After a decade of deadly delay, the U.S. FDA finally approved Omegaven IV nutrition
Today's story began 12 years ago with the sad story of babies born prematurely, without fully developed intestinal tracts.
As we reported in Fish Oil Saves “Preemie” Babies' Livers and Lives, there was a highly promising therapy for that deadly problem, but it wasn't readily available to American doctors.
Last month, the FDA finally approved use of that remarkable remedy — an IV solution featuring omega-3 fatty acids from fish oil.
Standard IV solutions provide only omega-6 fatty acids, and generally fail to keep young patients with short bowel syndrome healthy — or even alive.
The problem has been that IV solutions containing only omega-6 fatty acids appear to fuel inflammation that promotes "intestinal failure-associated liver disease" or IFALD.
In fact, IFALD afflicts 40% to 60% of infants and 15% to 40% of adults on long-term IV nutrition who are given a standard, omega-6-based feeding solution.
Six years after our initial report, we published a follow-up focused on the FDA’s unconscionable delay in approving the fish-oil-based remedy — see Preemies Saved by Omega-3s, FDA Drags Feet.
The FDA's foot-dragging has finally ended, providing a rewarding ending to a long and frustrating tale of bureaucratic fumbling.
Here's the story behind today's very good news.
Boston team proved that omega-3s save preemies' lives
Back in 1990, famed researcher Judah Folkman, M.D., of Boston's Children's Hospital found that omega-3 fatty acids prevented fat accumulation in the livers of rats.
Five years later, Drs. Jennifer Garza and Mark Puder of Children's Hospital were trying to save two premature infants made very ill by standard “parenteral” (intravenous) nutritional solutions containing only omega-6 fatty acids.
They asked the FDA for special “compassionate use” permission to try a German-made IV nutrition solution called Omegaven® PN, which contains omega-3 fatty acids (DHA and EPA) from fish oil.
Drs. Garza and Puder used it for their very young patients with astonishing success — a success later repeated in other cities.
But each time a young patient was failing on a standard IV nutrition solution, doctors had to request the FDA's “compassionate use” permission to use Omegaven®.
Several years ago, we spoke with Dr. Puder at a conference in Boston, where he expressed extreme frustration over the FDA’s delay in approving this obviously safe, effective solution for sick babies and children.
As Dr. Puder wrote in his 2007 acceptance of the Joan L and Julius H Jacobson Promising Investigator Award, “During my pediatric surgery fellowship, several infants died of IFALD, and this motivated me to help this vulnerable group. In fact, IFALD was one of the leading indications for liver transplantation in children under 4 years of age. It was a slow death, and … several nurses who cared for these children transferred to other nursing units to escape the helplessness they felt knowing that, unless the child was able to wean off PN or get a liver transplant, he or she would ultimately die, usually bleeding to death ...”.
Dr. Puder and his colleagues have treated about two dozen infants and young children with Omegaven IV nutrition. It's usually halted damage to the patients' livers and often restored near-normal liver function.
Fortunately, the FDA has finally approved use of Omegaven IV solution for babies and kids with intestinal failure-associated liver disease (IFALD).
We learned about this landmark development from an August 6, 2018 article in The Boston Globe, in which, as reporter Craig LeMoult wrote, “The FDA approved a drug last month that provides nutrition to children born with a scary condition. That approval is the result of 16 years of work by doctors and researchers at Children's Hospital in Boston.”
LeMoult's report centered on Ellie Brogan, who was born with short bowel syndrome, missing 90 percent of her small intestine and 30 percent of her large intestine.
She’d already developed the yellowed skin and eyes of jaundice — a sign of liver damage — when she was a few weeks old and taken to Boston’s Children’s Hospital.
Ellie’s father Gib related the key moment to the Globe reporter: “‘One day we were sitting at Children's and a doctor knocked on the door and he came in and said, 'My name is Dr. Mark Puder. I have something I'd like to talk to you about.’”
The Brogans agreed to try Omegaven, and as Dr. Puder told The Globe, “Over a relatively short period of time her jaundice cleared and so she improved very nicely, and she was not at risk for having to undergo a transplant.”
Ellie Brogan is now a happy 12-year-old who can enjoy normal activities — as long as she keeps her IV line dry and avoids certain foods.
We’re thrilled that Dr. Puder and his colleagues finally won their long fight to rescue kids with a simple, safe solution — a fight that should’ve ended long ago.
One final note. The FDA has also failed to act on ample evidence that omega-3 fatty acids also appear able to rescue brains damaged by physical or chemical trauma — for more on that, see Miner's Miracle Leads Stellar Omega-3 Summary, Teen's Brain Saved by Omega-3s, Fish Oil May Help Blunt Brain Damage, and Omega-3-Based Regimen Boosted Battered NFL Brains.
- Cerretani J / The Boston Globe. Care for short bowel syndrome helps Ellie enjoy being a kid. March 22, 2018. Accessed at https://thriving.childrenshospital.org/short-bowel-syndrome-care-ellie/
- Kelly DA. Preventing parenteral nutrition liver disease. Early Hum Dev. 2010 Nov;86(11):683-7. doi: 10.1016/j.earlhumdev.2010.08.012.
- LeMoult C / The Boston Globe. For Infants With Rare And Scary Disorder, FDA Approval Of Fish Oil Could Be Life Saving. August 6, 2018. Accessed at https://www.wgbh.org/news/science-and-technology/2018/08/06/for-infants-with-rare-and-scary-disorder-fda-approval-of-fish-oil-could-be-life-saving
- Nehra D, Fallon EM, Puder M. The prevention and treatment of intestinal failure-associated liver disease in neonates and children. Surg Clin North Am. 2011 Jun;91(3):543-63. doi: 10.1016/j.suc.2011.02.003. Epub 2011 Apr 15. Review.
- U.S. Food & Drug Administration. Drugs@FDA: FDA Approved Drug Products. New Drug Application (NDA): 210589. Company: FRESENIUS KABI USA. July 27, 2018. Accessed at https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=210589
- U.S. Food & Drug Administration. Omegaven Single Patient IND Packet. June, 2018. Accessed at https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2018/210589Orig1s000ltr.pdf