B2 Stealth

Quiet Progress: FDA OKs Setmelanotide Priority Review

We call it stealth progress. Rhythm Pharmaceuticals last week gained approval from FDA for a priority review of setmelanotide for rare genetic forms of obesity in children. The indications are for obesity treatment in POMC and LEPR deficiency. Furthermore, the FDA has indicated it doesn’t plan to hold a public hearing on this drug This could mean an approval before the end of the year.

In fact, the target date for FDA’s verdict on approval is November 27.

Highly Targeted for Rare Diseases

POMC deficiency is something that is a problem for no more 500 patients. LEPR deficiency is not quite as rare. It may affect as many as 2,000 patients in the U.S. These forms of obesity are very severe in children at an early age. Because this therapy is highly targeted, the clinical results are impressive, especially in treating POMC deficiency. Patients in clinical trials for the POMC indication lost an average of 70 pounds.

The groundbreaking nature of this research yielded high-profile publications in the New England Journal of Medicine and Nature Medicine. But following those impressive publications, Rhythm has been doing its work quietly, moving toward getting an approval to market this potential breakthrough.

Rare Conditions, High Prices

The term of art for a products such as this is specialty drugs. In a word, that means expensive. They are drugs that offer a lot of value because they treat conditions that are otherwise untreatable in a very small population. Bringing such drugs to market is very risky and expensive. But to recover those costs, the price of a specialty drug winds up being very high because there are so few patients. In fact, a recent AARP report pegged the average cost of specialty drugs at about $79,000 per year.

The most expensive specialty drug to date is Zolgensma, at a price of $2.1 million to treat a rare muscle wasting disease. It is a one-time treatment that can cure a costly condition if it’s given to children before they suffer irreversible harm from the disease. Caring for a child with this disease can cost many millions of dollars. Even with all that expensive care, the disease was often fatal before Zolgensma came along.

A Quiet Launch?

The real question is how quiet the launch of this drug will remain. On one hand, it might be just an arcane new drug that is interesting to very few patients and specialists.

However, it may well attract attention because it represents a breakthrough and it will likely be quite costly. It will be indicated for specific forms of obesity in children as young as six. Because it is so highly targeted, it will offer more efficacy than we’re used to seeing in obesity drugs.

If and when setmelanotide gains approval, it will be an important milestone. But it may also shine a light on unmet needs for other people living with obesity and on specialty drug pricing. It’s one to watch.

Click here and here for more on the recent news about setmelanotide. For our earlier reporting on this drug, click here.

B2 Stealth, photograph © Peter Miller / flickr

Subscribe by email to follow the accumulating evidence and observations that shape our view of health, obesity, and policy.


May 18, 2020