Cambridge researchers develop robotic nerve devices for neurological conditions

Neurological disorders, such as epilepsy or chronic pain, affect over three billion people worldwide Researchers at the University of Cambridge have developed small, flexible devices, combining electronics and soft robotics, to help treat a range of neurological conditions, including epilepsy and chronic pain. Published in Nature Materials, the nerve cuff implant has the ability to change shape through electrical activation, opening up a variety of avenues for new, highly targeted treatment options. Affecting more than three billion people worldwide, neurological disorders are conditions that affect the brain as well as the nerves found throughout the human body and spinal cord. Currently, the tools for interfacing peripheral nerves are outdated, bulky and carry a high risk of nerve injury, according to the University of Cambridge. “Nerves are small and highly delicate, so anytime you put something large, like an electrode, in contact with them, it represents a danger to the nerves,” ...

• Source: drugdu
• 89
• May 9, 2024

The BCR::ABL1 fusion gene plays a key role in the pathogenesis of several blood cancers, particularly chronic myeloid leukemia (CML). This gene results from a chromosomal translocation that causes constitutive activation of the ABL1 tyrosine kinase domain, triggering unchecked cell growth. Testing for the BCR::ABL1 fusion gene is crucial in managing leukemia, as it helps identify patients with Philadelphia chromosome-positive leukemia who might benefit from targeted therapies such as tyrosine kinase inhibitors (TKIs). While Sanger sequencing has been the standard method for this analysis, it has its limitations in sensitivity. Next-generation sequencing (NGS) offers a more comprehensive approach to detecting, quantifying, and analyzing the genetic alterations associated with leukemia, including minor BCR::ABL1 transcripts and mutations. NGS thus provides detailed insights into disease progression, treatment response, and potential resistance mutations, enabling personalized treatment plans. BioVendor MDx (Brno, Czech Republic) has introduced the first commercial NGS assay specifically for the BCR::ABL1 fusion ...

• Source: drugdu
• 73
• May 8, 2024

FDA Approves Azurity Pharmaceuticals’ Myhibbin as an Oral Suspension for Organ Transplant Recipients

Don Tracy, Associate Editor Myhibbin is reportedly the first FDA-approved liquid formulation of mycophenolate, a key immunosuppressant used to prevent organ rejection in transplant recipients. Axurity Pharmaceuticals announced that the FDA has officially approved Myhibbin as a ready-to-use mycophenolate mofetil oral suspension for organ transplant patients. Myhibbin is the first FDA-approved liquid formulation of mycophenolate, which is used to avoid organ rejection in transplant recipients, according to Azurity. The company stated that this approval provides a significant new option for both pediatric patients above the age of three months and adults who have undergone kidney, heart, or liver transplants.1 “We are very pleased that adult and pediatric organ transplant recipients will soon have access to the only FDA-approved ready-to-use oral liquid formulation of mycophenolate,” said Richard Blackburn, CEO, Azurity Pharmaceuticals, in a press release. “Patients are our priority, and our purpose is to bring them new formulations that help them ...

• Source: drugdu
• 77
• May 8, 2024

Nova’s Xromi approved in UK and EU for vaso-occlusive complications of SCD

Nova Laboratories’ Xromi (hydroxycarbamide) has received approval from the Medicines and Healthcare products Regulatory Agency and the European Commission to extend the indication for vaso-occlusive complications of sickle cell disease (SCD) to infants from nine months of age. SCD is a group of inherited red blood cell disorders that are caused by a genetic mutation that affects the production of haemoglobin, the protein in red blood cells that carries oxygen. The regulatory decisions were based on data from the pharmacokinetic, efficacy and safety study, HUPK, which showed that Xromi is expected to work in the same way in children from nine months of age as it does in older children. Furthermore, additional data from the phase 3 BABY HUG study suggests that the benefits and safety of Xromi in children as young as nine months of age are similar to those in older children. Dr James White, deputy managing director, ...

• Source: drugdu
• 83
• May 8, 2024

ClearPoint Neuro wins FDA clearance for neuro laser therapy accessory

BY SEAN WHOOLEY The ClearPoint Prism neuro laser therapy system. [Image courtesy of ClearPoint Neuro] ClearPoint Neuro (Nasdaq:CLPT) announced today that the FDA granted 510(k) clearance for its Prism bone anchor accessory. It marks the second new product introduction in 2024 for the company, with its stereotactic guidance system garnering clearance in January. The Prism system features the only non-cooled laser applicator on the market, the company said. Its next-generation laser applicator technology eliminates the need for external cooling. This simplifies setup, reduces power and ablation time and lessens imaging artifact for more efficient workflows. ClearPoint Neuro designed its Prism bone anchor accessory for use with commercially available stereotactic systems. These systems work for intracranial and neurosurgical procedures. Such procedures require accurate positioning of compatible small surgical instruments or accessories in the cranium, brain or nervous systems. The anchor provides short-term fixation and positioning of those instruments or accessories under image guidance. Chris Osswald, director, ...

