iDOSE TR®

FDA Approves iDose TR (travoprost intracameral implant) for the Treatment of Glaucoma

Glaukos Corporation, announced that the U.S. Food and Drug Administration (FDA) approved its New Drug Application (NDA) for a single administration per eye of iDose® TR (travoprost intracameral implant) 75 mcg, a prostaglandin analog indicated for the reduction of intraocular pressure (IOP) in patients with ocular hypertension (OHT) or open-angle glaucoma (OAG).

iDose TR is a first-of-its-kind, long-duration, intracameral procedural pharmaceutical therapy designed to continuously deliver 24/7 therapeutic levels of a proprietary formulation of travoprost inside the eye for extended periods of time. iDose TR is intended to improve the standard of care by addressing the ubiquitous patient non-compliance issues and chronic side effects associated with topical glaucoma medications.

Glaukos intends to commence initial commercial launch activities for iDose TR in the latter part of the first
quarter of 2024.

Read more…

IWILFIN™

FDA Approves Iwilfin (eflornithine) as Maintenance Therapy for High-Risk
Neuroblastoma

USWM, LLC (US WorldMeds) has announced that the U.S. Food and Drug Administration (FDA) has approved Iwilfin ™ (eflornithine) 192 mg tablets, a groundbreaking oral maintenance therapy for high-risk neuroblastoma. Iwilfin is indicated to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma who have demonstrated at least a partial response to prior multiagent, multimodality therapy, including anti-GD2 immunotherapy.

According to the American Cancer Society, 700-800 cases of neuroblastoma are diagnosed in the U.S. each year, with 90% of diagnoses coming before age 5. Over 50% of these cases are classified as high-risk. High-risk neuroblastoma is a challenging disease, with a high mortality rate driven primarily by the risk of relapse after achieving remission. Approximately half of children with high-risk neuroblastoma do not survive beyond five years from diagnosis. Although existing treatments are effective in helping patients achieve remission, patients lack options to sustain it. Avoiding relapse is crucial to improving survival rates.

Iwilfin is taken orally, with or without food, twice daily for two years. Iwilfin is generally well-tolerated, with side effects typically manageable through dose modifications. The most common side effects are hearing loss, otitis media, pyrexia, pneumonia, and diarrhea.

Read more…

Pounds Return Once Zepbound Users Quit the Weight-Loss Drug

Study By Ernie Mundell HealthDay Reporter

Folks who take the blockbuster weight-loss med tirzepatide (Zepbound) may regain much of the weight they lost soon after discontinuing it, new research shows.

A trial funded by Eli Lilly, the injected drug’s maker, found that “in patients with obesity or overweight, withdrawing tirzepatide led to substantial regain of weight.”

On the other hand, continuing with tirzepatide kept the weight off, over the full two years of the trial.

Of course, sticking with the drug could mean big bills for users.

Medicare and Medicaid do not cover the drug, and if your private insurance doesn’t cover Zepbound, it can cost over $1,000 per month. Lilly says certain commercial card savings programs it offers can reduce the monthly cost to about $550, or even lower.

Zepbound was approved for weight loss by the U.S. Food and Drug Administration on Nov. 8, and Lilly announced its availability to consumers on Dec. 6.

To trigger weight loss, tirzepatide mimics two hormones, GLP-1 and GIP, which stimulate the release of insulin in the body. It quells appetite and slows the rate at which food moves through the stomach, helping patients feel full.

It’s the first drug in its class to compete with another weight-loss blockbuster, Wegovy.

Wegovy, made by Novo Nordisk, uses a similar active ingredient, semaglutide, which only focuses on GLP-1.

That difference appears to translate to greater weight loss with Zepbound than Wegovy, a recent study found. Zepbound has been found to prompt up to a 20.9% drop in weight at higher doses, while Wegovy patients typically see a 15% reduction in weight.

But if patients quit Zepbound, does the weight come back?

Yes, according to the new study, which was led by Dr. Louis Aronne, a professor of clinical medicine at Cornell University. He also directs the Comprehensive Weight Control Center at Weill-Cornell Medical Center, in New York City.

The research involved 670 overweight or obese people without diabetes who averaged 48 years of age. About 70% were women.

All participants first took tirzepatide for nine months. Then some were randomized to either another year taking the drug, or to a year taking a “dummy” placebo. Neither the participants nor researchers knew whether a participant was taking the active drug or the placebo.

By the end of trial, nearly all (89.9%) of the those who got tirzepatide for all 22 months maintained at least 80% of the weight loss they’d already experienced by the nine-month mark, Aronne’s group reported. However, that was only true for about 17% of participants who’d been switched over to the placebo shot at the nine-month mark. By the study’s end, folks who’d stuck with tirzepatide lost a quarter (25.3%) of their baseline weight, compared to about 10% weight loss for those who’d stopped taking the drug at nine months.

The study was published Dec. 11 in the Journal of the American Medical Association.

