pitolisant
Harmony Biosciences Announces File Acceptance Of Its New Drug Application For Pitolisant
PLYMOUTH MEETING, PA, February 12, 2019 — Harmony Biosciences, LLC (Harmony), announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for its investigational product, pitolisant, and has granted Priority Review for this NDA. Pitolisant is a first-in-class molecule with a novel mechanism of action; it is a potent
and highly selective histamine 3 (H₃) receptor antagonist/inverse agonist for the potential treatment of excessive daytime sleepiness (EDS) and/or cataplexy in adult patients with narcolepsy. A Priority Review designation by the FDA indicates that, if approved, pitolisant would provide a significant improvement in the safety or effectiveness of the treatment of EDS and/or cataplexy in adult patients with narcolepsy when compared to existing treatments. Harmony’s goal is to obtain FDA approval to market pitolisant in the U.S. in 2019.
“The impact of narcolepsy can be significant and severely disruptive to everyday life for up to 200,000 Americans living with this disorder,” said John C. Jacobs, President and CEO at Harmony. “This is an important step for patients and for our company, whose mission is to develop novel treatment options for people living with rare and orphan diseases.”
“Pitolisant offers a novel approach to the treatment of both EDS and cataplexy in patients with narcolepsy, for which there have been no new treatment options in over 15 years,” said Jeffrey M. Dayno, M.D., Chief Medical Officer at Harmony. “We look forward to working with the FDA during its review of the pitolisant NDA, with our hope of being able to offer this new treatment option to help address an important unmet medical need for people living with narcolepsy.”
The NDA submission is based on results from the clinical development program in narcolepsy, which included over 300 patients, some of whom were treated for up to five years. It also included safety data in over 1500 patients across multiple patient populations.
About Pitolisant
Pitolisant is an investigational medication in the U.S. that is not approved by the FDA. It was granted orphan designation for the treatment of narcolepsy, Fast Track designation for the treatment of excessive daytime sleepiness (EDS) and cataplexy in patients with narcolepsy, and Breakthrough Therapy designation for the treatment of cataplexy in patients with narcolepsy. Pitolisant, a first-in-class medication, is a potent and highly selective histamine 3 (H₃) receptor antagonist/inverse agonist; it enhances the activity of histaminergic neurons in the brain that function to improve a patient’s wakefulness and inhibit attacks of cataplexy. It was designed and developed by Bioprojet, who has marketed the product in Europe since its approval by the European Medicines Agency in 2016. Harmony’s goal is to obtain FDA approval to market this new medication in the U.S. in 2019. If approved, pitolisant would represent the first new therapy in the U.S. in over 15 years for the treatment of both EDS and cataplexy in adult patients with narcolepsy.
About Narcolepsy
Narcolepsy is a rare, chronic, debilitating neurologic disorder of sleep-wake state instability that impacts up to 200,000 Americans and is primarily characterized by EDS, cataplexy, and other manifestations of REM sleep dysregulation, which intrude into wakefulness. In most patients, it is caused by the loss of hypocretin, a neuropeptide in the brain that supports sleep-wake state stability. This disorder affects men and women equally, with typical symptom onset in adolescence or young adulthood; however, it can take up to a decade to be properly diagnosed. Narcolepsy can cause significant burden for patients and their families, affecting their ability to perform routine tasks, limit achievement at school and work, impact social relationships and cause impairment in overall quality of life.
About Cataplexy
Cataplexy is one of several symptoms of narcolepsy that represent elements of REM sleep state intruding into wakefulness, characterized by sudden temporary loss of muscle tone. Cataplexy can be subtle, such as drooping of eyelids, or severe, such as knee buckling or total body collapse. Often times, symptoms of cataplexy may go unrecognized because of the subtle nature of the symptoms in some patients, variability of how cataplexy is expressed, and/or lack of patient complaints or physician recognition of the symptoms as manifestations of cataplexy. This symptom of narcolepsy can often cause significant impact on a person’s ability to carry out normal daily functions. Up to two-thirds of all patients with narcolepsy have cataplexy (known as Type 1 narcolepsy); cataplexy is one of the most debilitating symptoms of this chronic, rare neurologic disorder.
Harmony Biosciences, LLC
Harmony Biosciences, LLC, is a private biopharmaceutical company headquartered in Plymouth Meeting, PA. The company was established in October 2017 with a vision to provide novel treatment options for people living with rare and orphan diseases, with an emphasis on central nervous system disorders, starting with patients living with narcolepsy. Harmony is committed to advancing the understanding of narcolepsy and providing information and resources to individuals who live with, and healthcare professionals who treat patients with, this disorder. For
For pregnant women, good nutrition is essential for their health and the baby's health. But many aren't getting adequate amounts of the vitamins and minerals they need, a new study finds.
ReplyDeleteOn the other hand, some of actually taking higher levels of nutrients than is healthy, the same research reports.
And almost all are eating too much salt.
"Many pregnant women do not consume enough of key nutrients: specifically, iron, potassium, calcium, magnesium, zinc and vitamins A, C, D, E, K, B6, folate and choline -- even with the use of dietary supplements," said lead researcher Regan Bailey. She's an associate professor of nutrition science at Purdue University in West Lafayette, Ind.
Almost all pregnant women are at risk of excessive consumption of salt, and many are at risk of excessive consumption of folic acid and iron, especially among those who use dietary supplements, she said.
"It appears that supplements may be necessary for most pregnant women to meet nutrient recommendations," Bailey said. "However, our findings suggest that responsible formulations of prenatal products could help women achieve recommended intakes without the potential for excess."
