US FDA Accepts Regulatory Submissions for Review of Tafamidis to Treat Transthyretin Amyloid Cardiomyopathy
January 14, 2019 - Pfizer Inc. (NYSE: PFE) announced today that the US Food and Drug
Administration (FDA) accepted for filing the company’s New Drug Applications (NDAs) for tafamidis for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). Pfizer has submitted two NDAs based on two forms of tafamidis: meglumine salt and free acid. Tafamidis is the only product to complete a Phase 3 trial evaluating its efficacy, safety, and tolerability in patients with ATTR-CM, a rare, fatal, and underdiagnosed condition.1,2
The tafamidis meglumine form (20 mg capsule) has been granted Priority Review. The FDA grants Priority Review to medicines that may offer significant advances in treatment or may provide a treatment where no adequate therapy exists. The target Prescription Drug User Fee Act (PDUFA) action date for a decision by the FDA is in July 2019.
The tafamidis free acid form (61 mg capsule) will be under Standard Review. This form is bioequivalent to the 80 mg tafamidis meglumine dose, which was administered as four 20 mg capsules in the pivotal trial; it was developed for patient convenience to enable a single capsule for daily administration. The target PDUFA action date for a decision by the FDA is in November 2019.
“The diagnosis of ATTR-CM is often delayed, primarily because disease awareness is low and patients often present with symptoms similar to more common causes of heart failure. In fact, we believe less than one percent of patients living with this disease are currently diagnosed,” said Brenda Cooperstone MD, Senior Vice President and Chief Development Officer, Rare Disease, Pfizer Global Product Development. “The FDA’s filing acceptance is an encouraging step toward our goal of further raising awareness and providing a treatment option for ATTR-CM patients who are in desperate need of an approved pharmacologic therapy. We look forward to working with the FDA to bring the first treatment for this deadly disease to patients.”
The submission is based on findings from the pivotal Phase 3 Transthyretin Amyloid Cardiomyopathy (ATTR-ACT) study, which evaluated the efficacy, safety, and tolerability of tafamidis meglumine compared to placebo for the treatment of patients with ATTR-CM. In the primary analysis of the study, tafamidis met the primary endpoint, demonstrating a significant reduction in the hierarchical combination of all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo over a 30-month period in patients with wild-type or hereditary ATTR-CM (P=0.0006). Tafamidis was well tolerated, with an observed safety profile comparable to placebo.3 The primary results were presented in a Hot Line session at the ESC Congress 2018 in Munich, Germany, and simultaneously published online in the New England Journal of Medicine (NEJM) in August 2018. Results from additional sub-group analyses were presented during the Late Breaking Clinical Trials session at the Heart Failure Society of America 22nd Annual Scientific Meeting in Nashville, TN, in September 2018. For
You may want to check any frozen blackberries or mixed berries you bought recently: A Georgia-based distributor is recalling products sold at Walmart and Save-A-Lot stores due to potential contamination with norovirus.
ReplyDeleteIn a company statement, Alma Pak of Alma, Ga., stressed that so far there have been no illnesses linked to the bags of frozen blackberries or mixed berries. But "FDA testing of frozen blackberries was reported to have tested positive for norovirus," the company explained.
Norovirus is a highly contagious gastrointestinal illness that can bring on severe nausea, vomiting and diarrhea. The illness typically resolves within two days, but it can prove more long-lasting and dangerous for the elderly, small children and people with compromised immune systems.
The products under recall include 16-ounce bags of berries sold at Walmart as Great Value Frozen Blackberries and 12-ounce bags sold at Save-A-Lot under the brand name Tipton Grove Frozen Mixed Berries.
The Walmart berries were distributed to stores between Jan. 31 and May 31 of this year, and the Save-A-Lot berries under recall were distributed between May 5 and May 15, Alma Pak said in the release.
Berries were distributed to Walmart stores in Florida, Georgia, North Carolina and South Carolina; and to Save-A-Lot stores in Florida, North Carolina, New York, Tennessee and Wisconsin.
