In the United States, the Orphan Drug Act was developed to expedite the development and review of drugs or biologics intended to treat rare or life-threatening conditions. For the purpose of the Act, a rare disease or condition is one which affects under 200,000 Americans. According to Healio, the FDA recently granted Orphan Drug status to ADX-2191, a potential treatment for patients with primary vitreoretinal lymphoma. As there are no current FDA-approved treatments for this rare cancer, ADX-2191 has the potential to fulfill an unmet need within this patient community.
So what exactly is ADX-2191? According to drug developer Aldeyra Therapeutics (“Aldeyra“), ADX-2191 is an intravitreal formulation of methotrexate which reduces rates of retinal detachment in patients with proliferative vitreoretinopathy. In fact, the treatment even earned Fast Track designation for the same indication. Altogether, ADX-2191 is administered intravitreally via an injection. Through dihydrofolate reductase inhibition, the therapy works to significantly improve patient outcomes.
Since ADX-2191 received Orphan Drug designation, Aldeyra also receives a number of benefits. For example, in addition to fee waivers and tax credits, Aldeyra will be privy to increased FDA communication, as well as 7 years of market exclusivity if/when the drug is approved.
Vitreoretinal lymphoma (VRL) is a rare form of primary central nervous system lymphoma (PCNSL) which usually manifests as a highly malignant non-Hodgkin’s lymphoma. In early stages, primary vitreoretinal lymphoma may mimic uveitis symptoms, causing misdiagnosis. Vitreoretinal lymphoma most commonly affects females, especially those in their 50s and 60s. Early diagnosis may be difficult. However, in early stages, symptoms may include:
- Red eyes
- Floaters in the field of vision
- Blurred vision
- Vision loss (without pain)
- Photophobia (light sensitivity)
- Vitritis (inflammation of the vitreous cavity)
Because vitreoretinal lymphoma may occur in conjunction with other cancers, such as central nervous system lymphoma (CNSL), additional symptoms may appear. If other cancers are present, potential additional symptoms include:
- Muscle weakness or paralysis on one side of the body (hemiparesis)
- Impaired balance or coordination
Learn more about vitreoretinal lymphoma.