FDA approves new genetic disorder drug
The Food and Drug Administration (FDA) approved a new drug to treat a disorder causing the growth of noncancerous tumors on nerves throughout the body.
The federal agency said on Tuesday it greenlighted SpringWorks Therapeutics's drug Gomekli for patients dealing with neurofibromatosis type 1 (NF1) 'who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.'
The drug was approved after a multicenter, single-arm trial of 114 patients — 58 adults — with 'symptomatic, inoperable NF1-associated PN causing significant morbidity,' according to the FDA.
Gomekli showcased at least a 20 percent reduction in the volume of the tumor in more than 50 percent of pediatric patients and 41 percent of adult patients, Reuters noted.
'The NF1-PN patient community has a great need for more treatment options. With today's approval, we are honored to serve both adults and children with NF1-PN and provide them with a therapy that has the potential to shrink their tumors and offer meaningful symptomatic relief,' SpringWorks CEO Saqib Islam said in a statement on Tuesday.
'We are grateful to each clinical trial participant, their families, the investigators, and the patient advocacy groups involved in the journey towards making GOMEKLI available in the U.S.,' Islam added. 'I am proud that we are delivering on our commitment to patients with devastating diseases with our company's second FDA approval in less than 18 months.'
Copyright 2025 Nexstar Media, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
an hour ago
- Yahoo
Nano-Cap PolyPid's Lead Drug Candidate Cuts Surgical Infection Risk by 38% In Phase 3 Trial
PolyPid Ltd. (NASDAQ:PYPD) on Monday announced topline results from its pivotal SHIELD II Phase 3 trial of D-PLEX100 to prevent surgical site infections (SSIs) in patients undergoing abdominal colorectal surgery with large incisions. The SHIELD II trial is designed to assess the efficacy and safety of D-PLEX100 administered concomitantly with the standard of care (SoC), which includes prophylactic systemic antibiotics, compared to the SoC alone arm in the prevention of post-abdominal-surgery incisional infection in patients undergoing abdominal colorectal surgeries with large incisions. The trial's primary endpoint is measured by the proportion of subjects with a surgical site infection or mortality for any reason within 30 days post-surgery. FDA Pushes Pause On Axsome Fibromyalgia Drug, New Trial Required Patient safety will be monitored for an additional 30 days. The trial will enroll patients in centers in the United States, Europe, and Israel. D-PLEX100, PolyPid's lead product candidate, is designed to provide local, prolonged, and controlled antibacterial activity directly at the surgical site to prevent SSIs. Following the administration of D-PLEX100 into the surgical site, the PLEX (Polymer-Lipid Encapsulation matriX) technology pairs with Active Pharmaceutical Ingredients, enabling a prolonged and continuous release of the broad-spectrum antibiotic doxycycline, resulting in a high local concentration of the drug for 30 days for the prevention of SSIs, with additional potential to prevent SSIs caused by antibiotic-resistant bacteria at the surgical site. The trial met the primary efficacy endpoint, with a significantly lower proportion of primary endpoint events among patients who received D-PLEX100 plus SoC (n=405; 10.9%), compared to SoC alone (n=393; 18.1%), representing a 38% reduction (p<0.005). SHIELD II included three key secondary endpoints: The first key secondary endpoint was met, with a 58% reduction in deep and superficial SSI rates among patients who received D-PLEX100 plus SoC (3.8%) compared to those who received SoC alone (9.5%) (p<0.005). The second key secondary endpoint showed statistical significance in favor of D-PLEX100 plus SoC over SoC alone (p<0.005). The third key secondary endpoint was met with a 62% reduction of patients with an ASEPSIS1 score >20 in the D-PLEX100 plus SoC arm compared to the SoC alone arm (p<0.05). The ASEPSIS score is a clinical tool used to assess surgical wound infections objectively. The independent Data Safety Monitoring Board raised no safety concerns in SHIELD II. The company expects to submit a New Drug Application to the U.S. Food and Drug Administration in early 2026, with a Marketing Authorization Application in the E.U. to follow shortly thereafter. Price Action: PYPD stock is trading higher by 12% to $3.66 at last check Monday. Read Next:Photo by ittawit21 via Shutterstock Up Next: Transform your trading with Benzinga Edge's one-of-a-kind market trade ideas and tools. Click now to access unique insights that can set you ahead in today's competitive market. Get the latest stock analysis from Benzinga? This article Nano-Cap PolyPid's Lead Drug Candidate Cuts Surgical Infection Risk by 38% In Phase 3 Trial originally appeared on © 2025 Benzinga does not provide investment advice. All rights reserved.