Side effects could occur — people were more likely to experience “mild to moderate gastrointestinal events” while on tirzepatide long-term versus placebo, the team noted.

Aronne’s team also noted that the weight regain observed after stopping tirzepatide isn’t unique to the drug. At least four other trials focused on weight-loss drugs have found the same rebound effect after quitting, including one trial focused on semaglutide (Wegovy).

Together, the data suggests that “obesity is a chronic metabolic condition similar to type 2 diabetes and hypertension requiring long-term therapy in most patients,” Aronne’s team said.

LYFGENIA™

bluebird bio Announces FDA Approval of LYFGENIA™ for Sickle Cell Disease

bluebird bio, Inc. has announced the U.S. Food and Drug Administration has approved LYFGENIA™ (lovotibeglogene autotemcel), also known as lovo-cel, as a one-time intravenous suspension gene therapy that has the potential to resolve vaso-occlusive events (VOC’s) and is custom-designed to treat the underlying cause of sickle cell disease (SCD).

Lyfgenia uses a replication-incompetent, self inactivating lentiviral vector (LVV) to add functional copies of the beta-globin gene to the patient’s own blood stem cells, which results in the production of anti-sickling hemoglobin that may decrease or stop vaso-occlusive events. It is made specifically for each patient. Patients are admitted to a treatment center during this process.

SCD is a genetic, inherited, lifelong disease caused by an alteration in one of the genes in the red blood cell, the beta-globin gene, that causes the normal disc shaped red cells to take the shape of a sickle, causing anemia and VOCs, like a pain crisis.

VOCs are when sickled red blood cells block blood flow, depriving tissues of oxygen. Lyfgenia uses a common, well-studied viral vector to deliver genetic modifications to a patient’s blood stem cells, so no donor is needed.

Read more…

CASGEVY™

FDA Approves Casgevy™ – First-ever approval of a CRISPR-based gene-editing therapy in the U.S. for the Treatment of Sickle Cell Disease

Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced that the U.S. Food and Drug Administration has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 genome edited cell therapy, for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). This approval means that for the first time, approximately 16,000 patients with SCD may be eligible for a durable one-time therapy that offers the potential of a functional cure for their disease by eliminating severe VOCs and hospitalizations caused by severe VOCs.

Its approval in the U.S. and U.K. caps a decade of remarkable scientific progress translating CRISPR from academic breakthrough to new medicine.

CASGEVY is a genome-edited cellular therapy consisting of autologous CD34+ hematopoietic stem cells (HSCs) edited by CRISPR/Cas9 technology at the erythroid specific enhancer region of the BCL11A gene. It is intended for one-time administration for people aged 12 years and older with (SCD). The hematopoietic stem cell transplant procedure uses the patient’s own CD34+ cells which are modified to reduce BCL11A expression in erythroid lineage cells, leading to increased fetal hemoglobin (HbF) production. HbF increases overall hemoglobin levels and has been shown to improve the production and function of red blood cells. This can eliminate VOCs in people with SCD.

Sickle cell disease (SCD) is a debilitating, progressive and life-shortening disease. SCD patients report health-related quality of life scores well below the general population, and the lifetime health care costs in the U.S. of managing SCD for patients with recurrent VOCs is estimated between $4 and $6 million. SCD is an inherited blood disorder that affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. Due to misshapen or “sickled” red blood cells, SCD causes severe pain, organ damage and shortened life span. SCD requires a lifetime of treatment and results in a reduced life expectancy. In the U.S., the median age of death for patients living with SCD is approximately 45 years.

A cure for SCD today is a stem cell transplant from a matched donor, but this option is only available to a small fraction of patients living with SCD because of the lack of available donors.

Read more…

AVZIVI®

FDA Approves Avzivi®, a Biosimilar to Avastin®

Bio-Thera Solutions, Ltd, has announced that the U.S. Food and Drug Administration has approved Avzivi® (bevacizumab-tnjn), a biosimilar referencing Avastin®. Avzivi® is Bio-Thera’s second
USFDA approved product and is the second biosimilar researched, developed, and manufactured by a Chinese pharmaceutical company to receive FDA approval in the United States.

The FDA approval of Avzivi was based on a comprehensive analytical, non-clinical and clinical data package submitted by Bio-Thera to the FDA. Extensive analytical characterization between BAT1706 and US and EU Avastin were conducted on structural, physicochemical, and biological properties to support biosimilarity of BAT1706.

A randomized double-blind, single-dose, three-arm, parallel phase I study compared the pharmacokinetics, safety, and immunogenicity of BAT1706 with both the US and EU Avastin in healthy volunteers. A randomized, double-blind, three-arm parallel phase III study compared BAT1706 with Avastin for efficacy, safety, and immunogenicity in subjects with advanced non-squamous non-small cell lung cancer.

The totality of the evidence demonstrated that BAT1706 has similar efficacy, safety, immunogenicity, and quality as the reference product bevacizumab.

