The European Medicines Agency (EMA) has granted Priority Medicines (PRIME) status to AT-GTX-501, Amicus Therapeutics’ investigational gene therapy for Batten disease CLN6, also known as the late infantile neuronal ceroid lipofuscinosis 6 (vLINCL6) disease.
PRIME designation helps accelerate the development and regulatory review of promising drugs that target unmet medical needs. The goal of the program is to help these drugs reach patients faster.
“We are very pleased that the EMA has recognized the potential of our CLN6 gene therapy,” said John F. Crowley, Amicus President and CEO, in a press release.
AT-GTX-501 uses an inactive adeno-associated virus as a vehicle to provide cells with a functional copy of the CLN6 embarrassed. Fourteen different genes — CLN1 at CLN14 — can cause Batten’s disease, by allowing the accumulation of toxic deposits called lipofuscins, in particular within the cells of the central nervous system.
“Based on our preliminary clinical data, we believe that AT-GTX-501 could potentially be a transformative treatment option for children with CLN6 Batten disease, an ultra-rare wasting disease that presents in the early childhood and is often associated with infant death,” Crowley added.
These early data come from an ongoing Phase 1/2 clinical trial (NCT02725580) evaluating a single dose of the treatment in 13 CLN6 Batten patients, ages one year and older, who are ambulatory or able to walk with assistance.
Each received a dose of AT-GTX-501 in their spinal canal (an intrathecal injection) on day zero, then returned for follow-up visits on days seven, 14, 21, and 30. After this initial period, follow-ups take place every three months until month 24. Those who complete this study can participate in a long-term follow-up study (NCT04273243) that opened in January until 2035.
Amicus intends to present additional trial data at the Child Neurology Society’s virtual annual meeting, scheduled for October 12-23, and then provide an update on regulatory proceedings in early 2021.
Past interim results have shown signs that AT-GTX-501 may halt disease progression, as evidenced by changes in patients’ scores on the Hamburg Motor and Linguistic Scale, which measures mobility and speech disorders.
The results also demonstrated that the patients tolerated the therapy well, with mild side effects thought to be unrelated to the treatment itself.
The PRIME designation in the EU complements the Orphan Drug Designation and Rare Pediatric Disease Designation given to AT-GTX-501 in the United States. Similar to PRIME, these designations provide resources and incentives to accelerate the development of promising therapies that address conditions with few treatment options.