For the study, Bailey and her colleagues collected data on more than 1,000 pregnant women 20 to 40 years old who took part in the U.S. National Health and Nutrition Examination Survey from 2001 to 2014.
The researchers found that nearly 70% of the women were taking prenatal dietary supplements. But based on the recommended levels of the National Academies of Sciences, Engineering, and Medicine Dietary Reference, some weren't getting enough and some were getting too much.
At least 10% of the women weren't getting enough magnesium, vitamin D, vitamin E, iron, vitamin A, folate, calcium, vitamin C, vitamin B6 and zinc.
Others were getting too much potassium, vitamin K, folic acid, iron, calcium and zinc.
And 95% were consuming too much salt.
"Whether it's consuming too much salt or not getting enough of some minerals and vitamins, not adhering to recommended standards is a significant problem," said Dr. Rahul Gupta, chief medical officer at the March of Dimes.
Taking too much of some vitamins and minerals can be harmful, he added. For example, vitamin Bs can build up in the liver, causing it harm. Too much iron can also result in damage to the liver, heart and pancreas.
Having too little of some vitamins can also hurt women and their infants. For example, having enough folic acid has been shown to help prevent birth defects called neural tube disorders, which can affect the brain and spine, said Gupta, who wasn't involved with the new research.
"During pregnancy, it's critical to have a nutrition discussion with your doctor," he said. That talk should include what supplements the woman is taking and what fortified foods she's eating.
In addition, women need to tell their doctor what additional nutrients -- such as herbal supplements -- they are taking.
"That, along with lab tests, allows the doctor to provide the best informed recommendations for the patient," Gupta said.
It's even better if this discussion takes place before a woman gets pregnant, he added.
Gupta's main concern is with the amount of salt pregnant women are consuming. "We know there is a direct correlation between salt and high blood pressure," he said.
Hypertension during pregnancy can lead to preeclampsia, a pregnancy complication resulting in high blood pressure and damage to liver or kidneys. This can result in premature birth, Gupta said.
"It's critically important to make sure high blood pressure is controlled," he said. "But if the woman is taking in too much salt, it can affect outcomes for the woman and the baby."
The guidelines are well-known, Gupta said, but they need to be compared with what a woman is actually taking.
As Europe deals with its biggest measles outbreaks since the 1990s, U.S. health officials are urging travelers to be up-to-date on vaccination.
ReplyDeleteIn 2018, European countries reported more than 83,500 measles cases, including 74 deaths, according to the World Health Organization (WHO). A majority of cases were in the Ukraine, but Serbia, France, Italy, Greece, the Russian Federation and Georgia were all hard-hit as well.
And in just the first two months of this year, WHO said, more than 34,300 measles cases were reported across the European region.
"This is clearly not waning," said Dr. Kristina Angelo, of the travelers' health branch at the U.S. Centers for Disease Control and Prevention.
Given that, the CDC is urging travelers to make sure they and their kids are up-to-date on measles vaccination before heading to Europe.
The agency has long encouraged international travelers to have all necessary immunizations before taking off.
But, Angelo noted, people often think of Europe as a "low-risk destination" when it comes to infectious disease -- and they may not think to plan a pre-travel health care visit.
So the CDC is "reinforcing" its recommendations to Europe-bound travelers, she said.
Usually, children receive the measles-mumps-rubella (MMR) vaccine between the ages of 12 months and 15 months, then get a second dose between ages 4 and 6 years.
But if they are traveling, the advice is different, the CDC explained.
In that case, babies 6 to 11 months old should get a single MMR dose. Meanwhile, youngsters ages 12 months and up should get two doses, separated by 28 days -- if they have not yet received the standard MMR shots.
As for adults, make sure you have evidence of immunity against measles before traveling, the CDC recommends. That can include written documentation of adequate vaccination, a blood test showing immunity, or your age: People born before 1957 are presumed to be immune because they were probably naturally exposed to measles during outbreaks.
You don't necessarily need to dig through your attic looking for the medical record. States often maintain vaccination records, Angelo noted, and you or your health care provider may be able to access the information that way.
DeleteIf there's no evidence of presumed immunity, the CDC recommends getting the MMR.
The agency lays out its advice in the July issue of Pediatrics.
Dr. Paul Offit, a vaccine expert not involved in the report, agreed that its message is timely.
"Most people don't consider a trip to Western Europe as one in which they might be exposed to a potentially fatal infectious disease," said Offit, who directs the Vaccine Education Center at Children's Hospital of Philadelphia.
"But measles is common in several popular tourist destinations, like Italy and France," he said. "Travelers need to make sure they are up to date on their measles vaccine before getting off the plane."
Why are outbreaks hitting those countries? Low vaccination rates, the CDC says. In some European populations or communities, vaccination rates are below 70%. That's owing to a mix of factors -- from parents' worries about vaccine safety, to religious or cultural beliefs, to regional instability in certain countries, according to the report.
In the United States, Angelo said, most measles cases are related to international travel. Unvaccinated travelers can not only get sick -- but may also bring the infection back with them, potentially spreading it to others who have not been vaccinated.
Measles was considered eliminated in the United States in 2000. But so far this year, the CDC has received reports of 1,044 measles cases nationwide -- the most since 1992. Those cases have been confirmed in 28 states.
According to Angelo, it all underscores the importance of keeping all kids up-to-date on the MMR -- not only those who will be traveling.
Measles is a highly contagious virus, spread through the air when an infected person coughs or sneezes. The symptoms include a high fever, cough and runny nose; the telltale rash appears three to five days after the first symptoms, according to the CDC.
While most people recover, some develop serious complications like pneumonia and brain swelling. Up to 5% of children with measles suffer pneumonia, the CDC says, and one to three out of every 1,000 children with measles dies.