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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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.

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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.

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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.

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FDA Approves Ogsiveo for Adults with Desmoid Tumors

SpringWorks Therapeutics, Inc., announced today that the U.S. Food and Drug Administration has approved Ogsiveo™ (nirogacestat), an oral gamma secretase inhibitor, for the treatment of adult patients with progressing desmoid tumors who require systemic treatment. The FDA previously granted breakthrough therapy, fast track, and orphan drug designations to nirogacestat for the treatment of desmoid tumors.

Desmoid tumors are locally aggressive and invasive soft-tissue tumors that can lead to substantial morbidity. In addition, when vital structures are impacted, desmoid tumors can be life threatening. Although they do not metastasize, desmoid tumors are often refractory to existing off-label systemic therapies and associated with recurrence rates of up to 77% following surgical resection. Desmoid tumor experts and treatment guidelines now recommend systemic therapies as first-line intervention instead of surgery for most tumor locations requiring treatment.

Ogsiveo (nirogacestat) is not approved for the treatment of any other indication in the United States, or for any indication in any other jurisdiction by any other health authority.

SpringWorks is also evaluating nirogacestat as a potential treatment for patients with ovarian granulosa cell tumors and for patients with multiple myeloma as part of several B-cell maturation agent (BCMA) combination therapy regimens in collaboration with leaders in industry and academia.

SpringWorks expects to file a Marketing Authorization Application for Ogsiveo in desmoid tumors with the European Medicines Agency in the first half of 2024.

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Botanical-Be is voluntarily recalling all lots of Kuka Flex Forte Capsules, Artri King, Capsules, and Reumo Flex Capsules to the consumer level. FDA analysis has found the Kuka Flex Forte Capsules, Artri King Capsules, and Reumo Flex Capsules, to be tainted with Diclofenac. Diclofenac is an approved non-steroidal anti-inflammatory drug (NSAID), however, the presence of Diclofenac in Kuka Flex Forte, Artri King, and Reumo Flex renders them unapproved drugs for which safety and efficacy have not been established and, therefore, subject to recall.

Risk Statement: Consumption of undeclared diclofenac could result in serious adverse events that include cardiovascular, gastrointestinal, renal, and anaphylaxis in patients taking concomitant NSAIDs and/or anticoagulants, such as Warfarin, in those who have allergies to diclofenac, or those with underlying cardiovascular, gastrointestinal, renal, and hepatic illnesses. To date, Botanical-Be has not received any reports of adverse events related to this recall.

These tainted products are marketed as a dietary supplement for relief of pain and inflammation associated with arthritis and are packaged as followed:

• Artri King distributed in the bottles with 100 capsules.
• Kuka Flex distributed in the bottles with 30 capsules.
• Reumo Flex distributed in the boxes with 30 capsules.

The affected product lots include the following lot numbers and expirations: Artri King lot 35421, with an expiration date of December 19, 2025; Kuka Flex Forte; all lots with an expiration date of December 12, 2024, and UPC code 0736640810265; Reumo Flex; all lots with an expiration date of October 20, 2024.

These products were distributed nationwide via the internet. Botanical-Be is notifying its customers via email and is arranging for the return of all recalled products.

Consumers in possession of these products should cease usage immediately and return them to the place of purchase.

For any queries related to this recall, consumers can contact Botanical-Be by phone at (915) 412-6237 or by e-mail at botanical.be@gmail.com on Monday to Friday from 8:00 am to 5:pm, Mountain standard time. Consumers should contact their physician or healthcare provider if they have experienced any problems that may be related to taking or using this drug product.

(BeneCard does not carry the noted manufactured drugs)

FDA Approves Ryzneuta® for Chemotherapy-Induced Neutropenia

Evive Biotech (Evive), and Acrotech Biopharma (Acrotech), a subsidiary of Aurobindo Pharma USA Inc., have announced that the U.S. Food and Drug Administration has approved Ryzneuta®. Ryzneuta (Efbemalenograstim alfa), is indicated to decrease the incidence of infection, as manifested by febrile neutropenia, in adult patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a clinically significant incidence of febrile neutropenia. Neutropenia is a common side effect of chemotherapy and is characterized by persistently low levels of neutrophils (a type of white blood cell with infection-fighting functions) due to the use of chemotherapy and other types of anti-cancer drugs, which increases the risk of adverse reactions such as infection and fever in cancer patients during chemotherapy.

Ryzneuta is a novel long-acting Granulocyte colony-stimulating factor (G-CSF), which can stimulate the proliferation, differentiation, and release of neutrophil precursors. It helps to enhance the immune function of cancer patients and to prevent the side effects of neutropenia caused by chemotherapy.

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