FDA Approves First Gene Therapy for Severe Hemophilia A

BioMarin Pharmaceutical Inc., a global biotechnology company dedicated to transforming lives through genetic discovery, announced that the US Food and Drug Administration (FDA) has approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity of 1 IU/dL) without antibodies to adeno-associated virus. The one-time, single-dose infusion is the first gene therapy licensed in the United States for severe hemophilia A. ROCTAVIAN was approved by the European Medicines Agency in August 2022.

Adults with severe hemophilia A face a lifelong burden, with frequent infusions and a high risk of health complications, including uncontrolled bleeding and irreversible joint damage,” said Dr. Steven Pipe, an investigator in the Phase 3 study and a professor of pediatrics and pathology at the University of Michigan.  “The approval of ROCTAVIAN, the first gene therapy for severe haemophilia A, has the potential to transform the way we treat adults, based on years of bleed control following a single, one-time infusion.

The FDA approval is based on data from the global Phase III GENEr8-1 study, the biggest Phase III trial of any gene therapy in hemophilia. Of the 134 patients who got ROCTAVIAN in the research, 112 had baseline annualized bleeding rate (ABR) data gathered prospectively throughout at least six months of FVIII prophylaxis prior to starting ROCTAVIAN. The remaining 22 patients had their baseline ABR recorded retrospectively. All patients were followed for at least three years.

BioMarin will continue to evaluate the treatment’s long-term effects with an extension study that will follow all clinical trial participants for up to 15 years, as well as post-approval studies that will follow individuals dosed in a real-world scenario for 15 years or more. 

FDA Approves First Cellular Therapy to Treat Patients with Type 1 Diabetes

Lantidra, the first allogeneic pancreatic islet cellular therapy manufactured from deceased donor pancreatic cells, was approved by the US Food and Drug Administration for the treatment of type 1 diabetes. Lantidra is licensed for the treatment of adults with type 1 diabetes who, despite intensive diabetes care and education, are unable to achieve target glycated hemoglobin.

Severe hypoglycemia is a dangerous condition that can lead to injuries resulting from loss of consciousness or seizures,” stated Peter Marks, M.D., Ph.D., director of the FDA’s Centre for Biologics Evaluation and Research. “Today’s approval, the first-ever cell therapy to treat patients with type 1 diabetes, provides individuals living with type 1 diabetes and recurrent severe hypoglycemia with an additional treatment option to help them achieve target blood glucose levels.

Type 1 diabetes is a chronic autoimmune disease that requires lifelong care, including daily insulin infusions or continuous infusion via a pump. People with type 1 diabetes also monitor their blood glucose levels multiple times a day to help them control their diabetes.

The major mechanism of action of Lantidra is thought to be insulin production by infused allogeneic islet beta cells. In certain type 1 diabetes patients, these injected cells can create enough insulin that the patient no longer needs insulin to control their blood sugar levels. Lantidra is injected into the hepatic portal vein in a single dose. Depending on the patient’s response to the initial dose, an extra infusion of Lantidra may be administered. 

FDA Declines to Approve Amneal’s Parkinson’s Drug Over Safety Concerns

Amneal Pharmaceuticals announced that it has received a letter from the U.S. Food and Drug Administration (FDA) known as a Complete Response Letter (CRL) concerning the New Drug Application (NDA) for IPX203, a treatment for Parkinson’s disease

The letter stated that while there was sufficient scientific evidence supporting the safety of one of the ingredients, levodopa (LD), through pharmacokinetic studies, the evidence for the other ingredient, carbidopa (CD), was not deemed sufficient. The FDA has requested additional information regarding carbidopa. However, there were no concerns raised regarding the effectiveness or manufacturing of IPX203. Amneal Pharmaceuticals intends to collaborate closely with the FDA to address their comments and plans to meet with the agency to establish the best course of action going forward.

