The past weeks have been quite good for many pharmaceutical and biotech companies and the latest to enter the fray is Ocugen Inc (NASDAQ:OCGN). An orphan drug designation is almost always a major positive for a company and on Monday it emerged that the company had its fourth such designation.
On Monday, it emerged that its product OCU 400, which is meant for the treatment of Retinitis Pigmentosa, has been granted the coveted orphan drug designation by the powers that be in the United States. It is a significant development for Ocugen and after the news broke, the stock rallied by as much as 28%. In such a situation, it would be remiss if investors did not take a deeper look into the stock and figure out if it is going to be a worthy investment or not.
The product candidate in question, OCU 400, has been developed using gene therapy. Once it is delivered to the target cells in the retina, the medicine is meant for using an adeno-associated viral vector. Retinitis Pigmentosa is a condition that is hereditary in nature and is brought about by mutation of the PDE6B. The condition is characterized by the loss of eyesight by the time one hits his mid-life and in addition to that, the patient also suffers from continuous loss of the photoreceptors.
However, despite the positive news, investors are going to be more interested in knowing about the clinical trials and the company did provide an update on the matter. Ocugen stated that it is going to start two parallel Phase 1 and Phase 2 trials at some point in 2021. The company added that the two trials are going to target two specific retina diseases.
These are all highly important developments for the company and its product. The orphan drug designation is certainly a positive that is going to help Ocugen in the long term. That being said, all eyes are now going to be on the trials that are going to be conducted by the company next year. It will ultimately decide the future of medicine.
In addition to that, it is also necessary for investors to keep in mind that Ocugen already has three other orphan drugs under its control and hence, the company’s experience in this particular category of medicines might come in handy.
The United States Food and Drug Administration’s Office of Organ Products Development awards these designations to products that could be effective for the treatment of rare diseases or conditions. The condition or disease must not affect more than 200000 people in the United States every year. There are other incentives for the companies as well under the Orphan Drug Act and that is another factor behind the optimism around Ocugen.
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