The FDA’s authorization of ZILBRYSQ1 is substantiated by safety and effectiveness findings obtained from the RAISE study (NCT04115293), which was featured in The Lancet Neurology in May 2023. The RAISE study was a Phase 3, multicenter trial that was randomized, double-blind, and placebo-controlled. Its primary goal was to evaluate how well ZILBRYSQ worked and its safety and tolerance in adult patients who had generalized myasthenia gravis and were positive for anti-acetylcholine receptor (AChR) antibodies.
In this study, patients were randomly divided in a 1:1 ratio, and for 12 weeks, they either received daily subcutaneous injections of 0.3 mg/kg ZILBRYSQ or a placebo. The research revealed that ZILBRYSQ provided rapid, consistent, and statistically significant advantages in various outcomes reported by both patients and clinicians at the 12-week mark in a diverse population of adult patients with mild-to-severe gMG who tested positive for anti-AChR antibodies. The most frequent adverse reactions (experienced by ≥10% of gMG patients) included injection site reactions, upper respiratory tract infections, and diarrhea.
At week 12, treatment with ZILBRYSQ demonstrated a statistically significant improvement from baseline compared to placebo for MG-ADL total score and QMG total score. Other secondary endpoints included the proportion of patients with improvements of at least 3 and 5 points in the MG-ADL total score and QMG total score, respectively, at Week 12 without rescue therapy.
“This is an important development for the community because, with more FDA-approved treatments for generalized myasthenia gravis, physicians have additional tools to treat this disease in individualized ways that are the right fit for each individual patient,” said Samantha Masterson, President and Chief Executive Officer of the Myasthenia Gravis Foundation of America. “We are so grateful to UCB for being part of the myasthenia gravis community and their continued commitment to finding solutions for people living with this chronic, autoimmune, neuromuscular disease.”
Generalized Myasthenia Gravis is a rare, chronic, heterogeneous, unpredictable autoimmune disease characterized by dysfunction and damage at the neuromuscular junction (NMJ). As per DelveInsight, the total number of diagnosed Generalized Myasthenia Gravis prevalent cases associated in the 7MM countries was approximately 116,100 in 2021, which is expected to increase in the coming years. However, pharmaceutical companies including UCB, among others, are actively investing in cutting-edge research and development activities. The launch of the emerging therapies is anticipated to overcome existing market challenges and provide more effective solutions for patients, offering hope for improved quality of life and long-term management of this condition.
FDA Approves PENBRAYA, the First and Only Vaccine for the Prevention of the Five Most Common Serogroups Causing Meningococcal Disease in Adolescents
Pfizer Inc. announced that the FDA has approved PENBRAYA (meningococcal groups A, B, C, W, and Y vaccine), the first and only pentavalent vaccine that provides coverage against the most common serogroups causing meningococcal disease in adolescents and young adults aged 10 to 25. PENBRAYA combines the components of two meningococcal vaccines, Trumenba (meningococcal group B vaccine) and Nimenrix (meningococcal groups A, C, W-135, and Y conjugate vaccine), to help protect against the five most common meningococcal serogroups that cause the majority of invasive meningococcal disease (IMD) globally.
“One of our goals as a vaccine pioneer is to deliver vaccines that evolve the paradigm and help simplify the standard of care in the United States,” said Annaliesa Anderson, Ph.D., Senior Vice President and Head, Vaccine Research and Development, Pfizer. “Today is a significant step forward in the prevention of meningococcal disease in the United States.” By giving the widest meningococcal coverage in the fewest injections, PENBRAYA has the potential to protect more adolescents and young adults from this deadly and unpredictable disease in a single vaccine.”
Meningococcal disease is a rare but dangerous illness that can kill within 24 hours and leave survivors with life-altering, long-term disabilities. PENBRAYA decreases the total number of doses required to properly vaccinate persons against the five most frequent serogroups, simplifying the standard of care and perhaps increasing the number of adolescents and young adults vaccinated. Combining vaccines into fewer doses, according to the Centers for Disease Control and Prevention (CDC), may result in more adolescents and young adults receiving their recommended vaccines on time, resulting in fewer delays in protection against dangerous diseases.
The FDA’s decision is based on positive results from Phase II and Phase III trials, which included a randomized, active-controlled, and observer-blinded Phase III trial comparing the safety, tolerability, and immunogenicity of the pentavalent vaccine candidate to currently licensed meningococcal vaccines in the United States, with the goal of determining immunologic noninferiority. The Phase III trial (NCT04440163) included almost 2,400 patients from the United States and Europe.
