FDA Approves GSK’s Arexvy, the First RSV Vaccine for Older Adults

GSK plc stated that the US Food and Drug Administration (FDA) has approved Arexvy (respiratory syncytial virus vaccine, adjuvanted) for the prevention of lower respiratory tract disease (LRTD) caused by a respiratory syncytial virus (RSV) in people aged 60 and up. This is the first RSV vaccine for older individuals to be licensed anywhere globally.

“Today marks a turning point in our effort to reduce the significant burden of RSV,” said Tony Wood, Chief Scientific Officer of GSK. Arexvy is the first RSV vaccine authorized for older adults, adding to GSK’s industry-leading vaccination portfolio, which protects millions of people each year from infectious diseases. Our priority now is to ensure that eligible older persons in the United States receive the vaccination as soon as possible, and to forward regulatory approval in other countries.”

RSV is a common and contagious virus that can cause serious respiratory disease. Each year, it causes roughly 177,000 hospitalizations and an estimated 14,000 fatalities in persons aged 65 and older in the United States. Most RSV hospitalizations are caused by older persons, especially those with underlying medical disorders such as diabetes and chronic heart and lung disease. As per DelveInsight analysis on the latest published “Respiratory Syncytial Virus Market Report”, in 2022, the total incident cases of RSV in the 7MM were ~8.4 million, and these cases are anticipated to increase by 2032.

“For decades, AMGA and the healthcare community at large have been active in finding ways to increase adult immunizations,” said John Kennedy, MD, President of the American Medical Group Association (AMGA). As a result, we are thrilled to announce that a respiratory syncytial virus vaccine is now available to doctors for patient treatment. Americans over 60, particularly those with underlying health issues such as COPD, asthma, or congestive heart failure, will have a vaccine to help guard against potentially serious RSV outcomes with this vaccine.”

The FDA approval in the United States is based on GSK’s groundbreaking positive pivotal AReSVi-006 (Adult Respiratory Syncytial Virus) phase III study data. The vaccine was well accepted and had an acceptable safety profile. Injection site pain, weariness, myalgia, headache, and arthralgia were the most often encountered requested adverse effects. These were generally mild to moderate in severity and temporary in nature. The Advisory Committee on Immunisation Practises (ACIP) will provide recommendations on the vaccine’s appropriate usage in the United States in June 2023. The RSV vaccine will be accessible for older persons before the RSV season in 2023/24, which normally begins before the winter months.

CHMP Adopts Positive Opinion Recommending Gilead’s Hepcludex® (Bulevirtide) for Hepatitis Delta Virus

Gilead Sciences, Inc. announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for Hepcludex® (bulevirtide) for the treatment of adults with chronic HDV and compensated liver disease, and has recommended granting full Marketing Authorisation (MA) that is no longer subject to specific obligations. Bulevirtide was granted conditional marketing authorization in July 2020 in order to offer persons living with HDV with immediate access to treatment. The CHMP recommendation for full marketing authorization of bulevirtide follows the submission of data from the Phase III MYR301 Week 48 study, which validates bulevirtide’s effectiveness and safety profile for the treatment of HDV.

HDV is the most severe form of viral hepatitis, affecting an estimated 5% of people with hepatitis B (HBV), with a global prevalence of more than 12 million people. Mortality rates for persons living with HDV with extensive disease progression, including cirrhosis, can reach 50% within five years, posing a significant disease burden and high healthcare-related expenses.

“People with HDV have had very limited treatment options, and if they do not receive treatment, they frequently progress to severe liver disease or liver cancer.” We had no approved options for treating these patients until bulevirtide, but now we have a treatment conditionally approved specifically for HDV with a positive recommendation for full approval,” said Heiner Wedemeyer, MD, Director, Clinic for Gastroenterology, Hepatology, and Endocrinology at Hannover Medical School. “It provides clinicians with additional assurance about the efficacy and safety of longer-term use.”

The European Commission (EC) will assess the CHMP suggestion, and if approved, bulevirtide will be completely authorized for the treatment of individuals with chronic HDV and compensated liver disease in the European Union. Gilead is collaborating with many regulatory agencies on bulevirtide marketing applications in other parts of the world. The Food and Drug Administration (FDA) granted bulevirtide Breakthrough Therapy and Orphan Drug designations in the United States. While bulevirtide is not currently licensed in the United States, Gilead is in ongoing discussions with the FDA to deliver bulevirtide to patients living with HDV in the United States as soon as feasible.

FDA Grants 501K Clearance to Bladder EpiCheck for Surveillance in NMIBC

Nucleix has been granted permission by the FDA to sell its product Bladder EpiCheck as a way to monitor tumor recurrence in patients with non-muscle invasive bladder cancer (NMIBC) who have previously been diagnosed. This method does not require invasive procedures and can be used in combination with cystoscopy. Bladder EpiCheck is a unique urine test that uses a qPCR platform to evaluate 15 methylation markers linked to bladder cancer, and it has a high degree of accuracy in identifying disease-specific changes. Although the test has already been made available for purchase in Europe, it is the first of its kind methylation-based urine test on a qPCR platform to gain FDA clearance.

