In 2014, the U.S. Food and Drug Administration approved eight new drugs for ultra-orphan diseases, which affect fewer than one in every 30,000 Americans. BioMarin Pharmaceutical’s VimiZim (elosulfase alfa) was one of them. The drug was shown to be effective in young patients with Morquio syndrome type A, which affects approximately 800 Americans and around 3,000 people throughout the developed world. BioMarin priced the drug at $380,000 per year, making it one of the top 5 most expensive drugs in 2015.
VimiZim appears in life science consulting firm Trinity Partners’ second annual drug index, which evaluates the performance of all 41 novel drugs approved by the FDA in 2014.
Dubbed the Trinity Drug Index, it ranks new therapies based on three key categories – commercial performance, therapeutic value and R&D complexity. The scores are averaged for a composite grade of 1-5.
The report authors state that, as with last year’s index, products with the highest therapeutic value typically posted the best commercial performance (determined by total sales through to December 2016 and projected sales from 2017-2021). Except in the category of rare diseases.
So how did VimiZim fare?
“We see that nearly all ultra-orphan drugs have low commercial scores relative to their therapeutic scores, placing them near the bottom of our commercial performance versus therapeutic value plot,” the authors note.
The tiny market size means that VimiZim couldn’t deliver a lot, even with a price tag of $380,000. However, in an email forwarded by a company representative, Dave Fitzhenry, a managing partner at Trinity Partners, said there are still good opportunities in the field.
“Every company would love to have a blockbuster drug on their hands, but a company can be successful without one,” Fitzhenry wrote. “The small to midsize revenue opportunity for these niche drugs makes them more valuable to smaller companies that can develop them with leaner infrastructures.”
It’s food for thought as the United States continues its debate over the cost of prescription therapies and while so few rare diseases have an approved drug. Companies targeting orphan diseases are not necessarily making a killing, but they are treating a major unmet need.
A winning formula
At the other end of the spectrum are the checkpoint inhibitors. Merck’s Keytruda and Bristol-Myers Squibb’s Opdivo placed first and third respectively on the 2014 index. While the two drugs have delivered comparable therapeutic benefits, Keytruda topped Opdivo’s R&D score, earning it first place. (Harvoni, a combination hepatitis C drug manufactured by Gilead Sciences, ranked at number two.)
Keytruda and Opdivo hit on two important trends. The first is that novel specialty products with a high unmet need typically do well. Checkpoint inhibitors treat specific subsets of cancer patients and work in a targeted way. At the same time, this class of drugs has proven that it can treat many different forms of cancer. In other words, the drugs are targeted but the potential patient populations add up.
“Drugs targeting more than one indication tend to rise to the top, in terms of commercial success, given access to a larger patient pool and the ability of follow-on indications to benefit from the existing indication (and vice versa),” the authors of the Drug Index write.
While the authors somewhat expected these drugs to be successful, the performance of the diabetes drug on the list, which offered just an incremental therapeutic advance, was more unexpected.
“The commercial success of specific diabetes drugs (Trulicity, Farxiga and Jardiance [to some extent]) was our most surprising finding.”
The success of these products hinged on their ability to capitalize on moderate clinical differentiation (Trulicity’s once-weekly versus daily competitor dosing schedule, Farxiga/Jardiance’s rise with the cardiovascular benefit of SGLT2s) and strong [direct-to-consumer] marketing campaigns.
An omen for Gilead and Kite Pharma?
In their analysis, the authors of the report suggest that the timing of mergers and acquisitions might impact the commercial success of a drug.
Approximately 65 of drugs approved in 2014 were either in-licensed or acquired. Of note, all but one of the five products acquired during the regulatory review process or after FDA approval underperformed commercially. That list includes Esbriet, Northera (1.8/5), Dalvance (1/5), Sivextro (1.2/5), and Zerbaxa (1.2/5). Esbriet was the exception, with a commercial score of 3.6/5.
This phenomenon of last-minute acquisitions has continued. In August, Gilead Sciences acquired Kite Pharma for $11.9 billion. Kite’s lead cancer drug was under FDA review at the time. The drug, Yescarta, was given the green light in October.
“While it is common to acquire or in-license during the later stages of development, this may be a signal that the likelihood of product success decreases as the acquisition nears the time of market entry, a period that should be focused on meeting strategic launch objectives in a disruption-free environment,” the authors argue in the report.
No one is under the illusion that it will be an easy launch, but we’ll have to wait several years to know how it really performed.
Illustration: Getty Images
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