FSH Society Releases White Paper on Clinical Trial Readiness

New publication details findings from an industry collaborative workshop on facioscapulohumeral muscular dystrophy

The FSH Society today announced that it has published a report on its recent workshop with government regulators, biopharma companies, and academic thought leaders to discuss the current state of clinical trial preparedness for facioscapulohumeral muscular dystrophy (FSHD). The Society is the world’s largest research-focused patient advocacy organization for FSHD, a hereditary muscle-damaging condition that affects an estimated one million men, women, and children worldwide. There is currently no drug to treat or cure FSHD.

In seeking to speed up the development of treatments and a cure for FSHD, the FSH Society convened the workshop to identify gaps in clinical trial readiness that may delay or hamper existing therapeutic programs. The meeting was moderated by Dr. John Porter, former program director for neuromuscular disease at the National Institute of Neurological Disorders and Stroke (NINDS) and addressed five major areas of interest to the pharmaceutical industry. These included tractability of FSHD as an indication suitable for therapeutic development, status of patient registries and natural history studies, pharmacodynamic biomarkers, imaging biomarkers and clinical trial outcome measures.  For each session, the existing status of each area was presented by key opinion leaders and was followed by extensive discussions with all stakeholders, including academics, pharmaceutical companies and patient advocacy groups with regulatory agencies providing critical feedback and guidance on strategies for consideration.

“With the increasing number of therapeutics in development for FSHD, the timing of this meeting couldn’t have been more appropriate”, said Mark Stone, President and CEO of the FSH Society. “The aforementioned gaps impact all therapeutic programs and represent pre-competitive hurdles that can hamper clinical trials and potential treatments for our families.  Moreover, the identification of these perceive gaps provide an opportunity for the FSH Society, in concert with our industry partners, academic collaborators, sister patient advocacy groups and patient community to mobilize resources to address them for the benefit of the entire community.”

Jamshid Arjomand, PhD, Chief Science Officer of the FSH Society added “the frank and honest discussions by all participants helped highlight common areas of need which we can collectively address to help expedite therapeutic development for our patient community.  The FSH Society has launched a Therapeutic Accelerator program to resolve these issues and will be working with all interested stakeholders in the prioritization and strategic implementation of proposed solutions.”

This Workshop, “Industry Collaborative for Therapy Development in FSHD,” was designed to critically evaluate the current status of clinical trial readiness and identify opportunities and gaps that could be addressed through a collaboration among key stakeholders in the field—academics, industry, regulators, patient advocates, and the FSH Society.  Presentations and discussion sessions focused on the current status of and efforts needed to advance the identification and validation of pharmacodynamic and imaging biomarkers and clinical outcome assessment measures.  This Report summarizes the proceedings and key outcomes of the Workshop. The workshop was organized by John Porter, PhD, and funded by a generous grant from the Geraldi Norton Foundation.

Read the full white paper: Industry Collaborative Workshop for Therapy Development in FSHD.