logo
US FDA declines to approve Ultragenyx's gene therapy for rare genetic disorder

US FDA declines to approve Ultragenyx's gene therapy for rare genetic disorder

Reuters11-07-2025
July 11 (Reuters) - The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's (RARE.O), opens new tab experimental gene therapy to treat a rare inherited disorder, citing manufacturing concerns, the company said on Friday.
Shares of the California-based biopharma company fell 5.4% in extended trading.
The FDA in its "complete response letter" sought additional information related to the company's production processes and facilities. The regulator's decision comes more than a month ahead of the previously announced action date of August 18.
The agency's observations, related to facilities and processes, are addressable and not directly related to the therapy's quality, Ultragenyx said, adding that many of the issues have already been addressed.
"It looks like this is a speed bump to approval, rather than a roadblock," Leerink Partners analyst Joseph Schwartz said in a client note.
The therapy, UX111, was developed to treat a common type of Sanfilippo syndrome — a group of genetic conditions that begin in early childhood causing severe brain damage and early death.
The treatment involves managing symptoms as there are currently no approved disease-modifying medicines.
Ultragenyx said the FDA did not cite any review issues related to the clinical data submitted as part of the marketing application.
The company's application for UX111 was based on trial data that showed the gene therapy significantly reduced toxic buildup in the brain and improved cognitive and communication skills in children with Sanfilippo syndrome type A.
It plans to resubmit updated clinical data from current patients after resolving the FDA's concerns. A new review could take up to six months once the revised application is filed.
Ultragenyx acquired the global rights of the therapy from Abeona Therapeutics (ABEO.O), opens new tab through an exclusive license agreement on 2022.
Orange background

Try Our AI Features

Explore what Daily8 AI can do for you:

Comments

No comments yet...

Related Articles

Union Pacific and Norfolk Southern confirm merger talks to create coast to coast railroad
Union Pacific and Norfolk Southern confirm merger talks to create coast to coast railroad

The Independent

time24 minutes ago

  • The Independent

Union Pacific and Norfolk Southern confirm merger talks to create coast to coast railroad

Union Pacific and Norfolk Southern acknowledged Thursday that they were in merger talks that would create a single U.S railroad with rails stretching service from East to West Coasts. The Associated Press reported last week that the companies were discussing a tie-up but neither company confirmed until Thursday morning. The potential merger would combine the largest and smallest of the country's six major freight railroads. There's widespread debate over whether such a merger would be approved by U.S. regulators, which have established a high bar for consolidation in the crucial industry. THIS IS A BREAKING NEWS UPDATE. AP's earlier story follows below. Union Pacific reported Thursday that its adjusted profit grew to $1.8 billion in the second quarter as merger rumors to swirl around the nation's largest railroad. The Omaha, Nebraska, per-share earnings rose rose to $3.03, beating Wall Street expectations and easily topping the $2.71 per-share profit it reported in the same period last year. Analysts were expecting profit of $2.91 per share for the recent quarter. Operating revenue grew 2% over last year, to $6.2 billion, the company said. Union Pacific shares rose about 1% before the bell Thursday, to $233.30 each. They had slumped to around $208 in early April, their lowest level of 2025, as President Donald Trump rolled out sweeping tariffs that threatened to upend global trade. Shares of all U.S. railroads moved higher as well. Sources told The Associated Press last week that Union Pacific and Norfolk Southern are in merger talks that would create the a railroad in North America that essentially connects the East and West Coasts. Neither company has commented on the negotiations, which began during the first quarter of this year, according to a person familiar with the talks who wasn't authorized to speak publicly about them. It would combine the largest and smallest of the country's six major freight railroads. There is widespread debate over whether such a merger would be approved by U.S. regulators, which have established a high bar for consolidation in the crucial industry. That is largely because of a disastrous deal that involved Union Pacific. Union Pacific merged with Southern Pacific in 1996 and the tie-up led to an extended period of snarled rail traffic on U.S. rails. Three years later, Conrail was divvied up by Norfolk Southern and CSX, which led to more backups on rails in the East. However, just two years ago the U.S. Surface Transportation Board did approve a deal that created the CPKC railroad, allowing Canadian Pacific to acquire Kansas City Southern for $31 billion. That merger combined the two smallest major railroads in North America, however, but left only six major freight railroads. It was the first major rail merger approved in more than two decades. To be approved, any major rail merger must show it will enhance competition and serve the public interest under the 2001 rules. The CPKC merger was not judged under those rules because Kansas City Southern had an exemption from them as the smallest major freight railroad at the time.

