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The European Medicines Agency (EMA) has granted priority drug status (PRIME) to AT-GTX-501, Amicus Therapeutics’ investigational gene therapy for CLN6 Batten disease, also known as the variant of late infantile neuronal ceroid-lipofuscinosis disease 6 (vLINCL6).

The PRIME designation accelerates 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, President and CEO of Amicus, 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 uncomfortable. Fourteen different genes – CLN1 To CLN14 – can cause Batten disease by allowing toxic deposits called lipofuscins to build up, especially in 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 living with Batten disease CLN6, an ultra-rare debilitating disease that presents in early childhood and is often associated with infant death, ”Crowley added.

These initial data come from an ongoing Phase 1/2 clinical trial (NCT02725580) evaluating a single dose of therapy in 13 CLN6 Batten patients, aged 1 year and older, who are ambulatory or able to walk with assistance.

Each received a dose of AT-GTX-501 into their spinal canal (an intrathecal injection) on day zero, then returned for follow-up visits on days 7, 14, 21, and 30. After this initial period, follow-ups take place every three months through month 24. Those who complete this study may participate in a long-term follow-up study (NCT04273243) that begins in January and through 2035.

Amicus intends to present additional test data at the Child Neurology Society’s virtual annual meeting, scheduled for October 12-23, and then provide an update on regulatory procedures in early 2021.

Earlier 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 Language Scale, which measures mobility and skills. speech disturbances.

The results also showed that the patients tolerated the therapy well, with mild side effects considered unrelated to the treatment itself.

The PRIME designation in the EU complements the orphan drug designation and the rare pediatric disease designation assigned 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 treat conditions with few treatment options.


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