NO FREE LUNCH FOR OBESITY
A major lead in curing obesity came on the scene in the 1990s when researchers noticed that obese mice lacked a critical hormone dubbed leptin.
Suddenly, the tantalizing prospect of tackling obesity with leptin treatments sent both drug developers and casual dieters astir.
As the saying goes, there’s no free lunch, and excitement waned in recent years as it has become apparent that unlike mice, most obese people experience a lack of response to leptin.
It is not all bad news—people who have a rare genetic defect in leptin production now have the option of a drug.
RECOMBINANT DRUG MYALEPT APPROVED FOR LEPTIN DEFICIENCY
The FDA just approved Myalept for the treatment of leptin deficiency on February 24, 2014. Myalept is a recombinant version of leptin, and as such is considered a replacement therapy—it is replacing a hormone (leptin) that the body should be making but is not.
Leptin deficiency causes a rare disease known as lipodystrophy, leading to widespread loss of subcutaneous fat resulting in multiple metabolic complications including severe insulin resistance.
Myalept, now part of AstraZeneca’s drug arsenal, was first developed by Amylin Pharmaceupticals, then was picked up by Bristol-Meyers Squibb when they purchased Amylin in 2012. With the recent sell-off of BMS’s diabetes unit to AZ, Myalept has landed in a new home just as it received FDA approval as an orphan drug.
TERM OF THE WEEK: RECOMBINANT PROTEIN
Recombinant protein refers to protein therapeutics, a type of biologic. Drugs such as insulin, human growth hormone, or the newly-approved Myalept are examples.
The gene that codes for the therapeutic protein—for example, the LEP gene responsible for leptin—is first isolated and then recombined with vector DNA in order to transfer it into the cell line to be used for manufacturing. This technology is called recombinant DNA technology and its final product is a recombinant protein.
COCKTAIL FODDER: LEPTIN
The name “leptin” is derived from the Greek word for thin.
The term “lipodystophy” is derived from the Greek words “lipo” for fat and “dystrophy” for abnormal or degenerative condition.
ORPHANS OUTPACING OTHERS
With the first quarter of 2014 nearly behind us, orphan drugs have already won four of the five approvals from the FDA this year.
Orphan drug approvals are outpacing all others in the first quarter of 2014.
| Drug Name | Innovator | Approval Date | Indication | Orphan Drug? |
| Myalept | Amylin Pharmaceuticals | 2/24/2014 | Lipidystrophy | Yes |
| Northera | Chelsea Therapeutics | 2/18/2014 | Neurogenic Orthostatic Hypotension | Yes |
| Vimizim | BioMarin Pharmaceuticals | 2/14/2014 | Morquio A Syndrome | Yes |
| Hetlioz | Vanda Pharmaceuticals | 1/31/2014 | Non-24 Hour Sleep-Wake Disorder | Yes |
| Farxiga | Bristol-Meyers Squibb and Astra Zeneca | 01/08/2014 | Type 2 Diabetes | No |
