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AstraZeneca’s rare disease unit receives NICE recommendation for Wolman disease therapy in infants

AstraZeneca’s rare disease unit has received a recommendation from the National Institute for Health and Care Excellence (NICE) for the use of its enzyme replacement therapy in infants with Wolman disease. Alexion’s Kanuma (sebelipase alfa), which has been specifically recommended for use in patients who are aged two years or younger when administration begins, will now become the first treatment available on the NHS for the rapidly-progressive rare genetic disease. Occurring in around one in 350,000 births, Wolman disease causes a build-up of fat in cells in the liver, heart, blood vessels and digestive system. Symptoms in infants include enlarged liver and spleen, poor weight gain, low muscle tone, jaundice, vomiting, diarrhoea, developmental delay and anaemia. Until now, standard care for the disease has been palliative and limited to managing symptoms, with patients normally not surviving past the age of one without treatment. Administered as weekly intravenous infusions which can ...
- Source: drugdu
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- November 30, 2023
FDA investigates ‘serious risk’ of secondary cancer following CAR-T treatment

In a blow to CAR-T therapies, the FDA is investigating a “serious risk” of patients developing new cancers after treatment with these highly efficacious oncology drugs. The FDA unveiled the probe Tuesday. The agency said it has received reports of T-cell malignancies, including CAR-positive lymphoma, among patients who received BCMA- or CD19-directed CAR-T cell immunotherapies. Some patients involved have had to be hospitalized or died, according to the agency. The cases stem from clinical trials and postmarketing adverse event surveillance, the FDA said. The FDA has determined that the potential risk is applicable to all currently approved CAR-T therapies, as T-cell malignancies have occurred after patients received several different products. The FDA is now weighing potential regulatory action—even as the potential risk of developing secondary cancer is already included as a class warning on the labels of the CAR-T therapies. Currently marketed CD19 CAR-Ts include Yescarta and Tecartus from Gilead ...
- Source: drugdu
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- November 30, 2023
Bayer’s stock drops to new 18-year low after asundexian trial termination

Bayer’s stock price has plummeted to its lowest since 2005 after a clinical trial investigating the anti-coagulant drug asundexian was stopped early due to lack of efficacy. The Phase III OCEANIC-AF trial (NCT05643573) aimed to determine asundexian’s use as a treatment for people living with atrial fibrillation in a bid to prevent strokes or systemic embolisms. The Independent Data Monitoring Committee (IDMC) found that asundexian was inferior in terms of efficacy when compared to the control arm of the trial. The OCEAN-AF trial was a part of the overall OCEAN Phase III programme. However, the committee recommended continuing another Phase III OCEANIC-STROKE trial (NCT05686070) examining the use of the drug in stroke patients. Following the announcement on 19 November 2023, the German pharmaceutical firm’s stock value dropped from €41.45 ($45.38) per share down to €30.77 ($33.68), as of 28 November. It is the lowest price in 18 years. The last ...
- Source: drugdu
- 42
- November 30, 2023
Acelyrin claims errors by CRO Fortrea led to late-stage trial downfall

Immunology biotech Acelyrin has determined that its CRO partner Fortrea is responsible for the late-stage failure of its immunotherapeutic drug candidate for moderate to severe hidradenitis suppurativa (HS). The company says a programming blunder by a vendor recruited by the CRO resulted in two erroneously treated patient arms going undetected through the testing processes. Los Angeles-based Acelyrin was aiming to make waves in a fiercely competitive psoriasis market with a breakout $540m IPO in May and a steady stream of clinical data and patient enrolment updates on its izokibep clinical program. But in September, when the Phase IIb/III study (NCT05355805) failed to meet its endpoint of achieving a 75% reduction in total abscesses and inflammatory nodules, the company’s stock more than halved overnight and has not yet recovered to the levels seen before 11 September. While a press release issued by Acelyrin outlining the error did not identify Fortrea as ...
- Source: drugdu
- 52
- November 30, 2023
FDA withdraws from Global Harmonization Working Party

Dive Brief The U.S. Food and Drug Administration decided to withdraw from the Global Harmonization Working Party (GHWP), one of multiple groups working to align international standards for medical devices. Jeff Shuren, director of the FDA’s Center for Devices and Radiological Health, raised concerns about “divergent harmonization efforts” in a Monday letter to Jinghe Xu, chair of the GHWP and deputy commissioner of China’s National Medical Products Administration. Shuren’s concerns included efforts that do not align with the FDA’s priorities or international best practices and the lack of collaboration with the International Medical Device Regulators Forum (IMDRF), another international standards group. Dive Insight The FDA joined the GHWP in December 2021 with the goal of serving as a bridge between the organization and the IMDRF. At the time, the FDA didn’t realize how divergent the two organizations had become, Shuren wrote. “I would like to see GHWP and IMDRF collaborating ...
- Source: drugdu
- 39
- November 29, 2023
Biodegradable patch holds promise of correcting congenital heart defects in infants