Bladder cancer ranks sixth among the most common types of cancer in the United States. There are about 700,000 individuals in the country living with bladder cancer, with the majority being classified as NMIBC patients. Furthermore, more than 80,000 new cases of bladder cancer are diagnosed every year. Although NMIBC can be treated with surgical and therapeutic methods, the likelihood of recurrence is quite high, with up to 70% recurrence within five years. Therefore, frequent monitoring is necessary to detect high-grade recurrence early and provide prompt treatment before it becomes life-threatening. Currently, the standard of care involves invasive cystoscopies, which are performed up to four times per year for at least five years, and in some cases, for the rest of the patient’s life.

According to Aharona Shuali, M.D., who is the Vice President of Medical Affairs at Nucleix, monitoring NMIBC is a demanding and time-consuming process that can be highly invasive for patients. Therefore, a precise and reliable urine-based test such as Bladder EpiCheck could be an essential addition to the existing standard of care, allowing physicians to enhance their diagnostic capabilities.

Eli Frydman, Ph.D., who is the President of Nucleix, expressed satisfaction with the FDA’s decision to approve Bladder EpiCheck for commercial use in the United States. Dr. Frydman stated that the company is now able to offer healthcare professionals and their patients in the US access to a reliable, objective, and non-invasive test for bladder cancer that can be conducted at any licensed local or central lab, providing medical professionals with the flexibility to perform the test wherever it is required. This clearance reinforces the value of Nucleix’s EpiCheck technology and its ability to provide healthcare providers with non-invasive diagnostic tools that can improve patient care.

Nucleix is currently considering strategic collaborations and other activities to gain access to the market for the commercial release of Bladder EpiCheck in the United States.

ProMIS Neurosciences Announces FDA Clearance of Investigational New Drug (IND) Application for PMN310 in Alzheimer’s Disease

ProMIS Neurosciences, a biotech firm that specializes in developing antibody therapeutics that target harmful misfolded proteins found in neurodegenerative conditions like Alzheimer’s disease, amyotrophic lateral sclerosis, and multiple system atrophy, has announced that the U.S. Food and Drug Administration has granted clearance for its Investigational New Drug application for PMN310, a treatment for Alzheimer’s disease. PMN310 is a unique monoclonal antibody that has been developed to specifically target toxic oligomers of amyloid-beta (Aβ), which are thought to be a significant contributor to the progression of Alzheimer’s disease.

Gail Farfel, Ph.D., who is the Chief Executive Officer of ProMIS Neurosciences, stated that the company’s achievement of IND clearance for PMN310 is a significant accomplishment as they work towards providing patients with Alzheimer’s disease with next-generation treatments that can help slow cognitive decline. Dr. Farfel highlighted the preclinical data that showed PMN310’s stronger selective binding to harmful oligomers compared to other antibodies that target Aβ, which the company believes underscores the potential clinical profile of PMN310. She expressed excitement about the advancement of PMN310 into clinical development and the opportunity to share insights gained from this innovative work.

In studies conducted before clinical trials, PMN310 demonstrated strong ex vivo target engagement of harmful oligomers in brain samples from Alzheimer’s disease patients, with minimal or no diversion by Aβ monomers or plaque. In a recent presentation of in vitro data at the AD/PD 2023 conference, PMN310 was observed to be the least affected by monomer competition, compared to other Aβ-targeting antibodies, which resulted in a greater ability to target harmful oligomers. Moreover, PMN310 did not show binding to plaque, which could potentially reduce the risk of Aβ-related imaging abnormalities (ARIA) associated with plaque-binding antibodies. Based on these findings, the company believes that PMN310 has the potential for a different clinical profile compared to other antibody therapeutics being developed for Alzheimer’s disease.

Following the clearance of the IND application for PMN310, ProMIS Neurosciences intends to commence a Phase 1a clinical study aimed at assessing the safety, tolerability, and pharmacokinetics of various PMN310 dosages in healthy adult participants.

PharmaTher Submits Fast Track Application for Ketarx to US FDA

On May 02, 2023, PharmaTher Holdings Ltd. announced the submission of a Fast Track Application to the US Food and Drug Administration (“FDA”) for KETARX™ (ketamine) for the treatment of levodopa-induced dyskinesia in Parkinson’s disease (“LID-PD”). Ketamine is an FDA-approved N-methyl-D-aspartate receptor-modulating (NDMA) drug that is widely used as an anesthetic agent, either alone or in combination with other anesthetic agents. Additionally, Ketamine may also have additional benefits in the treatment of pain and depression, which are frequent comorbidities of Parkinson’s disease.

Following Its recently announced Type C meeting with the FDA for advancing KETARX™ towards Phase 3 clinical development, the Company is evaluating a Phase 3 clinical trial design to align with the FDA’s recommendations that would allow for a potential FDA approval. The safety and efficacy results from its previously announced presentation of a Phase I/II clinical study evaluating ketamine as a potential new treatment for LID-PD will be used to support the investigation of KETARX™ in a proposed Phase 3 clinical study.

