Orphan Drug Designation for PTP1B Inhibitor to Treat Rett Syndrome

The European Medicines Agency (EMA) has granted Orphan Drug Designation for a small molecule PTP1B inhibitor (DepYmed, Farmingdale, NY) for the treatment of Rett Syndrome (RTT).

“EMA’s decision to grant Orphan Drug Designation for our lead clinical candidate for the treatment of Rett Syndrome is another significant achievement for DepYmed and further validation among the world’s medical regulatory bodies of the potential for our compound,” said Andreas Grill, DepYmed’s president & chief executive officer. “In addition, it continues to align with our goal to provide treatments worldwide for patients with conditions where few if any options exist. There continues to be an urgent need to develop a treatment for patients with Rett Syndrome, a devastating rare genetic neurological disorder that occurs primarily in girls, and we are pleased to be working closely with EMA, as well as the FDA in the US to potentially address this need.”

Protein tyrosine phosphatases (PTP) have a significant role in the control of cell signaling pathways that are unsettled in many diseases; however, development of drug modulators of these enzymes has not been successful as of yet. An important PTP drug target, DepYmed has developed an orally bioavailable candidate that inhibits PTP1B.  This leading compound of the PTP1B inhibitor has shown promising efficacy in preclinical models of Rett syndrome, and a phase 1 clinical trial is anticipated in the second half of 2023.