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Clinicians and Access

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Allievex is developing novel therapies for the treatment of rare pediatric neurodegenerative diseases. Our lead program is tralesinidase alfa (AX 250) for the treatment of children with Sanfilippo syndrome type B, also known as Mucopolysaccharidosis IIIB (MPS IIIB).


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Visit to view information on all registered clinical studies for Allievex.

Allievex is focused on gaining regulatory approvals to make our therapies broadly available to patients as quickly as possible. Clinical trials are key research tools for advancing medical knowledge and utilizing innovative strategies to improve patient care. Allievex aims to partner with regulatory authorities, medical and patient communities to develop and market approved safe and effective products.


Compassionate Use

Allievex acknowledges potential interest to access our investigational therapies prior to regulatory approval through compassionate use (expanded access).

We are committed to providing access to tralesinidase alfa treatment to as many Sanfilippo syndrome type B patients as possible. To achieve this goal, we are focused on obtaining regulatory approval for tralesinidase alfa. At this point in time, we are unable to offer a compassionate use program. We will continue to evaluate the potential for compassionate use of tralesinidase alfa on an ongoing basis.

To receive updates on Allievex’s compassionate use program, please contact Allievex Expanded Access.

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Information on ongoing Allievex clinical studies

Visit to view information on all registered clinical studies for Allievex. This is a registry and results database of publicly and privately supported clinical studies conducted around the world. The U.S. Food & Drug Administration’s website provides valuable information on Expanded Access/Compassionate Use.

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