Ultragenyx Pharma Wins FDA’s First Drug Nod for Rare Metabolic Disorder

Xconomy San Francisco — 

Ultragenyx Pharmaceutical won FDA approval Tuesday for a therapy that treats a group of rare metabolic disorders that render the body unable to convert certain fats—such as those found in olive oil, fish, and nuts—into energy.

People who have long-chain fatty acid oxidation disorders (LC-FAOD) typically manage the condition with dietary changes or by taking supplements. The treatment from Novato, CA-based Ultragenyx (NASDAQ: RARE) is a purified, synthetic version of a triglyceride, triheptanoin (Dojolvi). It’s designed to provide fatty acids that a patient’s cells can metabolize as an alternative energy source.

LC-FAOD is a genetic condition that leads to a deficiency of enzymes needed to metabolize fats and it’s typically diagnosed through newborn screening. The inability to produce energy from fat can lead to problems such as low blood sugar, muscle rupture, and heart and liver disease. These disorders affect an estimated 2,000 to 3,500 patients in the US, according to Ultragenyx. The regulatory nod, which covers both children and adults, makes the oral product the first FDA-approved LC-FAOD treatment. It comes a month earlier than the agency’s July 31 target date for a decision.

The approval was based on data from a Phase 2 study enrolling 29 patients and an extension study that enrolled 75, including 24 who were part of the mid-stage clinical trial. Over the entire treatment period, Ultragenyx reported that patients had a median 67 percent reduction in the annual rate of “major clinical events,” such as heart, muscle, or blood sugar complications. The median annualized duration rate of these events was reduced by 66 percent. Ultragenyx submitted its drug for FDA review last August.

Triheptanoin will come in 500 ml vials. For adults, the average net price will be $138,000 per patient per year, Erik Harris, Ultragenyx’s chief commercial officer said on a conference call. The amount of drug a patient needs will depend on caloric intake. For infants, the average net price is $46,000 for the first year of life. But Harris added that patients who can’t afford the therapy will be able to obtain it for free through the company’s patient access program.

“We strongly believe that cost should not be an impediment to access,” Harris said.

In a research note, SVB Leerink analyst Joseph Schwartz wrote that the most patients will likely pay closer to $50,000 annually. That’s because most patients will likely be children and the dosing of the therapy will be based on the patient’s weight, translating to a lower overall price. SVB Leerink projects that the therapy could generate $13.4 million in sales in the 2020 fiscal year; $43 million in fiscal 2021.

Camille Bedrosian, Ultragenyx’s chief medical officer, said that the company will begin a long-term disease monitoring program to collect more data about caring for patients with LC-FAOD. She added that the company plans to submit the therapy for regulatory review in Brazil, Canada, and Europe. Ultragenyx expects the drug will become available in the US by the end of July.

Triheptanoin is based on research licensed from Baylor University in 2012. According to Ultragenyx’s securities filings, the company must pay Baylor royalties from the product’s sales, as well as $7.5 million upon the achievement of certain sales milestones.

The triheptanoin approval marks Ultragenyx’s fourth FDA nod and the second in the past two weeks. On June 18, the FDA approved burosumab (Crysvita) as a treatment for tumor-induced osteomalacia. That drug was first approved in 2018 as a treatment for the bone disease X-linked hypophosphatemia.

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