Summary results of the Phase I/II clinical study:

  • Enrolled subjects with moderate to advanced Parkinson’s disease, with a target infusion rate being 0.30 mg/kg/hr. Data highlight that ketamine was safe, and well-tolerated, and demonstrated that 100% of subjects treated with ketamine had a reduction in dyskinesias as measured by UDysRS. 
  • UDysRS showed a 51% reduction from baseline during Infusion 2 (p=0.003), 49% at 3 weeks (p=0.006), and 41% at 3 months (p=0.011) post-ketamine.
  • The maximum tolerated infusion rate ranged from 0.20-0.30 mg/kg/hr, which was dependent on either discomfort due to dissociation or hypertension. There were no adverse events post-infusion.

Globally, about 10 million people worldwide are affected by Parkinson’s disease. In 2022, the total diagnosed prevalent cases of Parkinson’s disease were the highest in the US among the 7MM, with about 1,152,000+ cases, and lowest in Italy with nearly 152,000+ cases. As per DelveInsight, in 2022 the Parkinson’s disease market size was ~USD 3,226 million in 2022, which is anticipated to increase during the study period 2019–2032. The current understanding of Parkinson’s disease has improved in recent decades, leading to growing awareness, improved management, and better outcomes. The emerging pipeline therapies such as KETARX™, among others, hold the potential to immensely improve the Parkinson’s disease treatment outlook

Vedanta Biosciences Receives Fast Track Designation for VE303 and Presents Phase 2 Data at Digestive Disease Week

On May 08, 2023, Vedanta Biosciences announced that the U.S. Food and Drug Administration (FDA) had granted Fast Track designation to its defined bacterial consortium candidate, VE303, to prevent recurrent Clostridioides difficile infection (rCDI). It consists of eight strains that were rationally selected using Vedanta’s discovery engine. VE303 is produced from pure, clonal bacterial cell banks, which yield a standardized drug product in powdered form and bypass the need to rely on direct sourcing of donor fecal material of the inconsistent composition. 

Moreover, Vedanta also announced the details of a podium presentation of research informed by the VE303 Phase 2 CONSORTIUM study at Digestive Disease Week (DDW) 2023, held in Chicago, Illinois, May 6-9. The presented analyses review multiple aspects of the pharmacodynamic response to VE303 in patients at high risk for rCDI, including how safety and efficacy correlate with the dosing regimen, consortium strain colonization, metabolic changes, and restoration of a patient’s gut microbial community. 

Vedanta reported positive topline results in October 2021 from the Phase 2 CONSORTIUM trial, in which VE303 was associated with a 31.7% absolute risk reduction in the rate of recurrence when compared with placebo, representing a greater than 80% reduction in the odds of a CDI recurrence. 

“The FDA’s decision to grant Fast Track designation to VE303 underscores the continuing need for medical innovation for this condition. Over 150,000 people experience recurrent CDI annually in the U.S. alone, and this requires scalable, effective treatment solutions. Fecal microbiota transplants and other donor-derived treatments are often prescribed—they can be effective, but they can vary greatly in composition and potency, and they may carry a risk of transmitting infectious agents. Vedanta’s approach of growing the strains for our defined bacterial consortia from pure clonal cell banks is a highly scalable process that obviates the need for fecal donors and leads to a consistently manufactured candidate. We look forward to initiating our global Phase 3 pivotal study for VE303, RestoratiVE303, later in 2023.”

Jeffrey L. Silber, M.D., Chief Medical Officer of Vedanta Biosciences.

Vedanta Biosciences received a $5.4 million research grant from the Combating Antibiotic Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) in 2017. Additionally, the positive results of the Phase 2 study, first reported in October 2021, triggered a $23.8 million contract option from the Biomedical Advanced Research and Development Authority (BARDA) to support a Phase 3 clinical study of VE303. In 2017, VE303 was granted Orphan Drug Designation and Fast Track Designation in 2023 by the U.S. Food and Drug Administration (FDA) for the prevention of recurrent CDI.
In the past 10–15 years, C. difficile infection (CDI) has emerged as an increasingly influential infectious disease worldwide. It is considered as one of the main etiological agents of bacterial diarrhea associated with the use of antibiotics. It is an important nosocomial pathogen and the main cause of morbidity and mortality.

As per DelveInsight analysis, the C. difficile infection (CDI) incident cases in the 7MM were found to be approximately 668,000+ in 2021. The highest number of incident cases of CDI was in the US with approximately 468,000+ cases, followed by EU-5 with approximately 141,000+ cases, and Japan with approximately 58,000+ cases in 2021. The cases are expected to increase in the US and EU-5 by 2032. As per DelveInsight, in 2021, the C. difficile infection (CDI) market size was USD 413.5 Million in the 7MM, which is anticipated to grow in the coming years. The increase in market size is a direct consequence of the increasing incidence and robust pipeline of CDI in the 7MM.