Read more…

FABHALTA®

FDA Approves Fabhalta for the Treatment of Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Novartis today announced that the U.S. Food and Drug Administration has approved Fabhalta® (iptacopan) capsules as the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH).

Fabhalta is a Factor B inhibitor that acts proximally in the alternative complement pathway of the immune system, providing comprehensive control of red blood cell (RBC) destruction within and outside the blood vessels (intra- and extravascular hemolysis [IVH and EVH]). In clinical trials, treatment with Fabhalta increased hemoglobin levels (≥ 2 g/dL from baseline in the absence of RBC transfusions) in a majority of patients and in APPLY-PNH nearly all patients treated with Fabhalta did not receive blood transfusions.

PNH has a significant unmet need not fully addressed by anti-C5 therapies (eculizumab or ravulizumab): despite treatment with anti-C5s, a large proportion of people with PNH may remain anemic, and dependent on blood transfusions.

It is estimated that approximately 10-20 people per million worldwide live with PNH. Although PNH can develop at any age, it is often diagnosed in people between 30-40 years old.

Read more…

JAYPIRCA

FDA Grants Accelerated Approval to Eli Lilly’s Jaypirca for Treatment of Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma

Eli Lilly and Company has received accelerated approval for Jaypirca (pirtobrutinib) by the U.S. Food and Drug Administration for adults with chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who have received at least two prior lines of therapy, including a BTK inhibitor and a BCL-2 inhibitor.

This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.

This application was granted priority review and orphan drug designation. FDA expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

Read more…

FDA Investigating Serious Risk of T-cell Malignancy Following BCMA-Directed or CD19-Directed Autologous Chimeric Antigen Receptor (CAR) T cell Immunotherapies.

The U.S. Food and Drug Administration has received reports of T-cell malignancies, including chimeric antigen receptor CAR-positive lymphoma, in patients who received treatment with BCMA- or CD19-directed autologous CAR T cell immunotherapies. Reports were received from clinical trials and/or post marketing adverse event (AE) data sources.

FDA has determined that the risk of T-cell malignancies is applicable to all currently approved BCMA-directed and CD19-directed genetically modified autologous CAR T cell immunotherapies. T-cell malignancies have occurred in patients treated with several products in the class. Currently approved products in this class (listed alphabetically by trade name) include the following:

• Abecma (idecabtagene vicleucel)
• Breyanzi (lisocabtagene maraleucel)
• Carvykti (ciltacabtagene autoleucel)
• Kymriah (tisagenlecleucel)
• Tecartus (brexucabtagene autoleucel)
• Yescarta (axicabtagene ciloleucel)

Although the overall benefits of these products continue to outweigh their potential risks for their approved uses, the FDA is investigating the identified risk of T cell malignancy with serious outcomes, including hospitalization and death, and evaluating the need for regulatory action.

Patients and clinical trial participants receiving treatment with these products should be monitored life-long for new malignancies. In the event that a new malignancy occurs following treatment with these products, contact the manufacturer to report the event and obtain instructions on collection of patient samples for testing for the presence of the Chimeric Antigen Receptor (CAR) transgene.

To report suspected adverse events including T cell malignancies, contact the FDA at 1-800-FDA 1088 or www.fda.gov/medwatch.

Read more…

FDA Warns of Rare but Serious Drug Reaction to the Antiseizure Medicines Levetiracetam and Clobazam

The U.S. Food and Drug Administration is warning that the antiseizure medicines levetiracetam (Keppra, Keppra XR, Elepsia XR, Spritam) and clobazam (Onfi, Sympazan), can cause a rare but serious reaction that can be life-threatening if not diagnosed and treated quickly. This reaction is called Drug Reaction with Eosinophilia and Systemic Symptoms (DRESS). It may start as a rash but can quickly progress, resulting in injury to internal organs, the need for hospitalization, and even death. As a result, we are requiring warnings about this risk to be added to the prescribing information and patient Medication Guides for these medicines.

This hypersensitivity reaction to these medicines is serious but rare. DRESS can include fever, rash, swollen lymph nodes, or injury to organs including the liver, kidneys, lungs, heart, or pancreas.

What is FDA doing?

The FDA is requiring manufacturers of these medicines to add new warnings about DRESS to the prescribing information and the Medication Guide for patients and caregivers. For levetiracetam (Keppra, Keppra XR, Elepsia XR, and Spritam), this involves adding a new warning in the Warnings and Precautions section of the prescribing information, which describes the most serious and significant potential safety issues. Currently the symptoms associated with this condition are described less prominently.

For clobazam (Onfi and Sympazan), the FDA is requiring a new warning specifically about DRESS to be added to the prescribing information. Symptoms related to this risk are already described more generally in other sections of the clobazam prescribing information.

FDA reviewed worldwide cases of Drug Reaction with Eosinophilia and Systemic Symptoms (DRESS) associated with levetiracetam and clobazam in children and adults reported to the FDA Adverse Event Reporting System (FAERS) database and can be found in the medical literature.

Read more…