Today's Orphan Drug Could Be Tomorrow's Blockbuster

In my last post (Orphan Drugs: A Shift in the R&D Paradigm), I alluded to how today's orphan drug could be tomorrow's blockbuster. Drugs initially may be approved for one indication in a small population, but later acquire additional uses after longitudinal studies and real world observations. These drugs present significant opportunity for innovation, while minimizing cost and time to market.

How many of you are familiar with the drug "Occulinum"? While this name may not universally resonate, it certainly represents the potential of orphan drugs.

In the late 1960's/early-70's, Dr. Alan Scott - an Ophthalmologist in San Francisco - investigated treatments for his patients who had crossed-eyes (strabismus) and uncontrollable blinking (blepharospasm). He found one particular compound to be pretty successful at managing both these conditions, and built a company - Occulinum, Inc. - entirely around his new drug.

Approved in 1984 as an orphan drug for uncontrolled blinking, neck pain and muscle spasms, Occulinum treatment typically lasts about four to six months. As its use only relieved the chronic conditions temporarily, patients needed to come back to see the doctor two to three times a year - sometimes more - over a long period of time.

Around the same time that Dr. Scott's company received approval for Occulinum, Canadian ophthalmologist Jean Carruthers was treating her patients in Vancouver with the same drug. Over time, her staff noticed that her patients were looking healthier, more vibrant, and even younger every time they came back for a treatment. This amazing phenomenon led Dr. Carruthers and her husband, Dr. Alistair Carruthers (a dermatologist) to begin experimenting among themselves...and even on themselves.

In 1989, Occulinum received broader approval. Then in 1991, Allergan bought the company for $9 million and decided to rename it. Shortly thereafter, the Drs. Carruthers published their study on the effect of treatment of "frown lines" with Allergan's new drug, -- you guessed it! -- Botox.

In 2002, FDA approved Botox for treatment of frown lines. In 2010, Botox gained approval for the treatment of chronic migraines. There are 5 million doses administered annually in North America, which translated into $1.5 billion in sales. Allergan has now applied for patents for around 90 different uses.

Although controversial, Botox epitomizes how an orphan drug once designated for a particular condition might progressively receive approval for several other indications. While past performance is no certain indication of future results, the Botox story provides a great example of the potential for orphan drugs to become the blockbuster of the future.

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