Laboratoires Kol received orphan drug designation from the European Medicines Agency (EMA) on 20 April 2026 for aganirsen in the prevention of corneal graft rejection. In the EU, orphan drug designation provides a 10-year period of marketing exclusivity following product approval, along with incentives including protocol assistance from the EMA and direct access to the centralized authorization procedure.
Aganirsen is an antisense oligonucleotide (ASO) that targets insulin receptor substrate-1 (IRS-1) mRNA, suppressing downstream production of vascular endothelial growth factor (VEGF) and inflammatory cytokines. That dual anti-angiogenic and anti-inflammatory mechanism has direct relevance to corneal graft rejection, where neovascularization of the cornea is a recognised risk factor for transplant failure. Prior clinical work, including the I-CAN study published in Ophthalmology in 2014 (DOI: 10.1016/j.ophtha.2014.03.038; PMID: 24811963), showed that topical aganirsen eye drops inhibited keratitis-induced corneal neovascularization and reduced the need for transplantation.
The molecule was originally developed by GeneSignal International SA, a Swiss biotechnology company. Laboratoires Kol, a French pharmaceutical company, now holds the sponsorship of the EU orphan designation, though the precise terms and timing of any transfer of rights between the two entities are not publicly documented.
The only identified formal interventional trial for aganirsen is the STRONG study (NCT02947867), a Phase II/III, randomized, placebo-controlled, double-masked, multicenter study sponsored by Gene Signal SAS, evaluating aganirsen in patients with ischaemic central retinal vein occlusion (iCRVO) at risk of neovascular glaucoma. That trial carries an “Unknown status” in the registry, and no confirmed active recruitment or completion data is available. The indication studied in that trial differs from the corneal graft rejection prevention indication covered by the current orphan designation, indicating that Laboratoires Kol is pursuing a distinct clinical development path. No marketing authorization for aganirsen has been identified in any jurisdiction.
The competitive landscape for pharmacological prevention of corneal graft rejection remains sparse. Current standard of care relies predominantly on topical corticosteroids and calcineurin inhibitors such as cyclosporine, which carry limitations including long-term tolerability concerns and incomplete protection in high-risk vascularized corneas. No therapy has received regulatory approval specifically for prevention of corneal graft rejection in the EU or US, which forms the basis for the orphan qualification. Aganirsen’s IRS-1 targeting mechanism is distinct from immunosuppressive approaches, operating upstream of both angiogenic and inflammatory cascades rather than directly modulating immune cell activity.