Limb-Girdle Muscular Dystrophy Leaders Advance Collaborative Dialogue to Accelerate Drug Development to Address the Urgent Unmet Need in Limb-Girdle Muscular Dystrophy

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Feb 27, 2024, 08:53 ET

Feb 8 LGMD Scientific Workshop brought together LGMD Community Leaders, FDA Regulators, Drug Developers and Patients
A recording and one page overview of the workshop is now available at thespeakfoundation.com

ROCKVILLE, Md., Feb. 27, 2024 /PRNewswire/ -- On February 8, 2024, The Speak Foundation, a patient-led 501(c)(3) organization for Limb-Girdle Muscular Dystrophy (LGMD), convened a multi-stakeholder LGMD Scientific Workshop in Rockville, Maryland. The workshop brought together academic medical experts, senior leaders from the U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER), highly experienced drug developers, and other specialists that all sought to answer important questions on how to drive momentum to fulfill the unmet, yet critical need of the patient community to accelerate drug development for LGMDs. The meeting focused on six LGMD subtypes: 2A/R1, 2B/R2, 2C/R5, 2D/R3, 2E/R4, and 2I/R9. 

LGMD Scientific Workshop - Moving drug development forward for Limb-Girdle Muscular Dystrophy

The Speak Foundation's CEO & Founder, Kathryn Bryant Knudson, with Peter Marks, MD, PhD (CBER) and Peter Stein, MD (CDER) of the U. S. Food & Drug Administration (FDA)

The LGMD Scientific Workshop provided scientific and medical experts, regulators, patients, and drug development stakeholders the opportunity to explore innovative drug development approaches and clinical trial designs targeting the causal pathways and appropriate clinical and surrogate endpoints tailored for each subtype. Stakeholders acknowledged the gravity and challenges of living with LGMD, the urgent need for disease-modifying therapies to help halt these progressive diseases and the necessity for regulators and drug developers to collaborate to bring patient focused drug development forward utilizing regulatory flexibility and accelerated pathways.

"The LGMD Scientific Workshop demonstrates that we are at a juncture with a clearly understood pathway forward to develop proposed treatments in many subtypes of LGMD. The abundance of scientific research also supports the opportunity to leverage accelerated approval based on surrogate endpoints. We are thrilled that the workshop advanced a collaborative dialogue and brought people together from every facet of the drug development process to drive progress for patients. This was truly a monumental event for the LGMD community and a model for others to follow in the future." said Kathryn Bryant Knudson, founder of The Speak Foundation.

The LGMD Scientific Workshop sessions included a fireside chat with CBER Director, Dr. Peter Marks, and CDER's Office of New Drugs (OND) Director, Dr. Peter Stein, moderated by Annie Kennedy of the EveryLife Foundation for Rare Diseases. The Keynote Address was provided by Dr. Jerry R. Mendell, retired attending neurologist of Nationwide Children's Hospital and Senior Advisor for Sarepta Therapeutics. Other session speakers included Dr. Nicole Verdun, CBER, Dr. Michelle Campbell, CDER, Dr. Nicholas Johnson of Virginia Commonwealth University, Dr. Jennifer Levy of the Coalition to Cure Calpain 3, Dr. Louise Rodino-Klapac of Sarepta Therapeutics, Dr. Douglas Sproule of ML Bio Solutions, a BridgeBio affiliate, Dr. Katherine Mathews of University of Iowa Health Care, Dr. Matthew Wicklund of University of Texas San Antonio, Dr. Peter Kang of University of Minnesota Medical School, Dr. Lindsay Alfano of Nationwide Children's Hospital and Dr. James Signorovitch of Analysis Group. Additional sessions included Patient and Caregiver Experiences and Treatment Preferences and LGMD Endpoints and Clinical Trial Design roundtable.

"The LGMD Scientific Workshop marked an important milestone in advancing therapeutic development for the LGMDs." said Dr. Jerry R. Mendell. "It was encouraging to see the community galvanize as a whole around the significant progress in science and the clinical understanding of the LGMDs that now presents near-term opportunities to address the significant and pressing unmet need experienced by patients living with LGMD."

In-depth documentation of the workshop's findings and next steps is in progress and will be shared when available.

A recording and one page overview of the LGMD Scientific Workshop can be found here.

About The Speak Foundation: 

The Speak Foundation is a 501(c)(3) nonprofit charity, which is focused on supporting patients living with all forms of LGMD and on facilitating development of treatments for these diseases.

About Limb Girdle Muscular Dystrophy:

Limb-Girdle Muscular Dystrophy (LGMD) is a group of muscular dystrophies caused by mutations in one of over 30 genes. The age of onset, symptoms, progression, and inheritance pattern varies among genetic subtypes, but generally result in severe disability and are often life-threatening due to cardiac and respiratory complications. There are estimated to be some 20,000+ people living with a form LGMD in the United States.

Media Contact:
Kathryn Bryant Knudson
The Speak Foundation
E: [email protected]
M: 706-580-1755

SOURCE The Speak Foundation