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XTALKS WEBINAR: Keys to Success in ClinicalTrials: A Strategic Guide for Biotechs and Startups Live and On-Demand: Thursday, May 22, 2025 , at 11am EDT (5pm CEST / EU-Central) Register for this free webinar to learn how biotechs can navigate endpoint challenges in clinicaltrials and accelerate their path to drug approval.
The US Food and Drug Administration (FDA) has approved Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator, to treat cystic fibrosis (CF) in patients aged six years and older with at least one F508del mutation or another responsive CFTR mutation.
It is claimed to be both the first re-dosable gene therapy and the first and only FDA-approved treatment for both recessive and dominant types of DEB, a rare and serious geneticdisease affecting the skin and mucosal tissues. The regulatory approval was supported by data from the GEM-1/2 and GEM-3 clinicaltrials.
The Food and Drug Administration on Friday approved the first treatment for Rett syndrome, a geneticdisease mostly affecting girls that causes severe neurologic impairments, robbing them of the ability to communicate or control muscle movement. The new drug, called Daybue, is made by Acadia Pharmaceuticals.
Hympavzi’s approval, with its innovative approach to balancing clotting, could offer a modern solution that adds to the global effort to reduce the risk of thrombosis. This is Pfizer’s second FDA-approved treatment for a rare genetic blood disorder this year.
The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinicaltrial portfolio.
Elevidys is the first FDA-approved gene therapy for DMD a rare genetic disorder characterized by progressive muscle degeneration. To date, over 800 patients have received the therapy in clinicaltrials and real-world settings. Late in 2024, Elevidys was recognized among Times Best Innovations.
In this episode, Ayesha discussed the FDAapproval of Sanofi’s enzyme replacement therapy Xenpozyme for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare genetic lysosomal storage disease, in adults and pediatric patients.
CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinicaltrials go forward for their urea cycle disorder programs late next year. In order to find the specific RNA sequences for a geneticdisease, the biotech is using an AI- and machine learning-based approach.
FDA’s Green Flag to Keytruda and Lenvima combination by Merck and Eisai for Advanced renal cell carcinoma (RCC). The FDAapproved the combination of Keytruda and Lenvima produced by Merck and Eisai, as a first-line treatment of adult patients with advanced renal cell carcinoma (RCC). The rarity of disease – 1.36
This approval also marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare geneticdisease and second FDAapproval for an inherited hemoglobin disorder, cementing our position as a gene therapy leader.”
For the first time in the US, adults living with familial chylomicronemia syndrome (FCS) have an FDA-approved treatment option. In clinicaltrials, it showed up to an 86 percent reduction in triglyceride levels. Investigational drug plozasiran , developed by Arrowhead Pharmaceuticals, is under review for FCS treatment.
Related: Arrowhead Pharmas New Rare Disease Campaign for FCS Highlights Importance of Lowering Triglycerides The FDAsapproval of Crenessity was based on two randomized, double-blind, placebo-controlled trials in 182 adults and 103 children with classic CAH.
“As we strive to help patients with limited or no treatment options, developing innovative treatments in rare diseases is an inspiring challenge and one we have taken on for 70-plus years as a leader in hematology. No adverse events were reported during the clinicaltrials following the administration of Adzynma.
FDAApproves Imcivree (setmelanotide) for Chronic Weight Management in Patients with Obesity Due to POMC, PCSK1 or LEPR Deficiency. Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare geneticdiseases of obesity, announced today that the U.S. BOSTON, Nov.
Amicus Therapeutics has recently received approval from the US Food and Drug Administration (FDA) for its drugs Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) for adults living with late-onset Pompe disease (LOPD) who are not improving on their current enzyme replacement therapy (ERT).
Related: UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD. Below are some facts and information about rare diseases, including rare diseaseclinicaltrials and orphan drugs. How is a Rare Disease Defined? Are Most Rare DiseasesGenetic?
The US Food and Drug Administration (FDA) has awarded approval to Orchard Therapeutics for its gene therapy Lenmeldy (atidarsagene autotemcel) for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).
FDAApproves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.
Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare geneticdiseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.
On September 1, 2022, Boehringer Ingelheim Pharmaceuticals announced in a press release that the US Food and Drug Administration (FDA) approved Spevigo (spesolimab-sbzo) intravenous injections for GPP flares in adults. Spevigo is the first approved treatment option for GPP in adults. ClinicalTrial Behind Spevigo.
Clinicaltrial results reported at this year’s European Haematology Association (EHA) congress showed that a one-time treatment with exa-cel had a significant benefit in both SCD and thalassaemia patients. .
In March, the US Food and Drug Administration (FDA) approved Daybue (trofinetide) , the first and only treatment for Rett syndrome. Daybue (trofinetide) is the first and only FDA-approved drug for Rett syndrome. Dr. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene.
An FDA advisory committee has delivered a blow to Reata Pharma, after voting unanimously that the drugmaker’s data on bardoxolone – a drug for kidney disease – did not show it is effective. Advisors also had some concerns about the safety of the drug and the design of the pivotal trial used to support the marketing application.
The approval was granted to BridgeBio Pharma, Inc. BridgeBio is dedicated to developing therapies for geneticdiseases with unmet needs. The announcement was made on Rare Disease Day, which raises awareness about the impact of rare diseases on patients and their caregivers. Nulibry Trial Results.
We talked about the emergence of dozens of CRISPR biotechs last year and the growing number of CRISPR-based therapies in clinicaltrials for conditions ranging from Duchenne muscular dystrophy (DMD) and sickle cell disease (SCD). The deal is worth $1.5 Prior to this, Lilly struck an agreement worth $1.25
Acromegaly Surrogate Endpoint: Serum growth hormone and serum insulin-like growth factor 1 (IGF-1) are acceptable surrogate endpoints for acromegaly clinicaltrials involving somatostatin analogs such as octreotide, lanreotide and pasireotide. Novartis’ Signifor (pasireotide) is also indicated in the treatment of the disease.
Related: Sebetralstat Gets NDA Accepted by FDA for Hereditary Angioedema Miplyffa was initially turned down by the FDA back in 2021. That submission was accompanied by data from just one clinicaltrial that used a different measure of disease severity known as the 5-domain NPC Clinical Severity Scale (5DNPCCSS).
As of December 16, 2022, there are seven US Food and Drug Administration (FDA) approved gene therapies. In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. In 2023, a number of gene therapies are expected to get the FDA green light.
The impact of CNS diseases extends beyond patients—to their families and society as well.” The assumption is SciNeuro will start with Parkinson’s disease and pain in addition to rare geneticdiseases, after scaling up staffing. .
XTALKS WEBINAR: Bridging Borders: Strategies for Successful Cross-Border Participation in Global Rare DiseaseClinicalTrials Live and On-Demand: Friday, February 28, 2025 , at 10:30am EST (4:30pm CET / EU-Central) Register for this free webinar to gain insights and strategies for success in rare diseaseclinicaltrials without boundaries.
For example, antisense oligonucleotide (ASO)-based therapies have gained traction, with 100 Phase I clinicaltrials initiated and around 25 percent of these advancing to Phase II or Phase III trials in recent years. Furthermore, CRISPR/Cas9 presents a promising avenue for overcoming geneticdiseases in the near future.
The mRNA vaccine candidate developed by Pfizer and Germany-based BioNTech demonstrated 95% efficacy in clinicaltrials. Food and Drug Administration (FDA) will review the data on Dec. The two geneticdiseases cause premature, rapid aging that dramatically decreases the lifespan of children affected.
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