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Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.
Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).
FDAApproves Merck’s ENFLONSIA™ to Protect Infants from Severe RSV Illness Merck operating as MSD outside the United States and Canada, has received a significant regulatory milestone with the U.S. Food and Drug Administration (FDA) granting approval for ENFLONSIA™ (clesrovimab-cfor).
Biogen Launches Global Phase 3 PROMINENT Trial Evaluating Felzartamab in Primary Membranous Nephropathy Biogen Inc. The trial is expected to complete in 2029. By selectively depleting these cells, felzartamab offers a promising, mechanism-driven approach to potentially halting disease progression.
FDAApproves Label Update for Lilly’s Amyvid, Expanding Its Role in Alzheimer’s Disease Diagnosis and Therapy Guidance In a major development for Alzheimer’s diagnostics, Eli Lilly and Company (NYSE: LLY) announced that the U.S.
FDAApproves ANDEMBRY (garadacimab-gxii) The First Once-Monthly Prophylactic HAE Therapy Targeting Factor XIIa CSL a leading biotechnology company with a strong track record of developing innovative medicines for patients with rare and serious disorders, today announced that the U.S.
Ionis Begins Pivotal Phase 3 REVEAL Study of ION582 in Angelman Syndrome, Dosing First Patient in Global Trial Ionis Pharmaceuticals , Inc. This trial will build on previous positive data from the earlier Phase 1/2 HALOS study and is designed to rigorously assess the potential of ION582 as a disease-modifying therapy. “We
Xywav: A Low-Sodium Alternative with FDAApproval Xywav is a uniquely formulated, low-sodium oxybate therapy, and remains the only product of its kind approved by the U.S. It is also approved for adult patients with idiopathic hypersomnia (IH). According to Dr. Richard J.
2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1] 5] In China, it was conditionally approved in 2023 for the treatment of NSCLC and full approval is contingent on results of phase 3 clinical trials. [6] Sunvozertinib CAS 2370013-12-8 DZD9008, 584.1 Sunvozertinib shows IC 50 s of 20.4,
Over 70% of new patients are diagnosed with stage IV disease, also called extensive stage disease (ES-SCLC). Tarlatamab triggered an ORR of ~40% in refractory (second-line or later) SCLC patients in the DeLLphi-301 clinical trial published in 2023. The overall survival rate (OS) at 5 years post-diagnosis is about 3% for ES-SCLC.
The announcement is particularly relevant given the lack of FDA-approved treatment options for AMR, a complication that poses a critical threat to transplant success and long-term graft survival. The company has emphasized that its investigational therapies are designed not only to control symptoms but to alter the course of disease.
Jump up to: a b c d e f g h i j k l “FDAapproves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene” (Press release). Food and Drug Administration (FDA). “Biogen’s antisense ALS drug shows promise in early clinical trial” FierceBiotech. 25 April 2023.
FDAApproves Dupixent as the First and Only Targeted Therapy for Adults with Bullous Pemphigoid In a groundbreaking development for patients suffering from a rare and debilitating autoimmune skin disease, the U.S. This results in separation of skin layers and subsequent blister formation. receiving placebo.
There is no cure or effective treatment for this rare genetic disease, but new research suggests a potential path to one.& & The new study, published in Cell , is the first time prime editing has been used to treat a neurological disease in animals, offering hope for treating people with AHC and other genetic brain disorders.
Based on a technology developed by Broad Institute core member David Liu’s laboratory, the treatment is the first in a series of new medicines being tested to treat rare diseases by repairing patients’ particular genetic misspellings. s to be produced at scale, making them the standard of care for life-threatening rare genetic diseases.
3] Inavolisib was approved for medical use in the United States in October 2024. [3] 19] Society and culture Legal status In October 2024, the US Food and Drug Administration (FDA) approved inavolisib for the treatment of PIK3CA -mutant breast cancer based on the results from the INAVO120 trial. [3] 3 November 2006.
2] Fitusiran was approved for medical use in the United States in March 2025. [2] 1] [2] Adverse effects The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal). [2] 26 March 2025.
2] Elacestrant was approved for medical use in the United States in January 2023, [1] [2] [5] [6] and in the European Union in September 2023. [3] 2] The FDA granted the application for elacestrant priority review and fast track designations. [2] Food and Drug Administration (FDA). 1] [4] It is taken by mouth. [1] PMC 9340905.
The $400,000 prize, to be shared among the four winners, recognizes their role in creating and advancing a technology that has reshaped how physicians treat leukemia, lymphoma, and multiple myeloma, and is now showing promise in treating autoimmune and infectious diseases. In the early 2000s, both teams began preparing for clinical trials.
The study, which is the largest to date that focuses exclusively on this patient population, shows high overall response rates (ORR) alongside deep and durable responses, offering much-needed hope for individuals whose disease typically carries a poor prognosis. Head of Myeloma Unit at Tel-Aviv Sourasky Medical Center in Israel.
7] Pirtobrutinib was approved for medical use in the United States in January 2023, [4] [8] [9] [10] and in the European Union in November 2023. [2] 4] The trial was conducted at 49 sites in 10 countries in the United States, Europe, Australia, and Asia. [6] 6] The same trial was used to assess safety and efficacy. [6]
Food and Drug Administration’s (FDA) accelerated approval of Lynozyfic™ (linvoseltamab-gcpt), a first-in-class BCMAxCD3 bispecific antibody, for the treatment of adults with relapsed or refractory (R/R) multiple myeloma (MM). Lynozyfic demonstrated early, deep, and durable responses in heavily pre-treated patients. . In the U.S.