• Source: https://www.massdevice.com/author/sean-whooley/
• 98
• May 4, 2024

FDA Approves Aquestive Therapeutics’ Libervant for the Treatment of Seizure Clusters in Children Aged 2 to 5 Years

Don Tracy, Associate Editor Approval of Libervant marks the first orally administered rescue medication for this age group to be cleared by the FDA for epilepsy. Image Credit: Adobe Stock Images/Justlight Aquestive Therapeutics announced that the FDA has officially approved Libervant (diazepam) Buccal Film to treat seizure clusters in children aged two to five years. According to the company, the film works by being applied inside the cheek, where it naturally dissolves and provides a steady dose of diazepam. Currently, most prescriptions are being prepared, with Medicaid expected to be added at a later date. Libervant is the first and only FDA-approved orally administered rescue medication for seizures in this age group.1 “We are thrilled to have received FDA approval for Libervant in patients between the ages of two and five,” said Daniel Barber, CEO, Aquestive, in a press release. “Patients have been waiting years for Libervant, the first and ...

• Source: https://www.pharmexec.com/authors/don-tracy-associate-editor
• 99
• May 4, 2024

FDA approves X4 Pharmaceuticals’ WHIM syndrome drug

The US Food and Drug Administration (FDA) has approved X4 Pharmaceuticals‘ XOLREMDI (mavorixafor) capsules for patients aged 12 and above with warts, hypogammaglobulinemia, infections and myelokathexis (WHIM) syndrome.   This marks a significant advancement in the treatment of WHIM syndrome, a rare combined primary immunodeficiency and chronic neutropenic disorder.   XOLREMDI is a selective CXC chemokine receptor 4 (CXCR4) antagonist. It is the first drug specifically indicated to treat patients with WHIM syndrome.   X4 Pharmaceuticals president and CEO Paula Ragan stated: “The approval of XOLREMDI is a transformational milestone both for X4 and, more importantly, for the WHIM syndrome community.   “We are incredibly grateful to the people living with WHIM syndrome, their families, and the investigators who took part in our clinical programme, to US regulators for their continued focus on rare-disease treatment development, and to our dedicated employees for making this targeted breakthrough therapy a reality.”   ...

• Source: https://www.pharmaceutical-technology.com/news/fda-x4-syndrome-drug/?cf-view
• 106
• May 3, 2024

Ono Pharmaceutical acquires cancer-focused biopharma Deciphera for $2.4bn

Deciphera Pharmaceuticals is set to be acquired in a $2.4bn buyout, as the biopharma announced that it has commenced a definitive merger agreement with Ono Pharmaceutical. Under the terms of the offered transaction, Ono will make a cash purchase of all outstanding shares of Deciphera’s common stock at $25.60 per share and subsequently merge Deciphera with a wholly-owned subsidiary of Ono upon the deal’s completion. The deal has been unanimously approved by both companies’ boards of directors and is expected to close in Q3 2024. Following the announcement of acquisition, Deciphera’s stock has jumped 71.9%. Waltham, Massachusetts-based Deciphera brings to the table an extensive kinase inhibitor pipeline, kinase drug discovery expertise, and a strong commercial and sales platform in the US and European markets that is meant to advance Ono’s capabilities and presence in the oncology space. Upon the successful completion of the acquisition, Ono will gain access to Deciphera’s ...

• Source: drugdu
• 107
• May 3, 2024

FDA Approves Utility Therapeutics’ Pivya to Treat Uncomplicated Urinary Tract Infections in Adult Females

Don Tracy, Associate Editor Pivya has shown efficacy against bacteria strains such as Escherichia coli, Proteus mirabilis, and Staphylococcus saprophyticus. The FDA announced that it has approved Pivya (pivmecillinam), for the treatment of uncomplicated urinary tract infections (UTIs) in adult females. According to the agency, the approval supports a communal health issue, with close to half of all women experiencing at least one UTI in their lifetime. Before reaching approval, Pivya, which is now available, was given Priority Review and Qualified Infectious Disease Product designations. As part of its support against UTIs, Pivya was designed to fight specific bacteria strains such as Escherichia coli, Proteus mirabilis, and Staphylococcus saprophyticus. The FDA based their approval on positive results from three different clinical trials comparing various Pivya dosing regimens to placebo, a different oral antibacterial drug, and ibuprofen. In all three trials, Pivya demonstrated a higher level of effectiveness in treating UTIs ...

• Source: drugdu
• 101
• April 28, 2024

Investors anticipate “bounce-back” in European private investment

The Swiss biotech ecosystem suffers from a “weak public market”, said Francesco de Rubertis, a cofounder at Medicxi Ventures. Despite this, De Rubertis and other experts at the recently concluded Swiss Biotech Day conference, anticipated an improvement in the investment market in the months to come. The conference, held in Basel 22–23 April, gathered experts from around the world to discuss trends in the pharmaceutical sector. At the “Decoding Swiss Biotech – the Investors perspective” session, panellists highlighted changes in the investment market and shared predictions for 2024 and onwards. Nicholas Riddle, the managing director of healthcare investment banking at JP Morgan, forecasts an uptick in the number of pharmaceutical initial public offerings in Europe this year. Riddle added, “It has been a pretty terrible time for private companies in the last several years. However, we are starting to see a bit more engagement from both European funds, who have ...

• Source: drugdu
• 74
• April 26, 2024