Monthly Archives: December 2023

FDA Approves Wainua (eplontersen) for the Treatment of Adults with Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis

Ionis Pharmaceuticals, Inc. has announced that the U.S. Food and Drug Administration (FDA) has approved Ionis and AstraZeneca’s Wainua™ (eplontersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN. Wainua is the only approved medicine for the treatment of ATTRv-PN that can be self-administered via an auto-injector.

U.S. FDA approval based on Phase 3 NEURO-TTRansform results showing Wainua demonstrated consistent and sustained benefit halting neuropathy disease progression and improving neuropathy impairment and quality of life.

ATTRv-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade. Wainua is a ligand-conjugated antisense oligonucleotide (LICA) medicine designed to reduce the production of TTR protein at its source. Wainua will be available in the U.S. in January 2024. Additional regulatory reviews for Wainua are underway in the rest of world.

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FDA Approves Filsuvez (birch triterpenes) Topical Gel for the Treatment of Epidermolysis Bullosa

Chiesi Global Rare Diseases, a business unit of the Chiesi Group has announced that the U.S. Food and Drug Administration (FDA) approved Filsuvez® (birch triterpenes) topical gel for the treatment of partial thickness wounds in patients 6 months and older with Junctional Epidermolysis Bullosa (JEB) and Dystrophic Epidermolysis Bullosa (DEB).

Filsuvez is the first approved treatment for wounds associated with JEB, a rare, moderate-to-severe form of epidermolysis bullosa (EB) with blisters beginning in infancy. Filsuvez joined the Chiesi portfolio as part of the agreement reached during the acquisition of Amryt Pharma in January of this year.

EB is a debilitating inherited skin disease that causes a person’s skin to be so fragile it can be injured just from touch. This rare, chronic, and distressing disorder affects infants, children and adults and is intensely painful; recurrent blistering and chronic wounds can result in intolerable pain with limited mobility. Living with EB entails daily challenges to navigate, including slow healing wounds at risk of infection and painful dressing changes.

Filsuvez is administered at home, allowing for integration into existing treatment routines. Filsuvez is applied topically to the wound at each dressing change.

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FDA Approves Alyglo (Immune Globulin Intravenous, Human-stwk) 10% Liquid for Adults with Primary Humoral Immunodeficiency (PI)

GC Biopharma Corp has announced that the U.S. Food and Drug Administration (FDA) has approved Alyglo (immune globulin intravenous, human-stwk) 10% Liquid, formerly referred to as “GC5107,” for the treatment of adult patients aged 17 years and older with primary humoral immunodeficiency (PI).

Primary Humoral Immunodeficiency (PI) refers to a group of disorders where the body’s ability to produce antibodies is impaired, affecting the immune system’s ability to fight infections. In PI, there’s a deficiency or absence of B cells or plasma cells, which are key in producing antibodies, particularly immunoglobulin G, leading to increased susceptibility to infection, especially bacterial infections affecting the respiratory and gastrointestinal tracts. People with PI might experience frequent infections, some of which can be severe or chronic. Treatment often involves immunoglobulin replacement therapy to boost the immune system.

Vaccinations and antibiotics may also be used to prevent and treat infections. The exact type and severity of PI can vary widely among individuals.

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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.

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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.

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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.

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.

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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.

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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.

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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.

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