Education
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Dearest Sanfilippo Community Members and Allies,
It is with deep sadness that we inform you of the impending liquidation of Allievex. Since the beginning of this drug program with BioMarin (as BMN 250), and then through its acquisition by Allievex (as AX 250), the National MPS Society has been a strong supporter of this drug program and of the Sanfilippo type B community who benefited from its development.
The Allievex treatment was an enzyme replacement therapy using tralesinidase alfa which was delivered weekly directly to the brain via a catheter. This therapy corrected the enzyme deficiency of MPS IIIB patients. The therapy reduced heparan sulfate levels in the brain and spinal cord in preclinical studies, and levels were lowered in the cerebrospinal fluid of treated patients. Especially in the cohort of trial patients who began treatment at a young age, therapy halted the neurodegenerative process and preserved neurological function in patients.
Despite these promising results, the collective efforts of Allievex’s team, and the support of rare disease advocates, researchers, clinicians, families, and allies around the world, the desired regulatory flexibility from the FDA around Accelerated Approval Pathway was not forthcoming during their early discussions with the FDA. They ceased all business operations in October 2023. However, the Allievex team remained hopeful, and pursued additional FDA engagement.
In March of this year, former Allievex employees met with the FDA Center for Drug Evaluation and Research, where the FDA changed course. Allievex was encouraged to file its Biologics License Application for consideration under the Accelerated Approval Pathway. While this was good news, it came too late to rescue the business. Today Allievex announced that they had entered an assignment for the Benefit of Creditors marking the formal liquidation of their assets.
We share in your deep despair over this outcome. Those families and patients who have benefited from this therapy and who now have nothing to look for in the future are an abiding concern to us. We hope for an acquisition that could revive Allievex but are doubtful after so much time. We are grateful that the Allievex program did play a very positive role in the efforts of other MPS treatment developers such as Denali, Ultragenyx, and REGENXBIO to access improved regulatory paths with the FDA, but today’s news still leaves our MPS IIIB community bereft.
The Society remains committed to advocating for and supporting research and treatments for all MPS disorders, and we will continue to communicate any news regarding the future of Allievex as it develops. If you have any questions, please contact Terri Klein at terri@mpssociety.org.
Together, we hope to find a path forward that ensures the best possible outcomes for everyone who is affected by MPS.
Sincerely and with both sadness at this news and hope for the future,
Matthew Ellinwood, DVM, PhD
Chief Scientific Officer
National MPS Society
Click here to read Allievex Founder Thomas Mather’s full statement.