Food and Drug Administration (FDA)-approved gene therapy for the treatment of Duchenne muscular dystrophy (DMD). The ultimate aim is to enable the administration of ELEVIDYS safely while honoring its potential to provide a dramatic and durable therapeutic benefit for children battling this profoundly challenging disease.
Project Facilitate A single point of contact call and information Help oncology healthcare providers or regulatory professionals with submitting an Expanded Access Request through FDAs Expanded Access Program for an individual cancer patient. Provides the Oncology Dosing Toolkit. Sponsors can request Project Orbis for their applications.
5] Leniolisib was approved for medical use in the United States in March 2023. [5] 5] [7] [8] It is the first approved medication for the treatment of activated PI3K delta syndrome. [5] 5] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [9] “Leniolisib: First Approval” Drugs.
1] [2] Adverse effects The FDA prescribing information for taletrectinib includes warnings and precautions for hepatotoxicity, interstitial lung disease/pneumonitis, QTc interval prolongation, hyperuricemia, myalgia with creatine phosphokinase elevation, skeletal fractures, and embryo-fetal toxicity. [1] 26 (6): 765–772. PMID 40170301.
The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDAapproval, in 2023. Gray, in London to discuss the significance of her recovery at the Third International Summit on Human Genome Editing , described Casgevy as “a new beginning for people with sickle cell disease.”
2] Crinecerfont was approved for medical use in the United States in December 2024. [2] 2] [3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [4] 2] Crinecerfont was approved for medical use in the United States in December 2024. [2] Food and Drug Administration (FDA) (Press release).
Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. But even now, more than a century later, TB remains the deadliest infectious disease on Earth, killing about 1.2 million people every year. Subscribe to Asimov Press.
FDA Grants Priority Review to Merck’s WINREVAIR™ Based on Landmark ZENITH Trial Showing Dramatic Reduction in Morbidity and Mortality in PAH Patients Merck (NYSE: MRK), operating as MSD outside the U.S. label of WINREVAIR based on compelling new evidence from the Phase 3 ZENITH trial. risk score of 9 or greater.
Clinical Trials: Pritelivir is currently in phase II clinical trials, with ongoing research into its effectiveness and safety. Clinical Trials: Pritelivir mesylate is currently under extensive study to evaluate its efficacy and safety profile, with promising results in early clinical trials. References ^ Wilck, M.B.;
FDAApproves KEYTRUDA® (Pembrolizumab) for Perioperative Treatment of Resectable Locally Advanced Head and Neck Squamous Cell Carcinoma Merck known as MSD outside the United States and Canada, recently announced that the U.S. 0.89; p = 0.00140).
Amodei also imagines the ways AI could accelerate biological research and yield miraculous cures in the 21st century; everything from the prevention and treatment of nearly all infectious and inherited diseases to the elimination of most cancers. Human constraints also play a role at a later stage, when clinical trials are involved.”
Deep Dive Library Events Press Releases Topics Sign up Search Sign up Search Pharma Biotech FDA Clinical Trials Deals Drug Pricing Gene Therapy An article from Dive Brief Moderna COVID vaccine gets full approval for children The approval comes amid regulatory upheaval under HHS head Robert F. Kennedy Jr. Kennedy Jr.
FDA Expands Approval of AbbVie’s MAVYRET® as First and Only 8-Week Treatment for Acute Hepatitis C in Adults and Children Aged 3 and Above AbbVie has received a significant regulatory boost for its hepatitis C treatment portfolio as the U.S. Safety outcomes from the trial further bolster the confidence in MAVYRET’s use.
Explain the FDAApproval Process Many patients are unaware of the rigorous approval process generic drugs must undergo. Educate them about the FDA’s role in ensuring the safety and efficacy of generic medications. These anecdotes can help alleviate fears and build confidence in generic medications. Shrank, W.H.,
TUESDAY, July 2, 2024 -- A new drug to treat Alzheimer's disease was approved by the U.S. In clinical trials, donanemab (Kisunla) modestly slowed the pace of thinking declines among patients in the early. Food and Drug Administration on Tuesday.
TUESDAY, July 2, 2024 -- A new drug to treat Alzheimer's disease was approved by the U.S. clinical trials, donanemab (Kisunla) modestly slowed the pace of thinking declines among patients in the early. Food and Drug Administration on Tuesday.In
A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. ITF Therapeutics provides the new drug in the US. Hyperbole or Hope?
Pulmonary arterial hypertension (PAH) is a progressive and fatal lung disease that is caused or influenced by multiple factors. Their early definition — as well as plans for recording and tracking — is a major factor in a trial’s success. Historically, the available drugs and U.S. None offers a cure for PAH.
Abstract Human immunodeficiency virus (HIV) causes acquired immunodeficiency syndrome (AIDS), a lethal disease that is prevalent worldwide. Later, a new type of non-nucleoside reverse transcriptase inhibitors (NNRTIs) were approved as anti-HIV drugs. Molecular insights of HIV Reverse transcriptase and it's inhibitors.
Food and Drug Administration (FDA) for the treatment of adult patients with deleterious or suspected deleterious BRCA -mutated ( BRCA m) metastatic castration-resistant prostate cancer (mCRPC). Patients should be selected for therapy based on an FDA-approved companion diagnostic for LYNPARZA. In the U.S., For the U.S.
The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. Earlier results were published in Nature Medicine.
Approval is based on positive Phase 3 data demonstrating improvements in key disease burden measures and establishing its safety profile Nexviazyme specifically targets the M6P receptor, the key pathway for enzyme replacement therapy, to effectively clear glycogen build-up in muscle cells.
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