(CercleFinance.com) – Poxel announces tonight that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to PXL065 and PXL770 for the treatment of patients with adrenomyeloneuropathy (AMN), the most common form of common cause of X-linked adrenoleukodystrophy (ALD).
A phase IIa proof-of-concept (POC) clinical study will be launched as soon as possible for each of these two drug candidates, subject to obtaining additional funding.
For Poxel’s CEO, Thomas Kuhn, this FDA designation is ‘an additional regulatory step for the development of PXL065 and PXL770’ in an indication that currently has no approved treatment.
‘This status could provide PXL065 and PXL770 with potential market exclusivity for seven years after FDA approval and, with the recent achievement of Fast Track status, reinforces our desire to continue the strategic reorientation towards rare diseases initiated last year,” he added.