Using laboratory engineered tissue, scientists at the University of Colorado Anschutz Medical Campus have created a full thickness, biodegradable patch that holds the promise of correcting congenital heart defects in infants, limiting invasive surgeries and outlasting current patches. The findings were published this week in the journal Materials Today. Jeffrey Jacot, PhD, study’s senior author, associate professor of bioengineering, University of Colorado School of Medicine, said, “The ultimate goal is to make lab-grown heart tissue from a patient’s own cells that can be used to restructure the heart to correct for heart defects.” About 10,000 babies are born with a complex congenital heart defect every year in this country, requiring surgery in the first year of life. Some of these operations require the implantation of a full-thickness heart patch. But the current materials used in the patch are non-living and non-degradable. They don’t grow with the patient and often fail ...
- Source: drugdu
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- November 29, 2023
Henry Schein reports more disruption as cyberattackers take credit

BY JIM HAMMERAND Henry Schein (Nasdaq: HSIC)+ said today that it is working to bring its ecommerce platform back up after more problems related to its cyberattack. The medical device manufacturer and distributor said last week that its ecommerce platform and other applications were unavailable and that “the threat actor from the previously disclosed cyber incident has claimed responsibility.” Today, the company said its ecommerce platform is back online in the U.S. and was expected to be restored in Canada and Europe “shortly.” Henry Schein said it continued to take orders via alternate methods previously communicated to customers, and continued shipping products. Earlier this month, the company warned customers and suppliers that their sensitive information may have been exposed in the cyberattack. Henry Schein first disclosed the cyber security incident in October. A ransomware gang known as BlackCat/ALPHV later said they encrypted the company’s systems and stole 35 TB of ...
- Source: drugdu
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- November 29, 2023
Phase 3 Clinical Trial for Dupixent Reports Significant Reduced COPD Exacerbations

Don Tracy, Associate Editor Trial results expected to accelerate the potential of Dupixent to become the first FDA-approved treatment for chronic obstructive pulmonary disease. Regeneron Pharmaceuticals and Sanofi have announced that a Phase 3 trial evaluating Dupixent (dupilumab) in the treatment of chronic obstructive pulmonary disease (COPD) showed the drug reduced exacerbations by 34%, reportedly confirming positive results from its earlier Phase 3 BOREAS trial. According to a Regeneron press release, the NOTUS trial also confirmed that treatment led to rapid and significant improvements in lung function by 12 weeks and were sustained at 52 weeks. Back in May, the companies also announced the success of the aforementioned BOREAS trial for investigational use of Dupixent in adults currently on maximal standard-of-care inhaled therapy (triple therapy) with COPD and evidence of type 2 inflammation. Earlier this year, the drug was granted FDA Breakthrough Therapy designation as an add-on maintenance treatment in ...
- Source: drugdu
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- November 29, 2023
FDA grants second orphan drug designation to Priothera’s mocravimod

The US Food and Drug Administration (FDA) has granted an orphan drug designation to Priothera’s mocravimod for leukaemia patients undergoing a stem cell transplant. The drug is indicated to help improve the outcome of patients with haematologic malignancies following a haematopoietic stem cell transplantation, according to a 27 November press release. Following the designation, the Dublin, Ireland-headquartered biotech will now be in line for tax credits for US-based clinical trials and, if the therapy is approved, have seven years of market exclusivity in the designated indication. Haematopoietic stem cell transplantation, also known as blood and bone marrow transplantation, is used to treat a wide range of blood cancers. There are nearly 60,000 new cases of leukaemia in the US each year. This is the second orphan drug designation for Priothera’s mocravimod. The first, granted in March 2022, was for the treatment of acute myeloid leukaemia (AML) in patients undergoing hematopoietic ...
- Source: drugdu
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- November 29, 2023
Sanofi/Regeneron’s Dupixent shows promise in chronic obstructive pulmonary disease

Sanofi and Regeneron have shared positive results from a late-stage trial of Dupixent (dupilumab) in chronic obstructive pulmonary disease (COPD). The phase 3 NOTUS trial has been evaluating the investigational use of Dupixent compared to placebo in adults currently on maximal standard-of-care inhaled therapy with uncontrolled COPD and evidence of type 2 inflammation. The trial met its primary endpoint, with Dupixent shown to reduce moderate or severe acute COPD exacerbations by 34% compared to placebo. Results from the interim analysis also demonstrated that Dupixent improved lung function from baseline by 139ml at 12 weeks compared to 57ml for placebo, with this benefit sustained at week 52. COPD is a chronic respiratory disease that causes lung function to progressively decline. Approximately 300,000 people in the US live with uncontrolled cases of the condition and evidence of type 2 inflammation. The positive readout from NOTUS builds on the previous results from the ...
- Source: drugdu
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- November 29, 2023

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