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Orphan Drug Designation for Treatment of Stargardt's Disease!


Say what?! Great news!!!

Acucela Receives Orphan Drug Designation from the FDA for the Treatment of Stargardt's Disease!

Prior to my research, I did not know what an orphan drug designation was. Here's my current understanding: Basically, it is a tax exemption for researching a drug that treats or cures a rare disease. It creates a financial incentive. As we know, Stargardt's is a rare disease that affects (over) 25,000 Americans (Low Vision Centers of Indiana, 2016). Pharmaceutical companies are not exactly interested in fighting for Stargardt's because this number is so low that profits would not be very grand. Bummer, right?! Well an orphan designation makes it so that those diseases with fewer than 200,000 people affected are not forgotten. In these situations, the FDA can work with the smaller companies to work towards treatment of the rare disease. Really good news!

I've pasted the article down below for you to read but here's my simple summary. Also you can go to the website here.

In healthy eyes, vitamin A is great! It is used to make an important molecule in our photo receptors. The thing is, during this pathway there are some byproducts that can be harmful if not cleared away. In a Stargardt's eye, we have a mutation in the gene that normally makes a protein to clear away this vitamin A byproduct. Without this protein, we accumulate these fatty deposits on and around our macula, impairing central vision. With time, these photoreceptors can die and cause a progressive vision loss (National Eye Institute, 2015).

Long story short (in my understanding), Emixustat Hydrochloride slows down this visual cycle which decreases the amount of vitamin A byproduct. They reported that in animals, vision loss was stopped and reversed, preserving the retina. Yay! Let's celebrate that a cure is in progress! Clinical trials are underway in patients with Stargardt's.

As always, live in the present and enjoy the little things along the way <3

Acucela Receives Orphan Drug Designation from the FDA for the Treatment of Stargardt's Disease

Source: Acucela

Friday, January 06, 2017 | FDA Approval/Clearance , Acucela

Acucela announced that the FDA granted orphan drug designation to Acucela’s leading drug candidate emixustat hydrochloride for the treatment of Stargardt disease. The orphan drug designation does not apply to other indications emixustat is being developed.

The Orphan Drug Act (ODA) provides for granting special status to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the U.S.

Emixustat Hydrochloride

The visual cycle is the process by which vitamin A is recycled in the eye; vitamin A is crucial to the visual process. Emixustat modulates the visual cycle by inhibiting a critical enzyme of this pathway, retinal pigment epithelium protein 65 (RPE65). Slowing the visual cycle reduces the availability of vitamin A derivatives (11-cis- and all-trans-retinal) to form precursors of A2E and related compounds. In animal models of Stargardt disease and retinal degeneration, emixustat was found to stop and reverse the accumulation of A2E and to preserve the integrity of the retina.

Emixustat when delivered orally was found to be generally well tolerated in human clinical studies with delayed dark adaptation being the most common ocular adverse event. Acucela is planning to explore emixustat’s potential to stop or slow the progression of vision loss in patients diagnosed with Stargardt disease in future clinical studies.

Dr. Ryo Kubota, MD, PhD, and Chairman, President and CEO of Acucela, states, “We are very pleased to receive FDA’s orphan drug designation for emixustat to treat Stargardt disease. We are actively advancing the development of emixustat to address this unmet medical need.”

"No object is mysterious. The mystery is your eye." - Elizabeth Bowen

#stargardts #research

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