Ocugen, Inc. obtained a third Orphan Drug Designation (ODD) for OCU400. Orphan designation is granted primarily for novel drugs or biologics, potentially treating rare conditions. The conditions is affecting not more than 200,000 patients in the United States. This only means that the U.S. Food and Drug Administration (FDA) is convinced that OCU400 has this potential.
The novel gene therapy is expected to be an effective solution in the treatment of RHO mutation. It can restore retinal integrity while functioning across a wide range of genetically inherited retinal diseases.
The ODD Is Important in the Development of a Broad-Spectrum treatment for RP
The RHO mutation has a direct link to the Retinitis Pigmentosa (RP) group of rare, genetic illnesses. Statistics indicate that more than 1.5 million individuals worldwide suffer from genetic retinal diseases related to over 150 gene mutations. The saddest part is that around 40% of patients cannot be diagnosed genetically, and this has been making it difficult to develop personalized therapies.
However, OCU400 is promising, according to the Chief Medical Officer of Ocugean, Dr. Mohamed Genead, and can attend to a broad-spectrum RP. “We believe OCU400 has the potential to address multiple genetic mutations associated with RP and, therefore, help a broader pool of patients,” Dr. Genead emphasized.
The Market Lacks Approved Treatments for Multiple Forms of RP
The one reason that Ocugean is excited about this very important step is that it will be bringing hope to patients seeking for novel treatments for their inherited diseases. Hundreds of them are in desperate need. Unfortunately, and despite the everyday cases of RP, there are no approved treatments for the same.
Nonetheless, the endorsement of OCU400 for ODD will turn around the situation. The anticipation is that it will help in slowing or stopping the evolution of multiple types of RP.
Meanwhile, the company is confident about its product portfolio and that it will be able to transform the pharmaceutical industry. It will benefit from the experience of their newly appointed chair of the Retina Scientific Advisory Board, Dr. Genead.