Scientific American
an hour ago
- Scientific American
Science-Backed Ways to Stay Healthy During Salmonella Outbreak in Eggs
3 min read To prevent salmonella food poisoning, refrigerate your eggs, cook them well, never eat them raw, and clean, clean, clean By edited by Dean Visser At least 79 people have been sickened by a salmonella outbreak linked to organic and cage-free eggs, distributed under various brand names across seven U.S. states. The eggs were processed by the August Egg Company in Hilmar, Calif., according to the Food and Drug Administration (FDA). On June 6, after FDA tests discovered the bacteria, the company issued a wide recall. The recall is just the latest in a string of callbacks linked to salmonella contamination. On June 7, TGD Cuts of Jessup, Md., recalled cucumber and salsa products, and on June 4 the FDA urged consumers to follow a recall that began in May of tomatoes packaged and shipped by various companies. What is salmonella, how can it make you sick, and how can you minimize your chances of getting it? Here are some basics. On supporting science journalism If you're enjoying this article, consider supporting our award-winning journalism by subscribing. By purchasing a subscription you are helping to ensure the future of impactful stories about the discoveries and ideas shaping our world today. What Is Salmonella and How Does It Spread? Salmonella is a bacterium that thrives in the intestinal tract of certain animals, including chickens and cows—and humans. It is shed through feces. Like many foodborne pathogens, salmonella is often spread by improper cleaning or handling of raw food—including meat, eggs, seafood, vegetables, fruits and rice—at farms or factories. It is often associated with chicken and eggs, although past outbreaks have also been linked to peanut butter, raw pork and beef, cucumbers and even raw flour in cookie dough. When compromised products are shipped, stores and consumers may be unaware of the contamination until enough people start becoming sick and investigators trace the source back to specific food lots. The U.S. Centers for Disease Control and Prevention (CDC) estimates that 48 million Americans get sick from food each year. What Are the Symptoms of Salmonella Food Poisoning? You may begin to feel sick anywhere from eight to 72 hours after you are exposed. As the bacteria multiply, they can cause stomach cramps, diarrhea, vomiting and fever. Children under age five often account for a large percentage of cases in a Salmonella outbreak, and elderly people or those with weakened immune systems are at greater risk. In rare cases people develop joint pain or eye irritation. How Is It Treated? Infections often clear within three or four days, but if diarrhea lasts for more than two days, or body temperature rises above 102 degrees Fahrenheit, health officials urge you to see a doctor. Complications such as diarrhea can cause dehydration, so drink plenty of fluids. How Can I Minimize My Chances of Getting Salmonella? Contaminated food is the chief source, so never eat raw foods that can carry the bacteria. Refrigerate eggs or other susceptible foods at 40 degrees Fahrenheit or less. When preparing meals, use separate cutting boards and knives for suspect foods, and thoroughly wash them—as well as kitchen surfaces and your hands after prep. Microwave dishwashing sponges on high. Cook food to at least 160 degrees Fahrenheit (71 degrees Celsius). Should I Stop Kissing my own Chickens? Birds, hedgehogs, turtles, guinea pigs and even pet bearded dragons can carry Salmonella and make you sick through direct contact, according to a 2024 Scientific American story. The craze among homeowners that expanded during Covid to raise chickens in the backyard is also a culprit. Salmonella bacteria can linger on an animal's skin, so regardless of how cute you think your chickens are, try to not snuggle them or kiss them! Which evidently is a thing.
Yahoo
2 hours ago
- Yahoo
Dilon Technologies, Inc. Closes $9 Million in Growth Capital Financing
NEWPORT NEWS, Va., June 9, 2025 /PRNewswire/ -- Dilon Technologies®, a global leader in medical devices for biosurgery and breast cancer treatment and diagnosis, today announced a $9 million growth capital investment from JGB Management Inc. The investment will support Dilon's continued double-digit revenue growth and operational scaling. Dilon Technologies has built a robust presence in surgical settings with a portfolio of products that improve clinical outcomes. "We are proud to partner with JGB as we advance our mission to deliver evidence-based surgical solutions worldwide," said George Makhoul, CEO of Dilon Technologies. "This funding empowers us to continue delivering best-in-class innovations that improve patient care." HEMOBLAST Bellows® is a groundbreaking advanced hemostatic agent used in a wide range of surgeries—including cardiac, general, and orthopedic procedures. It remains the only FDA-approved product specifically indicated for the control of minimal, mild, and moderate bleeding. Now utilized in several hundred U.S. hospitals, HEMOBLAST® Bellows continues to redefine standards in surgical hemostasis. Another key product, MarginProbe® an in-surgery, real-time breast cancer margin assessment device. It enables surgeons to assess lumpectomy margins intraoperatively, significantly reducing the need for repeat surgeries. Clinical studies show MarginProbe® reduces re-excision rates by 25% to 80%. A 2025 study published in The American Surgeon Journal highlighted a 42% reduction in re-excision for patients with DCIS. A next-generation version of the device has been submitted to the FDA, with approval anticipated by year-end. Dilon's Navigator™ Gamma Probe product line, a market-leading tool for radio-guided lymphatic mapping and tumor localization, continues to perform strongly worldwide. A new, enhanced version is expected to launch by late 2025. "We are pleased to partner with Dilon Technologies to help them grow in their mission to provide surgical solutions worldwide," said Brett Cohen, CEO of JGB Management Inc. For more information, visit About Dilon Technologies® Technologies® Inc. is a commercial-stage medical device company that develops, manufactures, and markets innovative technologies used in hemostasis, cancer detection and assessment, and airway management. Dilon's growing surgical portfolio includes: HEMOBLAST Bellows®: The only FDA-approved powder-form hemostatic agent for minimal, mild, and moderate bleeding. MarginProbe®: A groundbreaking real-time margin assessment tool for breast cancer surgery. Navigator™ System: A leading surgical gamma probe for tumor localization and lymphatic mapping. TrueView 100 Pro: A surgical specimen analyzer for intraoperative use. CoPilot VL+®: A compact, portable video laryngoscope for airway visualization. About JGB Management Inc. JGB Management Inc., founded by Brett Cohen in 2005, is an alternative asset management firm focused on investing in niche credit-oriented situations. JGB specializes in privately negotiated investments. The firm is based in Westport CT. View original content to download multimedia: SOURCE Dilon Technologies Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
