Ultragenyx's (RARE) NDA for LC-FAOD Drug Accepted by FDA

Ultragenyx Pharmaceutical Inc. RARE announced that the FDA has accepted for review the New Drug Application (NDA) for pipeline candidate UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD).

The FDA has assigned a standard review designation to the NDA and set a Prescription Drug User Fee Act (PDUFA) target date of Jul 31, 2020.

The NDA filing is supported by a comprehensive package of data, comprising results from a company-sponsored phase II study of UX007 in 29 patients and a long-term safety and efficacy extension study in 75 patients, including 20 patients who were previously naïve to UX007. The cumulative data also includes a retrospective medical record review of 20 original compassionate use patients, 67 patients treated through expanded access and a randomized, controlled, investigator-sponsored study of 32 patients showing an effect on cardiac function.

The agency indicated that it is currently not planning to hold an advisory committee meeting to discuss the application.

UX007, an investigational therapy, directly addresses the deficiencies in LC-FAOD by providing patients with an alternative energy source that can be metabolized to increase intermediate substrates in the Krebs cycle, a key energy-generating process.

We note that LC-FAOD is a set of rare metabolic diseases that prevent the conversion of fat into energy and can cause low blood sugar, muscle rupture, and heart and liver diseases.

A potential approval of the drug will broaden the company’s portfolio. Shares of the company have lost 4.2% year to date compared with the industry’s decline of 6.4%.


Ultragenyx primarily focuses on the treatment of serious, rare and ultra-rare genetic diseases. The already approved therapies and the clinical-stage pipeline consist of three product categories — biologics, small molecules and gene-therapy product candidates.

The biologic products include approved therapies — Crysvita (burosumab) and Mepsevii (vestronidase alfa). Crysvita is an antibody targeting fibroblast growth factor 23 or FGF23 developed for the treatment of X-linked hypophosphatemia (XLH), which is a rare, hereditary, progressive and lifelong musculoskeletal disorder characterized by renal phosphate wasting caused by excess FGF23 production. The FDA recently updated the label to include new clinical data demonstrating the superiority of treatment with Crysvita over oral phosphate and active vitamin D (conventional therapy) in pediatric patients with XLH, improvement in stiffness and maintenance of the efficacy of the drug in adult patients under long-term treatment. The indication has also been expanded to include infants as young as six months.

Mepsevii is an intravenous or IV enzyme replacement therapy, developed for the treatment of mucopolysaccharidosis VII. Ultragenyx also exercised its option with REGENXBIO Inc. RGNX to develop a gene therapy for the treatment of patients with CDD, using the latter’s adeno-associated virus (AAV) vectors like AAV9. The gene therapy pipeline includes DTX301 (an adeno-associated virus 8 designed for the treatment of patients with ornithine transcarbamylase) and DTX401 (treatment of patients with glycogen storage disease type Ia) in clinical development.

Moreover, the company has an agreement with Bayer BAYRY to research, develop and commercialize AAV gene-therapy products for the treatment of hemophilia A (DTX 201).

Zacks Rank & A Stock to Consider

Ultragenyx is currently a Zacks Rank #3 (Hold) stock.

A better-ranked stock in the biotech sector is Incyte Corporation INCY, which currently carries a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Incyte’s earnings per share estimates have increased from $2.44 to $2.57 for 2019 over the past 30 days.

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