New Muscular Dystrophy Drug Granted FDA Orphan Designation in US

Receiving any kind of serious diagnosis can make someone feel like their world is ending.  In a way, it is; devastating news can flip one's life on its head.  When the news revolves around a diagnosis of oculopharyngeal muscular dystrophy (OPMD), the patient and their friends and family must face a new reality of medical appointments, medication, therapy and worst of all, degeneration of muscles. It all, eventually, becomes a new norm.

Oculopharyngeal muscular dystrophy (OPMD) is by far one of the rarest forms of the disease, typically striking adults between the ages of 40 and 60 years old. OPMD is a slow moving disorder that affects the muscles primarily in the face, starting around the eyes and then progressin to the throat. As the disease progresses, patients may experience a number of symptoms such as muscle weakness, difficulty swallowing and weight loss. Eventually, it will become difficult for the patient to eat, drink and breathe without assistance. As it stands, there is no cure for OPMD and sadly, for those who do develop it, the prognosis is fatal.

Since OPMD is quite rare (in fact, it may be considered to be one of the rarest forms of muscular dystrophy out there), the disease is sometimes referred to as an orphaned disease. Orphan diseases are a name that is commonly given to the rare diseases and disorders that typically affect less than 200,000 people according to the Food and Drug Administration (FDA).

How are treatments developed for orphan diseases?

Thankfully even rare diseases are considered to be just as important as their more common afflictions, especially in the field of research and development. This is why, drug companies, work just as hard to develop groundbreaking new drugs or treatment options for people who are suffering from orphan diseases, as they do for other much more common disorders, such as cancer and diabetes.

Recently, United States OPMD patients hoping to find some relief from the disease were thrilled to hear the news that a drug that had been developed by an Australian based company called Benitec would soon be available after being granted an orphan drug designation by the FDA.  Prior to this development, the drug had been granted an orphan designation in Europe.

So, what does an orphan drug designation mean? 

Well, when a drug is developed for what is considered to be an orphan disease, and found to be safe and effective, the FDA will grant it an orphan drug designation.  In a nutshell, that means that it’s approved by the Food and Drug Administration to begin the processes required to go to market so that it can be used as a treatment option for a disease or disorder that affects fewer than 200,000 people in the U.S., or in other words, a rare orphan disease. The designation also means that the drug company has permission to perform all the regulatory steps needed for a full approval and designation as well as being exempted from paying the regular FDA application fees.