New Products, Rare Diseases

Ultragenyx lands first FDA OK, with another on the horizon in shift to commercial ops

Ultragenyx CEO Emil Kakkis is now operating a commercial operation, just as he dreamed he would when he started the company in 2010.

The FDA has approved Mepsevii (vestronidase alfa-vjbk) for an ultra-rare genetic enzyme disorder called MPS VII, and the OK comes with a priority review voucher that could easily be worth $125 million.

Emil Kakkis

The biotech $RARE has been signaling this advance for some time now, eagerly awaiting a green light along with a big approval in line for burosumab, which has a much larger market in view. Mepsevii — a replacement of the missing beta-glucuronidase enzyme that causes skeletal deformation as toxic material builds up in cells — was tested in 23 patients ranging from 5 months to 25 years, demonstrating a significant gain in walking distance for the patients in the drug arm.

Leerink’s Joseph Schwartz has highlighted the handful of patients who have MPS VII.

Given the low prevalence of MPS7 pts. WW (~200 estimated), pt. identification, disease awareness, and named pt. sales activities could lay an important groundwork for vestronidase alpha once it’s approved.

And it won’t be cheap. In a call with analysts Wednesday evening, Ultragenyx set the price at an average of $375,000 a year, well below the $550,000 price Schwartz had bet on. But it’s important to remember that the company price is based on weight — 4mg/kg Q2W ($2,115/vial) — with the average pegged at 55 pounds.

Novato, CA-based Ultragenyx has had its setbacks along the way, but it’s also been making steady progress toward the time when it can start fielding a 30-person US sales team to market a portfolio of rare disease drugs. Kakkis also recently decided to buy out the surviving gene therapy portfolio at the troubled Dimension Therapeutics, which ran into a thicket of trouble with weak hemophilia B data for the lead program.

“This approval underscores the agency’s commitment to making treatments available to patients with rare diseases,” said Julie Beitz, director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research (CDER). “Prior to today’s approval, patients with this rare, inherited condition had no approved treatment options.”

This approval also underscores the huge popularity of rare diseases for startups, a field where researchers can make real progress with small patient groups and payers are willing to cover big annual costs so long as the patient numbers are contained.


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