For INAD Patients and Families

What has happened:  A 2015 research publication by Retrotope collaborators on PLA2G6 associated neurodegeneration (PLAN) ( provided a rationale that Retrotope’s drug candidates against lipid peroxidation damage might provide a new treatment paradigm for Infantile Neuroaxonal Dystrophy (INAD). In October 2017 the FDA granted orphan designation for RT001 in the treatment of PLAN, which includes INAD. Orphan designation does not authorize a clinical trial; clinical trials require an Investigational New Drug (IND) application to be approved by the FDA. In early January 2018, encouraged by preliminary results in Expanded Access (Compassionate Use) patient trials in two INAD children during 2017, Retrotope filed background documents with the FDA in advance of a scheduled early February meeting with FDA whereby the Company will receive guidance for a formal clinical trial in INAD.

What to expect:  If the FDA agrees with the company’s proposed trial design at this meeting, Retrotope expects to quickly file an IND application with the FDA, and subsequently to initiate a clinical trial for INAD using RT001.  At that time, full details will be available from the FDA website and from the company.  You may contact Retrotope’s Clinical Trials Operations at 650-437-0700 x109 or email for up-to-date information on this process.