KORLYM: The Cortisol Connection
A repurposed drug shunned by some for ethical reasons has gained fresh momentum as the model for a new line of therapies seeking
to mediate the impact of an essential human hormone—cortisol—on a wide range of life-threatening conditions, from diabetes
to osteoporosis and even cancer.
By William Looney
Rare diseases are a rich and growing source of innovation in drug discovery—nine of the 27 new medicines approved by the FDA
last year were for "orphan" indications affecting fewer than 200,000 people. But numbers alone don't reveal the significance
the rare disease segment holds in spreading the institutional roots of innovation to new areas of unmet medical need. What
really matters is how the orphan designation has helped broaden the horizons for biomedical entrepreneurs. These are individuals—scientists,
academics, and clinicians, often from outside big Pharma—with the grit and vision to launch start-ups featuring medicines
that extend treatments to patients underserved by the current R&D paradigm.
The science that yields these breakthroughs is often unconventional, but understanding the patient experience is an absolute
pre-condition for success. Victims of rare diseases are angry, disillusioned, and often sadly uninformed: how do you cater
to a customer base with a history of interactions with a health system that has very little to offer, and where the average
time to an accurate diagnosis is measured in years? Patient expectations are high; researcher's reputations, as well as their
wallets, are placed on the line; yet that lining is paved in silver, because ultimately everyone benefits from this expansion
of the medicines frontier. Knowledge gained from the investments in rare diseases has led to the development of drugs for
more common conditions like cancer, epilepsy, and dementia. The phenomenon even works in reverse, where older medicines in
wide use have been repurposed as therapies for these small-population disorders.
A spiffy example is the best suit cloth for insight, so this year Pharm Exec decided to extend its "Brand of the Year" recognition to include an orphan drug for rare disease. We went looking for a medicine
that combined three strengths:
» A novel clinical profile.
» An advance in the state of care for a difficult to treat condition, affecting a neglected cohort of patients.
» Distinctive, multi-channel marketing geared to building community awareness around the disease, not the product.
We also considered the human element, embodied in someone who connected an untouched area of science to a new treatment opportunity—entrepreneurs,
please step forward—as well as the brand's potential to seed growth in adjacent therapeutic areas, perhaps as the vanguard
for an entirely new class of drugs.