Kristina Haack
Senior Global Head Clinical Development Inherited Neurometabolic Diseases Sanofi
Kristina An Haack, MD, is Senior Global Head of Clinical Development for Inherited Neurometabolic Diseases at Sanofi, where she leads the clinical strategy and development of therapies for rare genetic and metabolic disorders. A paediatrician by training, she has dedicated her career to inherited metabolic diseases and other rare paediatric conditions, spanning clinical practice, academic research, and pharmaceutical development. With more than 25 years of experience across academia, research, drug development, and project leadership, Dr. An Haack is recognized for advancing innovative therapies for underserved patient populations. She also serves as a leader within Sanofi’s Pediatric Medicines Network, championing the development of better treatments for children living with rare diseases.
Seminars
As the landscape of therapeutic approaches for muscular disorders expands, so must the endpoints used to assess them. Demonstrating meaningful clinical benefit remains one of the biggest barriers to successful approval, and while functional assessments continue to play an important role, sponsors are exploring novel biomarkers, digital measures and disease-specific endpoints that can provide earlier, more sensitive evidence of treatment effect. Attendees will gain practical insights into endpoint selection, validation and implementation from experts navigating today’s evolving regulatory landscape.
This workshop will:
- Explore the shortfalls of traditional functional assessments such as the 6-minute walk test and the North Star Ambulatory Assessment to identify the key challenges like variability in a largely heterogeneous patient population
- Discuss aligning endpoint selection with disease biology to demonstrate clinically meaningful benefits
- Discuss novel strategies including digital endpoints to detect treatment effects earlier and generate evidence that supports regulatory decision-making
- Evaluating the use of validated biomarkers and surrogate endpoints as earlymindicators of treatment benefit to generate rapid evidence for regulatory bodies
- Exploring regulatory expectations for novel endpoint qualification in rare muscular disorders
- Learning from successful accelerated approval case studies to identify best practices and increase the likelihood of regulatory success
