FDA Action Alert: Jazz, ANI and Ultragenyx


Wrapping up the month of July, there are three PDUFA dates on the U.S. Food and Drug Administration (FDA) calendar, although one of the drugs has already been approved and another company is in the process of refiling their application. Here’s a look.

Jazz Pharmaceuticals’ Drug for Cataplexy

Jazz Pharmaceuticals has a target action date of July 21 for its New Drug Application (NDA) for JZP-258 for cataplexy or excessive daytime sleepiness (EDS) in patients seven years of age and older with narcolepsy. The drug is a novel oxybate product with a unique composition of cations resulting in 92% less sodium than Jazz’s Xyrem (sodium oxybate).

At this time, Xyrem is the only product approved to treat both cataplexy—a sudden loss of muscle tone with retained consciousness—and EDS in patients with narcolepsy ages seven years and older.

“We developed JZP-258 to be a safer and long-term treatment option for patients,” said Robert Iannone, executive vice president, research and development of Jazz, back in March when the FDA accepted the NDA application. “JZP-258 represents between 1,000 and 1,500 milligrams daily reduction of sodium for patients currently treated with Xyrem, depending on the dose. Given the broad scientific consensus that reducing daily sodium consumption is associated with clinically meaningful reductions in blood pressure and cardiovascular disease risk, we believe that JZP-258 has the potential to be an important treatment option for patients living with the life-long condition of narcolepsy. Narcolepsy patients are known to be at increased risk of comorbidities, including obesity, hypertension, diabetes and dyslipidemia.”

ANI Pharmaceuticals’ Purified Cortrophin Gel Prior Approval Supplement

ANI Pharmaceuticals had a target action date of  July 23, 2020, for its supplemental new drug application (sNDA) for Purified Cortrophin Gel (Repository Corticotropin Injection USP). In an announcement in March, when it submitted the sNDA, the company noted the current annual market for the drug is about $950 million and there is only one competitor.

Cortrophin Gel was originally approved by the FDA in 1954 and halted use in the 1980s. The drug has more than 54 indications in its previously approved label, including acute exacerbations of multiple sclerosis, rheumatoid arthritis, systemic lupus erythematosus and ulcerative colitis. The company acquired the NDA from Merck in January of 2016 and spent more than four years and over $100 million to re-establish and validate the commercial corticotropin active pharmaceutical ingredient and the drug’s manufacturing processes. This included rebuilding a U.S.-based supply chain.

On April 28, the FDA issued a Refusal to File (RTF) letter regarding the sNDA. The FDA said on preliminary review, certain portions of the Chemistry, Manufacturing and Controls section were not sufficiently complete to permit a substantive review. On June 1, ANI provided an update and indicated it plans to refile the sNDA when it completes the review process.

Ultragenyx Pharmaceutical’s UX007 for Long-chain Fatty Acid Oxidation Disorders

Ultragenyx Pharmaceuticals has a target action date of July 31 for UX-007 for long-chain fatty acid oxidation disorders (LC-FAOD). On June 30, the FDA approved UX007 (triheptanoin) under the trade name Dojolvi.

LC-FAOD are a group of rare, lifelong and life-threatening genetic disorders. In them, the body cannot convert long-chain fatty acids into energy. Dojolvi is a highly purified, synthetic, 7-carbon fatty acid triglyceride designed to provide medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement for LC-FAOD patients. When people are born in the U.S. and certain European countries, LC-FAOD is included in newborn screening panels because of the risk for serious health issues, including early death. The current treatments include avoiding fasting, low-fat/high-carbohydrates diets, carnitine and even-carbon medium-chain triglyceride (MCT) oil, a medical food product. These diseases affect about 2,000 to 3,5000 children and adults in the U.S.

“FDA approval of Dojolvi for the treatment of long-chain fatty acid oxidation disorders is a huge milestone for patients and their families, as it is the first such medication,” said Jerry Vockley, Professor of Human Genetics and chief of Medical Genetics at Children’s Hospital of Pittsburgh of UPMC. “While we have been able to identify these disorders at birth for many years, treatment options have been limited. Dojolvi brings hope as a new tool now available to clinicians taking care of these patients.”


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