The FDA has granted orphan drug designation to Aldeyra Therapeutics’ ADX-2191 (methotrexate for intravitreal injection) for the treatment of primary vitreoretinal lymphoma (PVRL). The US developed drug is the first approved treatment for PVRL, a rare and aggressive cancer, with approximately 600 new cases diagnosed annually in the US.
“PVRL is an often fatal cancer that creates unique challenges for diagnosis and treatment,” said Dr Todd Brady, president and CEO of Aldeyra. “ADX-2191 represents the first methotrexate preparation specifically formulated for intraocular injection. The designation is another important step forward for our retinal disease programme, complementing our ongoing clinical development of ADX-2191 for the prevention of proliferative vitreoretinopathy, the leading cause of failure of retinal reattachment surgery.”
Aldeyra announced in 2019 that the FDA had fast-tracked the drug’s development, allowing faster processing of reviews and approvals. By June 2020 the European Commission had granted ADX-2191 orphan drug status. The FDA’s orphan drug designation allows financial incentives to sponsors for developing drugs for rare diseases and conditions. Sponsors of designated orphan drugs are eligible for tax credits for clinical trial costs, waiver of the user fee for marketing applications and, upon approval, consideration for seven years of marketing exclusivity.
