bone Diseases) @ Shriners Hospital, Montreal From 1997 to 2002 (5 years) Fellow, Endocrinology @ Adult osteoporosis, PTHrp From 1996 to 1997 (1 year) Fellow, Metabolic Bone Diseases @ Pediatric and adult osteoporosis From 1993 to 1996 (3 years) Fellow, Pediatric Endocrinology @ From 1992 to 1993 (1 year) Resident, Pediatrics @ From 1988 to 1992 (4 years)
MD @ University of Buenos Aires School of Medicine From 1982 to 1987 Horacio Plotkin is skilled in: Clinical Research, Clinical Trials, Biotechnology, Medicine, Endocrinology, Pharmacovigilance, Pharmaceutical Industry, Clinical Development, Pediatrics, Drug Development, Immunology, Hospitals, Cross-functional Team Leadership, Medical Affairs, Creative Problem Solving, Clinical Research Studies development and management, Orthopedics, Infectious Diseases
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