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Fda Approval

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FDA approves first-in-class Auvelity for agitation in Alzheimer’s disease
health25 days ago

FDA approves first-in-class Auvelity for agitation in Alzheimer’s disease

The FDA has approved Axsome Therapeutics’ Auvelity (dextromethorphan–bupropion) as a first-in-class treatment for agitation in Alzheimer’s disease, targeting NMDA and sigma-1 receptors. Agitation affects up to about 70% of patients and worsens caregiver burden and mortality. In phase 3 trials ACCORD-2 and ADVANCE-2, Auvelity delayed relapse of agitation compared with placebo (ACCORD-2: hazard ratio 0.275, P=0.001; relapse 8.4% vs 28.6%). ADVANCE-2 did not meet its primary CMAI endpoint but showed numerical improvements. Auvelity had FDA Breakthrough Therapy designation and an sNDA was submitted in 2025. Experts say this provides a meaningful new option with a favorable safety profile, potentially easing patient and caregiver burden.

via Psychiatric Times|
#agitation#alzheimer-disease#axsome-therapeutics
FDA Clears Non-Antipsychotic Help for Agitation in Alzheimer’s Dementia
health25 days ago

FDA Clears Non-Antipsychotic Help for Agitation in Alzheimer’s Dementia

The FDA expanded the use of Auvelity (dextromethorphan hydrobromide and bupropion hydrochloride) to treat agitation in adults with Alzheimer’s disease–related dementia, marking the first non-antipsychotic approved for this symptom. In two randomized trials, Auvelity improved agitation measures and extended time to relapse with continued treatment. Common side effects include dizziness, upset stomach, headache, diarrhea, drowsiness, dry mouth, sexual dysfunction, and sweating; there is a boxed warning for suicidality in adolescents/young adults and risks of seizures, high blood pressure, and mood activation. Pre-treatment assessments include blood pressure checks and bipolar-history screening. The approval was granted to Axsome Therapeutics, supported by breakthrough therapy and priority review designations.

via fda.gov|
#agitation-in-dementia#alzheimers-disease#auvelity
Alzheimer's blood tests spark debate over predicting risk and guiding care
health29 days ago

Alzheimer's blood tests spark debate over predicting risk and guiding care

Alzheimer's blood tests that detect amyloid or tau are being considered for early risk assessment, but doctors say their predictive value for asymptomatic people is uncertain, with potential false positives/negatives and no clear benefit from early intervention; FDA has cleared two tests for symptomatic patients, not for screening; patient interest is high, with surveys showing many would take a test if advised, though distress is common.

via NBC News|
#alzheimers#amyloid#blood-tests
FDA backs first gene therapy to treat a rare genetic deafness (Otarmeni) with free US access
health1 month ago

FDA backs first gene therapy to treat a rare genetic deafness (Otarmeni) with free US access

The FDA approved Otarmeni, Regeneron’s gene therapy for OTOF-related deafness—the first gene therapy for this rare inherited hearing loss—delivered via a single cochlear injection to restore otoferlin production. In a 20-child trial, several participants showed hearing improvements, with five of 12 followed for 11 months reaching near-normal hearing. Regeneron will provide Otarmeni at no cost to US patients, and the therapy was fast-tracked under the Commissioner’s National Priority Voucher program. While promising, effects were generally safe, the condition affects about 50 US babies born each year, and this advance could pave the way for more gene therapies and potentially reduce reliance on cochlear implants.

via New York Post|
#fda-approval#gene-therapy#health
FDA approves Otarmeni, first gene therapy for rare OTOF-related infant hearing loss
health1 month ago

FDA approves Otarmeni, first gene therapy for rare OTOF-related infant hearing loss

FDA approves Regeneron's Otarmeni, the first gene therapy for a rare OTOF-mutation–driven hearing loss in infants; in a trial, a single-dose inner-ear injection improved hearing in 16 of 20 children, with some able to detect whispers; the company will provide the drug for free to U.S. patients, while pricing outside the U.S. has not been determined since it is not approved there.

via NBC News|
#fda-approval#gene-therapy#health
Merck’s IDVYNSO earns FDA nod as a once-daily, two-drug HIV regimen
health1 month ago

Merck’s IDVYNSO earns FDA nod as a once-daily, two-drug HIV regimen

FDA approved IDVYNSO, a once-daily two-drug regimen (doravirine/islatravir) for adults with virologically suppressed HIV-1, intended to replace their current antiretroviral therapy. It is the first non-INSTI, tenofovir-free two-drug therapy to demonstrate non-inferiority to a three-drug regimen in two Phase 3 trials (052 vs BIKTARVY and 051 vs baseline ART). The product will be available in pharmacies after May 11 and carries contraindications with strong CYP3A inducers and with lamivudine or emtricitabine, plus safety considerations including potential severe skin reactions and drug interactions. Trials enrolled 708 participants with diverse demographics, supporting another option in HIV treatment.

via Merck.com|
#fda-approval#health#hiv
FDA Grants Accelerated Approval for KRESLADI to Treat Pediatric LAD-I
healthcare1 month 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
health2 months 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
healthcare2 months 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
health2 months 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
health2 months 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
health2 months 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
technology3 months 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