BIMZELX Approved by the FDA for the Treatment of Adults with Moderate to Severe Plaque Psoriasis
UCB has received FDA approval for BIMZELX (bimekizumab-bkzx), making it available for the treatment of moderate to severe plaque psoriasis in adult patients who are eligible for systemic therapy or phototherapy. Bimekizumab stands out as the first and solitary psoriasis treatment with approval, specifically designed to target two crucial cytokines responsible for inflammatory processes: interleukin 17A (IL-17A) and interleukin 17F (IL-17F). The endorsement of bimekizumab is substantiated by data stemming from three Phase III clinical trials, conducted at multiple centers and employing randomized, placebo, and/or active comparator control groups (BE READY, BE VIVID, and BE SURE). These trials encompassed 1,480 adults grappling with moderate to severe plaque psoriasis.
Today’s FDA approval of BIMZELX marks a significant milestone in our ongoing commitment to enhancing the standard of care for plaque psoriasis and redefining treatment expectations. In our Phase III trials, we observed remarkable results, with 85-91% of patients achieving clear or nearly clear skin by week 16, and 59-68% achieving complete clearance,” stated Emmanuel Caeymaex, Executive Vice President of Immunology Solutions and Head of U.S. at UCB. With BIMZELX now approved for psoriasis, we will promptly pursue additional indications in the U.S.
Psoriasis afflicts over 7.5 million American adults, and its repercussions extend far beyond skin deep. As per DelveInsight analysis, in 2022, the total diagnosed prevalent cases of psoriasis in the US were ~8 million, projected to increase during the forecast period (2023–2032). As per DelveInsight analysis, the total age-specific cases of psoriasis in pediatrics and adults in the US were 73K and 7.8 million cases in 2022.
The FDA advises psoriasis patients to use bimekizumab at a dosage of 320 mg. This is typically administered as two separate subcutaneous injections, with each injection containing 160 mg. These injections should be given at Weeks 0, 4, 8, 12, and 16. After the initial 16 weeks, the dosage can be reduced to an injection every 8 weeks. However, for patients weighing 120 kg or more, a dosage of 320 mg every 4 weeks after week 16 may be considered. Bimekizumab can be administered by a healthcare professional or by the patient, provided the patient has received appropriate training. The medication is available in both autoinjector and pre-filled syringe forms. Bimekizumab is expected to be accessible in the U.S. in approximately one month.
FDA Approves BioMarin’s VOXZOGO for Children Under 5 Years with Achondroplasia
BioMarin Pharmaceutical Inc. announced that the US Food and Drug Administration (FDA) has accepted the supplemental New Drug Application (sNDA) for VOXZOGO (vosoritide) to improve linear growth in pediatric patients with achondroplasia with open epiphyses (growth plates). This indication has been authorized under accelerated approval due to an increase in annualized growth velocity. Previously, VOXZOGO was recommended for children aged 5 and up. This extended indication now encompasses children of all ages with open growth plates.
“We are pleased that VOXZOGO is now available for children of all ages with achondroplasia,” stated Hank Fuchs, M.D., BioMarin’s president of Worldwide Research and Development. “We are grateful for the achondroplasia community’s, physicians’, and children’s and families’ contributions to the advancement of this clinical program.” We also hope to learn more about the potential function of VOXZOGO in other inherited low-stature diseases, such as hypochondroplasia.”
“VOXZOGO is the only FDA-approved treatment for achondroplasia in children.” Until now, it has only been allowed in the United States for children aged 5 and older with open growth plates,” stated William Wilcox, M.D., Emory University professor of human genetics. “I am delighted that VOXZOGO is now approved for younger children where we hope to have potentially greater impact by starting treatment earlier and, as a result, a much longer treatment window.”
BioMarin conducted a randomized, double-blind, placebo-controlled Phase II clinical trial in children aged 5 and younger to assess the safety and efficacy of VOXZOGO (Study 111-206). Based on the findings of this trial, as well as evidence from an adequate and well-controlled Phase III study in pediatric patients aged 5 years and older (Study 111-301), the safety and efficacy of VOXZOGO in pediatric patients of all ages for the improvement of linear growth in children with achondroplasia with open epiphyses have been established. VOXZOGO’s overall safety profile in children under the age of five was similar to that seen in older children.
VOXZOGO is now licensed in Europe for children with achondroplasia who are 2 years old or older and have open growth plates. In September, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending marketing authorization to expand the indication for VOXZOGO for injection to treat children aged 4 months and older with achondroplasia whose epiphyses are not closed. The European Commission is likely to make a final clearance decision later this year that will be consistent with the CHMP proposal.
VOXZOGO is also licensed in Japan for children with achondroplasia with open growth plates from birth. Furthermore, it is permitted in Brazil for infants aged 6 months and older with open growth plates, as well as in Australia for children aged 2 years and older with achondroplasia and open growth plates.
Lisata Therapeutics Announces European Medicines Agency Has Granted LSTA1 Orphan Drug Designation for the Treatment of Pancreatic Cancer
Lisata Therapeutics, Inc. (Nasdaq: LSTA) disclosed on October 17, 2023, that the European Medicines Agency (“EMA”) Committee for Orphan Medicinal Products has awarded orphan drug status to LSTA1, the company’s primary product candidate designed for managing pancreatic cancer. Presently, LSTA1 is under investigation in numerous ongoing and scheduled clinical trials taking place worldwide, focusing on different solid tumor categories, including pancreatic cancer, in conjunction with a range of anti-cancer treatment approaches.
