September 19, 2024
eMedClub News
On September 17 local time, Atsena Therapeutics announced that its investigational AAV gene therapy ATSN-201 had been granted Orphan Drug Designation by the FDA for the treatment of X-linked retinoschisis (XLRS). This is the second FDA designation for ATSN-201, following the Rare Pediatric Disease Designation it received last month.ATSN-201 is a potential "best-in-class" gene therapy that uses Atsena Therapeutics' novel diffusion capsid AAV.SPR to achieve therapeutic gene expression levels in the photoreceptors of the central retina, while avoiding the surgical risks associated with macular detachment.XLRS is a condition caused by mutations in the gene encoding retinoschisin. RS1Monogenic X-linked diseases caused by gene mutations. Its characteristics include abnormal splitting between retinal layers, leading to uncorrectable vision loss through glasses, and gradually progressing to blindness. XLRS predominantly affects males and is typically diagnosed in early childhood. In the United States and the European Union, approximately 30,000 males are affected by XLRS, yet there are currently no approved treatments available on the market.2025 (3rd) Biopharmaceutical Innovation Industry Conference
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ATSN-201 is an adeno-associated virus (AAV) gene therapy that utilizes the company's novel propagation capsid technology, AAV.SPR, to overcome the challenges associated with intravitreal delivery of AAV in treating XLRS. When injected into the extra-macular region, it laterally spreads beyond the subretinal injection site, enabling safe and effective transduction of the central retina (which primarily exhibits schisis cavities in XLRS patients).
FDA Rare Pediatric Disease Designation, primarily used to treat serious or life-threatening rare diseases in patients under the age of 18. This designation for ATSN-201 will grant the therapy a Priority Review Voucher (PRV) upon approval, greatly incentivizing companies like Atsena Therapeutics to invest in rare pediatric diseases.
This orphan drug designation for ATSN-201 aims to provide a safe and effective treatment, diagnostic, or preventive therapy for rare disease patients in the United States affecting fewer than 200,000 people. It also offers certain incentives, such as tax credits for clinical trial expenses after approval and waivers of Prescription Drug User Fee Act (PDUFA) fees. Products granted FDA orphan drug status enjoy seven years of market exclusivity for the designated orphan indication, which is independent of intellectual property protection.
Currently, the Phase I/II clinical trial of ATSN-201 is underway to evaluate the safety and tolerability of the therapy. This trial is an open-label, dose-escalation, and dose-expansion clinical study (LIGHTHOUSE study), enrolling participants aged 6 years and above with a clinical diagnosis of RS1Male patients with XLRS caused by gene mutations.
▲ Atsena Therapeutics Pipeline Diagram (Source: Official Website)Atsena Therapeutics is a clinical-stage gene therapy company dedicated to developing best-in-class therapies for reversing or preventing inherited retinal diseases. The company’s lead program is conducting a Phase I/II clinical trial of ATSN-201 for the treatment of XLRS, a progressive inherited condition typically diagnosed in childhood that affects boys and men. Another ongoing Phase I/II clinical trial aims to evaluate ATSN-101 forGUCY2D(LCA1) Efficacy in patients with Leber congenital amaurosis caused by biallelic mutations. This therapy has received FDA Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy (RMAT) Designation, and Orphan Drug Designation. Additionally, ATSN-301 is a gene therapy based on dual AAV vectors, still in preclinical research, aimed at prevention.MYO7ARelated to Usher syndrome (USH1B) causing blindness.1.https://www.globenewswire.com/en/newsrelease/2024/09/17/2947307/0/en/Atsena-Therapeutics-Receives-Orphan-Drug-Designation-from-the-U-S-FDA-for-ATSN-201-Gene-Therapy-to-Treat-X-linked-Retinoschisis.htmlRegistration and Event Inquiry
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