Endocrine and Metabolic Disease Clinical Development Expertise

Overcoming complexities to bring more metabolic disease treatments to market successfully

Metabolic diseases are a rising therapy area focus for clinical research due to high demand for novel therapies that are challenged with proving safety and efficacy. The development process is complicated and full of unknowns, and even the best metabolic therapeutic might not make it to the patients that need it, not because it does not work but because the design, data collection, and analysis were not done right.

At Veristat, we have assembled an extraordinary team of scientific-minded experts who have mastered the complexities of running metabolic disease trials. Our team has supported >180 clinical trials for endocrine/metabolic disorders and the preparation of 15 marketing applications - 13 of which have been approved so far!

Success with Lifestyle, Genetic, and Rare Metabolic Therapies

We strive to advance your novel metabolic therapies from Phase I-III clinical development to market with confidence.

Our experienced teams are poised to plan and implement these efforts quickly, providing:

Development of regulatory strategy, expedited pathways, and regulatory agency interactions
Clinical program planning, inclusive of statistical planning and analysis
Agile patient recruitment strategies to support virtual trial and hybrid approaches
Rapid deployment of clinical trial databases and data analysis and migration into CDISC formats
Virtual and remote clinical and medical site monitoring
Writing of clinical trial, safety, and regulatory documents
Preparation and defense of Marketing Applications – NDAs, BLAs, NDSs, MAAs, jNDAs, etc.
Post-Marketing Pharmacovigilance and Safety Monitoring to ensure patient safety beyond regulatory approval

Proven Expertise for Success
	> 180 Endocrine/Metabolic Trials

	80% are Rare Disorders

	> 70% are Genetic Disorders

Get the right strategic consultation, clinical program implentation, regulatory approval registration, and post-marketing support for endocrine and metabolic therapies based upon our extensive first-hand knowlege.

Breadth of Therapy Indications
Achondropasia	Gaucher Disease	Lipodystrophy
Adrenoleukodystrophy (ALD) and Thalassemia	Generalized Lipodystrophy	Lysosomal Acid Lipase (LAL) Deficiency
Alpha-mannosidosis (AM)	Gout (Hyperuricemia)	Maroteaux-Lamy Syndrome
Anorexia	Growth Hormone Deficiencies/Abnormalities	Molybdenum Cofactor Deficiency
Bardet-Biedl Syndrome and Alstrom Syndrome	Hereditary ATTR (hATTR) Amyloidosis	Mucopolysaccharidosis type IIIB
Cerebral Adrenoleukodystrophy	Homocystinuria	Multiple Lysosomal Storage Disorders
Chronic Pancreatitis	Homozygous Familial Hypercholesterolemia (HoFH)	Non-Alcoholic Steatohepatitis (NASH)
Congenital Adrenal Hyperplasia	Hypercholesterolemia	Obesity
Diabetes	Hyperoxaluria	Pharmacodynamic Markers
Dyslipidemia	Hypertriglyceridemia	POMC deficiency
Fabry Disease	Hypoleptinemic Dysmetabolic Disorder	Pompe Disease
Familial Amyloid Polyneuropathy	Hypoparathyroidism	Pyruviate Kinase Deficiency
Familial Chylomicronemia Syndrome (FCS)	Immunoglobulin A (IgA) Nephropathy	Vasomotor symptoms (VMS)

Learn More with These Resources

Case Study

Preparing Data for a Marketing Authorization Application ...

Read the case study to learn how we helped a biotech prepare the MAA submission for a recently acquired product. Our creative approach included utilizing one DB for ...

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Conference Presentation

Turning Sponsor/CRO Relationships into Partnerships for ...

Get the presentation that was given at the Evolution Summit Fall 2019. Veristat and thier client Ascendis Pharma share thier thoughts on how to truly work as a ...

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