Ultragenyx Pharmaceutical Inc . RARE announced that the FDA accepted its most recent proposal to submit a New Drug Application (NDA) for UX007 for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD) based on existing data.
The company expects to provide more details on timing following a pre-NDA meeting, which is scheduled for the second half of 2018.
The data submitted to the FDA for evaluation included the 78-week phase II study results in 29 patients, retrospective medical record review of 20 patients, and 56 emergency IND cardiomyopathy and other patients. Data from the 78-week phase II study showed a 48.1% reduction in the mean annualized rate of major clinical events (MCEs) and a 50.6% reduction in the median annualized rate of MCEs after 78 weeks of treatment with UX007 compared to an annualized rate of MCEs in the 18 months prior to treatment with UX007. The data was also supported by an investigator study of 32 patients showing an effect of triheptanoin on cardiac function. A 50.3% reduction in the mean annualized duration of MCEs and a 76.7% reduction in the median annualized duration of MCEs following 78 weeks of UX007 treatment was observed.
Earlier, the FDA notified that the data are confounded by the changes in dietary care of patients during the study, and were not sufficient to support an NDA.
Meanwhile, Ultragenyx will discuss these data with the European Medicines Agency (EMA) and expects to update on the same in the second half of 2018.
The candidate is also being studied for the treatment of glucose transporter type-1 deficiency syndrome. A phase III study completed enrollment and data is expected later in 2018.
Shares of Ultragenyx have increased 80.9% year to date against the industry 's decline of 1.7%.
The company also has two approved therapies in its portfolio - Crysvita and Mepsevii.
The European Commission (EC) recently approved the Marketing Authorization Application (MAA) for Mepsevii for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII), also known as Sly syndrome.
Zacks Rank & Stocks to Consider
Ultragenyx has a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Gilead Sciences Inc. GILD , Ligand Pharmaceuticals Inc. LGND and Celgene Corp. CELG . While Gilead and Ligand sport a Zacks Rank #1 (Strong Buy), Celgene carries a Zacks Rank #2 (Buy). You can see the complete list of today's Zacks #1 Rank stocks here.
Gilead's earnings per share estimates have increased from $6.11 to $6.57 for 2018 and from $6.36 to $6.48 for 2019 over the past 60 days. The company delivered a positive earnings surprise in three of the trailing four quarters with an average beat of 6.43%.
Ligand's earnings per share estimates moved up from $4.93 to $5.64 for 2018 and from $5.39 to $5.59 for 2019 in the last 30 days. The company delivered a positive earnings surprise in all of the trailing four quarters with an average beat of 59.5%. Share price of the company has increased 87.4% year to date.
Celgene's earnings per share estimates have increased from $8.76 to $8.52 for 2018 and from $10.18 to $10.65 for 2019 over the past 60 days. The company delivered a positive earnings surprise in all of the trailing four quarters with an average beat of 2.38%.
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