“Pancreatic cancer has one of the highest mortality rates of all cancers and affects hundreds of thousands of patients worldwide each year. Although progress has been made in understanding and treating pancreatic cancer, there remains significant unmet medical need,” stated Kristen K. Buck, M.D., Executive Vice President of R&D and Chief Medical Officer of Lisata. “To date, LSTA1 has demonstrated favorable safety, tolerability, and activity to enhance delivery of standard-of-care chemotherapy for patients with metastatic pancreatic cancer. Obtaining orphan drug designation from the EMA reinforces our belief that LSTA1 offers major improvement in treating patients with this terrible disease. We are excited by the promise of LSTA1 for the treatment of pancreatic cancer and other solid tumors and are committed to advancing our development programs with the goal of providing a benefit to patients.”
LSTA1 is an experimental medication designed to trigger a novel uptake pathway, which enhances the ability of concurrently administered or tethered anti-cancer drugs to better infiltrate solid tumors. LSTA1 activates this active transport system in a tumor-specific manner, resulting in a more efficient penetration and accumulation of co-administered anti-cancer drugs within the tumor. Furthermore, LSTA1 has the potential to modify the tumor microenvironment, rendering tumors more receptive to immunotherapies.
Lisata and its research partners have amassed substantial preclinical data demonstrating improved delivery of various established and emerging anti-cancer treatments, including chemotherapy, immunotherapies, and RNA-based therapies. Additionally, LSTA1 has displayed favorable safety, tolerance, and efficacy in clinical trials aimed at enhancing the delivery of standard-of-care chemotherapy for pancreatic cancer. Lisata is actively investigating the potential of LSTA1 to enhance the efficacy of a range of therapeutic approaches for the treatment of various solid tumors.
As per the latest assessment by DelveInsight, In 2021, the total pancreatic cancer incident cases in the 7MM countries were 175,700+, and these cases are anticipated to increase in the upcoming years. Among the European countries, Germany had the highest incident cases of pancreatic cancer in 2021, i.e., 21,000+ cases, followed by France which had 14,000+ incident cases in 2021. On the other hand, Spain had the lowest incident cases of pancreatic cancer, i.e. 8,000+ cases in 2021. Similarly, Japan had 43,000+ incident cases of pancreatic cancer in 2021. Pharmaceutical companies such as Lisata Therapeutics, among, others are pioneering innovative therapies and personalized medicine approaches to tackle the challenges in the pancreatic cancer market, offering new hope for patients.
FDA Grants Fast Track Designation to ANPD001, Autologous Investigational Cell Therapy for the Treatment of Parkinson’s Disease
October 19, 2023, Aspen Neuroscience revealed that the U.S. Food and Drug Administration (FDA) has awarded Fast Track Designation to ANPD001 for the enhancement of motor function in the treatment of Parkinson’s disease (PD). ANPD001 is an investigational personalized (autologous) cell therapy designed to address Parkinson’s Disease by replacing lost dopamine neurons.
ANPD001 is an experimental cellular therapy under investigation for Parkinson’s disease, aiming to replace neurons with the patient’s own cells. Aspen’s tailored manufacturing process consists of three stages, beginning with a small sample of the patient’s skin cells, followed by reprogramming into iPSCs, and subsequent differentiation into DANPCs. These DANPCs are surgically transplanted into the patient to replace cells lost or damaged due to the disease. At each manufacturing phase, the quality of the patient’s cells is evaluated using Aspen’s unique artificial intelligence-driven genomics assessments.
“We are pleased that FDA has granted Fast Track Designation, underscoring the potential of an autologous dopamine replacement therapy such as ANDP001 to serve as a meaningful treatment option. People with Parkinson’s disease have a significant unmet medical need and have been waiting for many years for more advanced treatment options,” said Damien McDevitt, PhD, Aspen president and CEO.
“Achieving Fast Track Designation is an important milestone that furthers our ability to collaborate with FDA and facilitate the development of ANPD001,” said Ana Sousa, MSJ, Aspen Senior Vice President, Regulatory Affairs & Quality. “Our goal is to bring this treatment to patients as safely and expeditiously as possible.”
Aspen intends to commence a Phase 1/2a clinical trial involving Parkinson’s Disease patients with moderate to severe symptoms, marking the first patient-centric trial of ANPD001. This milestone comes after the successful 2022 Trial-Ready Screening Cohort Study, which facilitated the screening, enrollment, and production of cells for prospective candidates in the clinical trial. Notably, this initiative represents the initial multicenter Phase 1/2a trial of an autologous iPSC-derived therapy in the United States.
