Workshop Focuses on Accelerating Approval for Neuropathic MPS Disorders Treatments

Presenters at Reagan-Udall Foundation Meeting ● Photo Credit: National MPS Society

WASHINGTON, March 18, 2024 (VSNewsNetwork.com) - A workshop hosted by the Reagan Udall Foundation, in partnership with the FDA, Cure Sanfilippo Foundation, National MPS Society, and The Ryan Foundation, was held on February 21, 2024, to discuss the potential of cerebrospinal fluid-heparan sulfate (CSF-HS) as a primary biomarker for accelerated approval in neuropathic MPS disorders. The session aimed to address the urgent need for new treatment pathways in neurodegenerative ultrarare diseases.

Cara O’Neill, MD, FAAP, of the Cure Sanfilippo Foundation, emphasized the critical need for expedited treatment options, stating, "We are asking the FDA for equitable access to the Accelerated Approval pathway... We truly need access now in Sanfilippo syndrome or we are going to lose another generation of our children."

The workshop convened experts from various fields to discuss the neuropathic MPS disorders' pathophysiology, clinical and preclinical data, and the role of CSF-HS as a surrogate biomarker. Matthew Ellinwood, DVM, PhD, from the National MPS Society, reflected on the significance of the event, saying, "During my quarter century working in the field of MPS research, this workshop was the most unified, consistent, and impactful presentation of the case of CSF heparan sulfate as a surrogate biomarker in neuropathic MPS disorders."

The discussion highlighted the challenges faced by patients with ultra rare diseases, including the failure of clinical studies to meet FDA criteria and the halting of therapy development programs. Mark Dant, Volunteer Executive Director of The Ryan Foundation, urged the FDA to act, noting, "At least ten companies developing therapies for Sanfilippo syndrome have halted their programs while regulation is struggling to keep up with the science in the field."

The workshop underscored the importance of collaborative efforts to qualify biomarkers that support rare disease regulatory pathways, focusing on the accelerated approval pathway as a viable option for evaluating treatments for neuropathic MPS diseases.

For more details on the workshop and MPS disorders, visit www.mpssociety.org.

Source: National MPS Society via Newswire