Cannabidiol, commonly known as CBD oil, is a chemical in marijuana.
ReplyDeleteIt's increasingly popular among people looking for muscle relaxation and anxiety reduction. But researchers still have a lot to learn about the substance.
MedlinePlus reminds consumers that:
There is insufficient evidence on CBD's effectiveness for most conditions.
Some medications cannot be taken with CBD.
Side effects range from dry mouth to drowsiness.
CBD does not contain the psychoactive component of marijuana.
Proper dosages have yet to be defined.
Nicole and Jonathan Hughes, a teacher and a physician with three young children, were acutely aware of the dangers of swimming pools and lakes. From fenced-off pools to life jackets to constant supervision, they did everything right.
ReplyDeleteTragedy struck anyway.
Last June, as the family was about to head to an Alabama beach for an evening crab hunt, 3-year-old Levi somehow slipped out of their vacation house and found his way to the pool. In what seemed like an instant he was face down in the water, and couldn't be revived.
"We weren't neglectful, but somehow we failed to keep him safe," Nicole said. "Everybody knows about statistics, but the reality is nobody thinks it's going to happen to them."
Nicole, who lives in Bristol, Tennessee, has channeled her grief into activism, speaking out about a leading cause of preventable death among children: drowning.
"There's such a stigma that it's even hard to say the word," she said. "People say it's about 'water safety.' No, it's about drowning. We have to stop tiptoeing around it."
According to the Centers for Disease Control and Prevention, drowning is the No. 1 cause of unintentional death between ages 1 and 4, and the No. 2 cause between ages 5 and 9. The latest national data show 702 U.S. children under age 15 drowned in 2017.
The figures are not broken down by ethnic group, but minorities may be at even greater risk. The USA Swimming Foundation reports 64 percent of African American children and 45 percent of Hispanic children have low or no swimming ability, compared to 40 percent of white children.
The American Academy of Pediatrics recently updated its recommendations for preventing drowning in children, ranging from vigilance at bath time and emptying buckets and wading pools immediately after use to teaching children to swim and encouraging teenagers to learn CPR.
"We've got to do a better job," said Dr. Sarah Denny, lead author of the new guidelines. "Drownings continue to happen. Parents must be aware of the risks and take action to prevent drowning."
Denny, a pediatrician at Nationwide Children's Hospital in Columbus, Ohio, is particularly concerned about young children.
"Toddlers are curious, they're newly mobile, they're exploring their environment and they have no concept of the risk of water," she said.
She stressed that "layers of protection" are key to avoiding tragedy. Those layers include secure fences around pools, close supervision when children are around water, risk assessment and planning for children at every age level, and education, from the pediatrician's office to public service announcements.
ne current announcement features Olympic gold medal skier Bode Miller and his wife Morgan, whose 19-month-old daughter Emmy drowned in a pool in California ; on the same day that Levi Hughes died in Alabama.
Delete"You have to be vigilant, as if it's a lion waiting to snatch your child," Morgan says in the video message.
Nicole Hughes wants to encourage that vigilance with a "Water Guardian" tag, complete with lanyard, that adults can hand off to one another to emphasize the responsibility of supervising children in the water. They're available from her new foundation Levi's Legacy.
"It's another layer of protection," she said. "But the most important goal is just awareness."
If a drowning emergency should occur, "every second makes a difference," said Dr. Vinay Nadkarni, a critical care expert at The Children's Hospital of Philadelphia.
His recommendations start with calling 911 immediately. Get the victim to a safe area "and assess for signs of life. If they're not breathing normally, start CPR."
For drowning victims, the American Heart Association recommends CPR with chest compressions and rescue breaths.
"Prevention is the best cure," but a devastating accident can still happen, Nadkarni said, which makes knowing CPR no less important than a good fence.
"If you do not know CPR," Nadkarni said, "you should not have a pool."