“We are fully dedicated to advancing IPX203 for the treatment of Parkinson’s disease, as it has been specifically developed to provide longer-lasting therapeutic benefits with a reduced number of doses compared to existing formulations,” stated Chirag and Chintu Patel, the Co-Chief Executive Officers at Amneal. They further added, “We intend to closely collaborate with the FDA to address their feedback, and we maintain our confidence in bringing this innovative treatment to Parkinson’s patients at the earliest opportunity.”

The submission of the New Drug Application (NDA) is supported by the findings from the Phase 3 RISE-PD clinical trial. The trial demonstrated that IPX203’s extended-release formula significantly increases the duration of “Good On” time and reduces the occurrence of “Off” time in comparison to immediate-release CD/LD, even with less frequent dosing.

It is important to note that this development does not affect Amneal’s financial guidance for 2023, as the revenues from IPX203 were not included in the initial guidance.

NLS Pharmaceutics Receives FDA Approval for Phase III Clinical Trials of Quilience

NLS Pharmaceutics, a clinical-stage biopharmaceutical company based in Switzerland that focuses on developing innovative therapies for patients with rare and complex central nervous system disorders, announced that the U.S. Food and Drug Administration (FDA) has granted authorization to proceed with the Phase 3 program for Quilience® (Mazindol ER). This program, called the AMAZE Program, will consist of two double-blind Phase 3 trials, each involving 50 participants. The trials will compare Mazindol ER to a placebo in adult patients with narcolepsy, and they are scheduled to begin in the upcoming summer at various locations across the United States.

Both Phase 3 trials, named NLS-1031 and NLS-1032, will primarily evaluate the weekly occurrences of cataplexy episodes over an 8-week treatment period. Patients who successfully complete these studies will have the opportunity to participate in a 12-month open-label extension (OLE) study, referred to as Study NLS-1033. To be eligible for enrollment in the program, patients must be at least 18 years old and have a confirmed diagnosis of narcolepsy with cataplexy.

In a previous report, NLS shared the results of the Phase 2 study conducted on narcolepsy patients, in which Quilience (Mazindol ER) successfully achieved all primary and secondary endpoints. Patients who received treatment with Mazindol ER in the randomized Phase 2 trial displayed ongoing improvement when transitioning to the open-label extension (OLE) study. Moreover, patients who initially received a placebo in the randomized Phase 2 trial but later switched to Mazindol ER in the OLE study achieved comparable outcomes to those treated with Mazindol ER in the Phase 2 trial. Data from the Phase 2 studies will be presented at the SLEEP 2023 conference, hosted by the American Academy of Sleep Medicine (AASM) and the Sleep Research Society (SRS), held from June 3 to 7, 2023, in Indianapolis.

The European Commission Grants Orphan Drug Designation to Temferon™ for Treatment of Glioma

On June 29, 2023, Genenta Science (NASDAQ: GNTA) announced that the European Commission has granted Orphan Drug Designation (ODD) to Temferon™ for the treatment of Glioma. Glioblastoma Multiforme (GBM) is the first clinical indication of Temferon.

The European Medicine Agency’s Committee reviewed Genenta’s ODD application for Temferon and agreed on the potential significant benefit that Temferon could contribute to patients suffering from Glioblastoma Multiforme if approved. The ODD designation supports and facilitates the development of our cell therapy-based technology platform for solid tumors. The EMA ODD designation follows the orphan drug designation granted by the US Food and Drug Administration to Temferon for the treatment of Glioblastoma Multiforme in March 2023. The preliminary interim results of Genenta’s ongoing phase 1/2a trials in newly diagnosed patients with unmethylated MGMT gene promoter reviewed by EMA included an Overall Survival of two years, which is longer than the median Overall Survival described in published reports.

