The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has granted conditional marketing authorisation to Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel), marking the world’s first approval of a CRISPR-based gene editing therapy. Casgevy has been approved to treat patients with sickle cell disease with recurrent vaso-occlusive crises and transfusion-dependent beta-thalassemia aged 12 years or older, for whom a human leukocyte antigen (HLA) matched related hematopoietic stem cell donor is not available. Also known as exa-cel, Casgevy is currently being evaluated by the US Food and Drug Administration (FDA). The Prescription Drug User Fee Act (PDUFA) target action date for the drug is set for 8 December in severe sickle cell disease and 30 March 2024 in beta-thalassemia, as per a 16 November press release. Casgevy is a genetically modified autologous CD34+ cell population that contains clustered regularly interspaced short palindromic repeats (CRISPR) edited human haematopoietic stem and progenitor ...
Swiss company Versameb will initiate a clinical trial investigating a messenger ribonucleic acid (mRNA) to treat stress urinary incontinence, after receiving clearance from the US Food and Drug Administration (FDA). The FDA approved the company’s investigational new drug (IND) application for a Phase IIa trial of VMB-100 – an intramuscularly locally delivered, sequence-engineered mRNA. The study is scheduled to start in the first half of 2024. Versameb’s candidate encodes human insulin-like growth factor-1 (IGF-1). According to Versameb, preclinical studies have shown that VMB-100 can induce the expression of IGF-1 in human muscle cells, and the candidate accelerated the regeneration of the urinary sphincter muscle and restored urinary sphincter function in animal models of stress urinary incontinence, after a single dose of treatment. Stress urinary incontinence is a condition where there is an involuntary leakage of urine due to a weakened urinary sphincter muscle. It is a condition that particularly affects ...
Takeda’s Adzynma (ADAMTS13, recombinant-krhn) has been approved by the US Food and Drug Administration (FDA) as the first therapeutic option for congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare inherited blood clotting disorder. Estimated to affect fewer than 1,000 people in the US, cTTP is caused by a deficiency in the ADAMTS13 enzyme that regulates blood clotting. Patients with cTTP can experience severe bleeding episodes, strokes and damage to vital organs, and mortality rates are high if left untreated. “Without treatment, cTTP is ultimately fatal,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research. He continued: “[The] approval reflects important progress in the development of much-needed treatment options for patients affected by this life-threatening disorder.” Until now, cTTP treatment has typically involved plasma therapy, which Takeda has previously described as “insufficient in restoring ADAMTS13, time-consuming and costly”. Adzynma, which has been authorised for use as a preventative ...
Candel Therapeutics has announced positive results from a mid-stage trial of its viral immunotherapy candidate in non-metastatic pancreatic cancer. The ongoing phase 2 trial has been evaluating the candidate CAN-2409 plus valacyclovir together with standard of care (SoC) chemo-radiation followed by resection in patients with borderline resectable pancreatic ductal adenocarcinoma (PDAC). Interim results demonstrated an estimated survival rate of 71.4% at months 24 and 36 months in patients who received the CAN-2409 regimen prior to surgery, compared to 16.7% in those receiving SoC chemo-radiation alone. Five out of seven patients who received CAN-2409 were still alive at the time of the data cut-off, with two patients surviving more than 45 months from enrolment, while only one patient randomised to control SoC chemotherapy remained alive at the data cut-off. A “consistent and robust activation of immune response” was also observed after dosing with CAN-2409, the company said, and that the candidate ...
The third quarter marked a momentous one for Sarepta Therapeutics, bringing the long-anticipated launch of its Duchenne muscular dystrophy (DMD) gene therapy Elevidys. After traveling a tumultuous road to the June approval, Sarepta reaped $69.1 million from Elevidys’ first quarter on the market. The number was more than enough to beat analysts’ expectations of between $20 million and $24 million. Sarepta stayed mum on exactly how many patients have received an infusion and instead provided revenue as a marker for uptake, CEO Doug Ingram said on Sarepta’s third-quarter earnings call. The drug is priced at $3.2 million per infusion. Also on the call, Sarepta reiterated its confidence that its phase 3 EMBARK study can potentially support a label expansion for Elevidys despite missing its primary endpoint. The company’s discussions with FDA leadership have shown that the agency is open to the idea of a label expansion “if supported by review ...