Alcohol use has increased among women — and so have cases of liver disease
Alcohol use has increased among women — and so have cases of liver disease

The Independent

time24 minutes ago

  • The Independent

Alcohol use has increased among women — and so have cases of liver disease

Alcohol-related liver disease has more than doubled in the U.S. over the last 20 years. The increase is tied to four groups that make up a greater share of heavy drinkers than they did two decades ago: Women, adults ages 45 and older, people living in poverty, and those with metabolic syndrome. Metabolic syndrome refers to a number of conditions, including high blood pressure, blood sugar, and cholesterol, that increase the risk of heart disease, stroke and type 2 diabetes. Exactly why these groups are drinking more remains unclear. Vanderbilt University School of Medicine's Dr. Peter Martin previously told NBC News that 'it's become more and more socially acceptable for women to drink as much as men' and George Koob, director of the National Institute on Alcohol Abuse and Alcoholism, told The New York Times that he believed older Americans are even less 'likely' to understand the hazards of alcohol. 'Alcohol-related liver disease is the main cause of liver-related death and these results are a major wakeup call to the dangers of drinking,' researcher Dr. Brian Lee, a hepatologist and liver transplant specialist at Keck Medicine of the University of Southern California, said in a statement after the new study published Wednesday. The findings, Lee said, provide the first comprehensive look at the demographics of heavy drinking and their relation to liver disease since the 1990s. Because the average drinking rate in the U.S. was unchanged over the last 20 years - outside of the pandemic - it suggested factors such as changing health and demographics may be playing a role. The researchers analyzed data from the National Health and Nutrition Examination Survey of adults and children in the U.S. from 1999 through 2020. They tracked the total increase in significant liver disease, a point when scar tissue impairs the organ's function, often caused by heavy drinking. More than 51,600 adults died from liver disease in 2020 in the U.S. The researchers looked at the demographic and health profiles of adults, age 20 or older, who drank heavily - eight drinks per week for women and 15 for men, according to the Centers for Disease Control and Prevention. Previous research had tied the four groups looked at in this study to a higher risk of liver disease when exposed to alcohol. A separate 2024 study, authored by Lee, found that heavy drinking rose at the pandemic's peak and continued for two years after that. Lee hypothesized that increase may have been due to stress. Liver disease deaths have also roughly doubled over the last 20 years, and the number of annual alcohol deaths due to cancer has doubled in the same time period. Lee believes the results will help to provide doctors with necessary updates to better treat patients and potentially result in more screenings and interventions for Americans in high-risk populations. 'Our results show that the makeup of the American public with heavy alcohol consumption has changed compared to 20 years ago,' he said.

UnitedHealth says it is under a federal investigation and cooperating
UnitedHealth says it is under a federal investigation and cooperating

The Independent

time24 minutes ago

  • The Independent

UnitedHealth says it is under a federal investigation and cooperating

Shares of UnitedHealth Group slipped Thursday after the health care giant said it was under a Department of Justice investigation. The company said it has started complying with both criminal and civil requests from federal investigators and it was cooperating with them. '(UnitedHealth) has a long record of responsible conduct and effective compliance,' the company said in a Securities and Exchange Commission filing. Earlier this year, The Wall Street Journal said federal officials had launched a civil fraud investigation into how the company records diagnoses that lead to extra payments for its Medicare Advantage, or MA, plans. Those are privately run versions of the government's Medicare coverage program mostly for people ages 65 and over. The company's UnitedHealthcare business covers more than 8 million people as the nation's largest provider of Medicare Advantage plans. The business has been under pressure in recent quarters due to rising care use and rate cuts. UnitedHealth Group Inc. said in February that it wasn't aware of the start of any new activity as the paper reported. The company said Thursday that it reached out to the Justice Department 'after reviewing media reports about investigations into certain aspects of the company's participation in the Medicare program.' UnitedHealth runs one of the nation's largest health insurance and pharmacy benefits management businesses. It also operates a growing Optum business that provides care and technology support. Company shares have mostly shed value since December, when UnitedHealthcare CEO Brian Thompson was fatally shot in midtown Manhattan on his way to the company's annual investor meeting. The stock price dropped 2%, or $6.13, to $286.50 Thursday morning.

DOWNLOAD THE APP

Get Started Now: Download the App

Ready to dive into a world of global content with local flavor? Download Daily8 app today from your preferred app store and start exploring.
app-storeplay-store