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Fda Approval

All articles tagged with #fda approval

FDA Grants Accelerated Approval for KRESLADI to Treat Pediatric LAD-I
healthcare14 days ago

FDA Grants Accelerated Approval for KRESLADI to Treat Pediatric LAD-I

Rocket Pharmaceuticals announced that the FDA granted accelerated approval for KRESLADI (marnetegragene autotemcel), an autologous hematopoietic stem cell–based gene therapy for pediatric patients with severe LAD-I due to ITGB2 variants who lack an HLA-matched donor. Approval is based on increased neutrophil CD18/CD11a expression and will rely on longer-term follow-up data and a post‑marketing registry to confirm benefit. The FDA also awarded Rocket a Rare Pediatric Disease Priority Review Voucher, which the company plans to monetize. LAD-I is ultra-rare and causes recurrent life-threatening infections; safety concerns include infections during conditioning, veno-occlusive disease, engraftment failure, potential insertional oncogenesis, hypersensitivity, anti-retroviral interactions, and possible HIV test false positives, necessitating long-term monitoring. A conference call is planned for March 27, 2026.

via Business Wire|
#fda-approval#gene-therapy#healthcare
Rocket's Kresladi gene therapy wins FDA approval for ultra-rare LAD-1
health15 days ago

Rocket's Kresladi gene therapy wins FDA approval for ultra-rare LAD-1

The FDA approved Rocket Pharma’s one-time gene therapy Kresladi to treat severe LAD-1 in children without a matched sibling donor, following a 2024 manufacturing-based rejection. LAD-1 is ultra-rare (about 1 in a million), with roughly 25 new cases per year, and the therapy is expected to carry a multimillion-dollar price tag but is unlikely to become a major moneymaker.

via statnews.com|
#fda-approval#gene-therapy#health
J&J’s new psoriasis pill could reshape the treatment landscape
healthcare24 days ago

J&J’s new psoriasis pill could reshape the treatment landscape

Johnson & Johnson won U.S. approval for Icotyde, a daily oral medication for moderate-to-severe plaque psoriasis in patients 12 and older, designed to mimic the effects of top-selling injectables like Skyrizi and Tremfya without injections. The pill could expand the patient market and is projected to reach more than $5 billion in peak annual sales, potentially reshaping competition in a multibillion-dollar field.

via statnews.com|
#fda-approval#healthcare#icotyde
FDA narrows leucovorin's promise: approved for cerebral folate deficiency, not autism
health1 month ago

FDA narrows leucovorin's promise: approved for cerebral folate deficiency, not autism

The FDA approved leucovorin only for cerebral folate deficiency caused by pathogenic FOLR1 variants, not as a treatment for autism, despite earlier pitches by officials that it could help many autistic children. The supporting evidence for autism benefit has been weak and a major trial was retracted due to data concerns. The rare genetic form this approval covers affects a small number of cases, and the agency is encouraging more research to assess any broader autism effects while signaling the treatment’s utility remains limited to the specific cerebral folate deficiency condition.

via Gizmodo|
#autism#cerebral-folate-deficiency#fda-approval
FDA approves leucovorin for cerebral folate deficiency in rare patients
health1 month ago

FDA approves leucovorin for cerebral folate deficiency in rare patients

The FDA approved leucovorin (folinic acid) as the first treatment for cerebral folate deficiency—a rare genetic disorder that prevents folate from reaching the brain—in select adults and children. The decision was based on a systematic review of published case data rather than a randomized trial and did not establish broad efficacy for autism, though future autism studies remain possible. The approval covers generic leucovorin and GSK’s Wellcovorin, and regulators urged manufacturers to boost production to meet rising demand; GSK has no plans to relaunch the product.

via CNBC|
#autism#cerebral-folate-deficiency#fda-approval
Brain-implant adapts brain stimulation to ease Parkinson’s tremors
health1 month ago

Brain-implant adapts brain stimulation to ease Parkinson’s tremors

An adaptive deep brain stimulation (aDBS) device—essentially a brain pacemaker that adjusts electrical pulses in real time—has begun to transform Parkinson’s care. After FDA approval in 2025, the implant, tested in international trials, is designed to tailor stimulation to brain activity, reducing tremors and medication side effects for some patients while not offering a cure. The first patient in the pivotal study reported dramatic tremor relief and cognitive improvements, signaling a promising new frontier in personalized neurotherapy.

via CNN|
#adaptive-deep-brain-stimulation#brain-implant#clinical-trial
Boston AI startup forecasts breast cancer risk from mammograms before tumors form
technology1 month ago

Boston AI startup forecasts breast cancer risk from mammograms before tumors form

Boston-based Clairity, led by radiologist Dr. Connie Lehman, has FDA authorization to use an AI system that analyzes mammograms to predict a woman's risk of developing breast cancer, enabling preventive actions and tailored screening. Trained on over 400,000 mammograms with five-year follow-ups and tested on 77,000 images, the deep neural network outperformed traditional risk calculators and could expand to younger ages and more diverse populations, though the model remains a 'black box' in parts; investors include ACE Global Equity and Santé Ventures as Boston's health-tech scene grows.

via The Boston Globe|
#ai-in-healthcare#boston-tech-scene#breast-cancer
FDA Approves Wearable TTFields Therapy for Locally Advanced Pancreatic Cancer
health1 month ago

FDA Approves Wearable TTFields Therapy for Locally Advanced Pancreatic Cancer

The FDA approved Optune Pax, a wearable device delivering abdominal tumor treating fields (TTFields) to treat locally advanced pancreatic cancer in combination with gemcitabine and nab-paclitaxel, based on PANOVA-3 which showed longer median overall survival and pain-free survival with TTFields versus chemotherapy alone; skin reactions were common; the device costs about $21,000 per month and includes patches, a generator, and support services.

via Medscape|
#fda-approval#health#novocure
Wegovy Goes Oral: New Pill Delivers Similar Weight-Loss Benefits as Injection
health2 months ago

Wegovy Goes Oral: New Pill Delivers Similar Weight-Loss Benefits as Injection

The FDA has approved Wegovy as an oral pill for adults, delivering the same active ingredient (semaglutide) as the weekly injection. Because the pill must pass through the digestive system, it’s taken once daily on an empty stomach with a full glass of water and a 30‑minute wait before eating. The pill uses a higher daily dose (25 mg) than the injection (2.4 mg weekly), and trials suggest similar weight‑loss results between forms (about 13.9% with the pill vs 14.4% with the injection). Side effects are primarily gastrointestinal for both forms. The pill is not yet approved for adolescents (12+), can be stored at room temperature, and costs and insurance coverage may influence which form a patient chooses.

via Live Science|
#fda-approval#glp-1#health
Novo Nordisk Launches Wegovy as First Oral Weight-Loss Pill in the U.S.
health3 months ago

Novo Nordisk Launches Wegovy as First Oral Weight-Loss Pill in the U.S.

Novo Nordisk has launched an oral version of its weight-loss drug Wegovy in the U.S., offering a convenient alternative to injections. The pill, containing semaglutide, was approved by the FDA in December and is available for $149 per month for initial doses, with prices increasing later in April. Clinical trials show significant weight loss benefits, making it a notable innovation in obesity treatment.

via CBS News|
#fda-approval#health#novo-nordisk