By Tristan Manalac Sarepta Therapeutics on Monday posted topline data from the Phase III EMBARK study, showing that its Duchenne muscular dystrophy gene therapy Elevidys (delandistrogene moxeparvovec-rokl) fell short of its primary efficacy endpoint, unable to significantly improve functional mobility versus placebo. Nonetheless, based on what the company calls “robust evidence” of “clinically meaningful treatment benefit” across all EMBARK’s pre-specified key functional secondary endpoints, Sarepta will push through with filing for a label expansion for Elevidys. “We have shared the EMBARK topline results with FDA leadership and they have confirmed that, based on the totality of the evidence, they are open to such label expansion if supported by review of the data, and that they intend to proceed rapidly with consideration of the submission,” Sarepta CEO Doug Ingram said in a statement. In EMBARK, a randomized, two-part crossover and placebo-controlled study, Sarepta dosed 125 Duchenne muscular dystrophy (DMD) patients aged ...
BY SEAN WHOOLEY The Allia IGS Pulse system. [Image courtesy of GE HealthCare] GE HealthCare (Nasdaq:GEHC)+ announced today that the FDA granted 510(k) clearance to its Allia IGS Pulse system. The latest addition to GE HealthCare’s image-guided system (IGS) offerings features a new imaging chain. GE HealthCare says it engineered this feature to provide exceptional imaging at the right dose. This enables visible impact in complex cardiology interventions regardless of patient size. Allia IGS Pulse provides a personalized workspace to meet the operator’s specific needs and preferences. As part of the new image chain, the system features a monopolar X-ray tube used to capture images for interventional procedures. GE HealthCare says the tube — the first of its kind — provides powerful, yet quiet imaging capabilities. Its small footprint also helps clinicians reach steep angulation for better understanding of the coronary artery anatomy. More about the GE HealthCare Allia IGS ...
Despite the promise of cell therapies, manufacturing the personalized medicines at scale has been a limiting factor for many companies. Now, as BlueRock Therapeutics advances its lead prospect, its parent company Bayer is ready to kick production into high gear.Bayer on Tuesday opened its first Cell Therapy Launch Facility in Berkeley, California, which is expected to create capacity to bring cell therapies to patients worldwide. Bayer has invested $250 million to build the plant, which will initially crank out materials for late-stage clinical trials across 100,000 square feet of space, the drugmaker said in a release. The plant is also equipped to support the potential commercial launch of BlueRock’s experimental cell therapy for Parkinson’s disease, bemdaneprocel. At the moment, BlueRock says planning is underway for its phase 2 study of bemdaneprocel, which is expected to start enrolling patients in the first half of 2024. The plant features flexible, modular space ...
Nanobiotix has shared positive new data from an early-stage study evaluating a drug designed to enhance the efficacy of radiotherapy in patients with locally advanced pancreatic cancer. Results from the phase 1 study of NBTXR3, which is being conducted as part of an ongoing collaboration between Nanobiotix and the University of Texas MD Anderson Cancer Center, were presented at this year’s American Association for Cancer Research Special Conference on Pancreatic Cancer. Pancreatic ductal adenocarcinoma (PDAC) is associated with a poor prognosis and remains one of the leading causes of cancer-related death worldwide. For more than 90% of patients with locally advanced disease who are not eligible for surgery, there are few treatment options with curative intent, and the five-year overall survival rate for patients with unresectable PDAC remains less than 5%. Radiotherapy is one of the most common treatment modalities for cancer, with approximately 50% of patients undergoing it at ...
Agomab Therapeutics has received the US Food and Drug Administration (FDA)’s fast track designation for AGMB-129 to treat fibrostenosing Crohn’s disease (FSCD). Designed to inhibit ALK5 in the gastrointestinal (GI) tract, AGMB-129 is an oral, small molecule GI-restricted ALK5 (TGF-βRI) inhibitor currently in clinical development. The single and multiple-dose AGMB-129 was well-tolerated at all doses in a Phase I study in healthy participants. The company has also announced the commencement of the STENOVA Phase IIa clinical study to assess AGMB-129 for patients with symptomatic FSCD. This global, placebo-controlled, multi-centre, double-blind study will assess 36 patients with symptomatic FSCD at investigational sites in Europe, Canada and the US. Subjects will be randomised to receive one of two doses of AGMB-129 or a placebo for a period of 12 weeks. Tolerability and safety of AGMB-129 in FSCD patients are the primary endpoints of the trial. The pharmacokinetics and target engagement at the ...
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