Pierluigi Paracchi, Chief Executive Officer at Genenta

The most developed version of Genenta’s product, Temferon, uses the patient’s own stem progenitor cells that have been engineered using Genenta’s platform to express interferon alpha (IFNa) in solid tumors. IFNa is a well-known immunomodulatory protein that has been employed in clinical practice for years to treat a number of malignancies, but its present application is restricted due to systemic toxicity. The platform developed by Genenta is intended to prevent systemic toxicity and deliver therapeutic activity just to the solid tumor. Temferon has been found to disrupt tumor-induced tolerance in pre-clinical trials, enabling the immune system to recognize the tumor and generate a long-lasting immunological response.

Glioblastoma Multiforme is the most prevalent malignant primary brain tumor and the most aggressive diffuse glioma; roughly 60% of GBMs were found to have an unmethylated MGMT promoter status. As per DelveInsight’s assessment, the Glioblastoma diagnosed incident population in the 7MM was 32,000+ in 2021 and the number of cases is projected to increase in the coming years. Among the EU-5 countries, Germany accounted for the highest number of Glioblastoma cases, followed by France, whereas Spain accounted for the lowest number of cases in 2021. To overcome the unmet needs in the therapeutics domain, globally, several companies are actively working in the Glioblastoma Multiforme market

GSK Receives US FDA Fast Track Designation for Investigational Vaccine Against Gonorrhea

On 7 June 2023, GSK plc (LSE/NYSE: GSK) announced the US Food and Drug Administration (FDA) has granted a Fast Track designation for its Neisseria Gonorrheae investigational vaccine (NgG). The vaccine candidate is currently in an ongoing Phase II trial and aims to demonstrate proof of concept by assessing the efficacy of the NgG vaccine in healthy adults, 18 to 50 years of age, considered at risk of Gonorrhea. 

We welcome the FDA’s decision to grant Fast Track designation for our new vaccine candidate against Neisseria Gonorrheae infection. With a high and growing incidence, Gonorrhea is a major concern for sexual and reproductive health around the globe. This designation recognizes the potential for a vaccine that could help protect millions of people across the world against the serious health consequences of infection with a bacterium that is considered a ‘high priority’ pathogen by the World Health Organization.

Phil Dormitzer, Global Head of Vaccines R&D, GSK

The GSK experiment is a Phase I/II first-time in-human research (FTiH) assessing the safety and efficacy of the vaccine candidate in people ages 18 to 50, regardless of prior Gonorrhea history. The study’s first phase, which involved healthy individuals in a dose-escalation safety lead-in for FTiH, is already over. The study’s continuing Phase II aims to show proof of concept by evaluating the effectiveness of the NgG vaccination in healthy persons thought to be at risk for Gonorrhea. A total of 750 participants from 8 nations—the United States, the United Kingdom, France, Germany, Spain, Brazil, the Philippines, and South Africa—will participate in the study, which began in November 2022.

Gonorrhea is a sexually transmitted infection (STI) caused by a bacterium called Neisseria Gonorrheae (Ng). An estimated 82 million new cases of Gonorrhea are reported worldwide each year, making it the second most common bacterial STI. 710,151 cases of Gonorrhea were reported to the CDC in 2021, a rise of 118% in reported rates from 2009 to 2021. Over the past 80 years, antimicrobial resistance (AMR) to Gonorrhea has risen, rendering several kinds of antibiotics used to treat the condition useless. By assisting in the prevention of bacterial, viral, and other diseases, vaccines can be extremely effective in the fight against AMR. There are currently no Gonorrhea vaccines that have been approved anywhere in the world. It is recognized as an urgent unmet medical need due to its growing global incidence and reduced efficacy of available treatments as drug-resistant strains are increasing. However, several major pharma and biotech giants including GSK and several others are actively working in the Gonorrhea therapeutics market. As per DelveInsight, the Gonorrhea market is anticipated to evolve immensely in the coming years owing to the improvement in the diagnosis methodologies, raising awareness of the diseases, incremental healthcare spending across the world, and increasing prevalence of the disease.