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Alnylam has announced FDA approval of supplemental new drug application for Oxlumo (lumasiran) in advanced primary hyperoxaluria type 1.

The leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) approved a label expansion for Oxlumo^® (lumasiran), an RNAi therapeutic administered via subcutaneous injection, now indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate (UOx) and plasma oxalate (POx) levels in pediatric and adult patients.

Oxlumo is administered once a month for three months, then monthly for patients weighing less than 10 kg, and quarterly for patients weighing more than 10 kg.

The most common adverse reaction (reported in ≥20% of patients) is injection site reactions.

Theratechnologies’ Trogarzo (ibalizumab-uiyk), an antiviral medicine that prevents human immunodeficiency virus (HIV) from multiplying in your body, has been approved by the FDA for 30-Second Intravenous (IV) Push, simplifying HIV treatment for heavily treatment-experienced population.

Trogarzo is used to treat the small percentage of patients who have multidrug-resistant human immunodeficiency virus type 1 HIV (MDR HIV-1 Infection) and who have failed other HIV therapies. In a clinical trial, most patients (33 of the 40 patients, or 83%) experienced a significant decrease in their HIV-RNA levels one week after Trogarzo was added to their ineffective antiretroviral regimens.

Trogarzo is administered intravenously (IV) once every 14 days by a trained medical professional

Catalyst Pharmaceuticals CPRX announced that the FDA approved its supplemental new drug application (sNDA) for Firdapse (amifampridine) tablets in 10 mg dosage to include pediatric patients (six years and older) for treating Lambert-Eaton myasthenic syndrome (“LEMS”).

The company submitted an sNDA to the FDA for the use of Firdapse in treating pediatric LEMS patients in the first quarter of 2022. LEMS is an ultra-rare disease autoimmune disorder characterized by muscle weakness of the limbs. Post the sNDA approval by the FDA, Firdapse is now a treatment option in the United States for all LEMS patients beyond six years of age.

Firdapse (amifampridine phosphate) is a nonspecific, voltage-dependent, potassium (K+) channel blocker for the treatment of Lambert Eaton myasthenic syndrome (LEMS) in adults and pediatric patients six years of age and older.

The FDA approved Relyvrio, a drug developed by Amylyx Pharmaceuticals, after extensive discussions about the safety and efficacy of the drug. Used to treat adults with amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig’s disease, the drug was initially rejected by an FDA advisory panel in March of this year. Earlier this month, the panel reconvened and reanalyzed, voting in favor of Relyvrio after being presented with additional data. The final ruling was delivered on September 29, 2022.

“FDA approval of Relyvrio is an exciting milestone for the ALS community and is a major step toward achieving our mission to one day end the suffering caused by neurodegenerative diseases,” said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx.

The U.S. Food and Drug Administration (FDA) granted accelerated approval to LYTGOBI (futibatinib) for the treatment of adult patients with previously treated, bile duct cancer that has spread or cannot be removed by surgery. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

The approved recommended dosage of LYTGOBI is 20 mg orally (five 4 mg tablets) once daily until disease progression or unacceptable toxicity occurs. It is used in patients who have already received a previous treatment, and whose tumor has a certain type of abnormal FGFR2 gene. The tablets can be taken with or without food and swallowed whole, at approximately the same time each day.

Additional information regarding dosage and administration as well as warnings and precautions about ocular toxicity, hyperphosphatemia and soft tissue mineralization, and embryo-fetal toxicity can be found in the full prescribing information.

The U.S. Food and Drug Administration approved Regeneron/Sanofi’s Dupixent (dupilumab) for the treatment of adult patients with prurigo nodularis (PN). Dupixent is the first medication approved for PN, a rare inflammatory skin condition that can cause severe itching and have a significant negative impact on sleep and quality of life.

• Dupixent is an anti–interleukin (IL)-4 and anti–IL-13 biologic that was first approved to treat moderate to severe atopic dermatitis in 2017 and has since gained additional indications to treat several other type 2 inflammatory-driven conditions, such as asthma.

• In the Phase 3 PRIME and PRIME2 clinical trials, patients receiving Dupixent experienced superior itch reduction and achieved higher rates of clear or almost clear skin at 24 weeks compared with patients receiving placebo.

• IPD Analytics recommends prior authorization (PA) for Dupixent in the treatment of PN with a step through at least one medium- to super high-potency topical corticosteroid (TCS). Additionally, the PA criteria should match the labeled age and indication for PN. IPD also recommends including certain inclusion criteria from the PRIME and PRIME2 trials in the PA criteria.

• Utilization management of Dupixent should be high on payers’ radar, as utilization of the drug is expected to continue to grow for at least the next 5 years and biosimilar competition likely will not reach the market until 2031.

Celltrion USA announced that the U.S. Food and Drug Administration (FDA) has approved Vegzelma (bevacizumab-adcd), an anti-cancer monoclonal antibody treatment and a biosimilar to Avastin (bevacizumab), for the treatment of six types of cancer: metastatic colorectal cancer; recurrent or metastatic non-squamous non-small cell lung cancer (nsNSCLC); recurrent glioblastoma; metastatic renal cell carcinoma; persistent, recurrent, or metastatic cervical cancer; and epithelial ovarian, fallopian tube, or primary peritoneal cancer.

Vegzelma is a recombinant humanized monoclonal antibody which binds to vascular endothelial growth factor (VEGF), the key driver of vasculogenesis and angiogenesis, and thereby inhibits the binding of VEGF to its receptors Flt-1 (VEGFR-1), and kinase insert domain receptor (KDR) (VEGFR-2), on the surface of endothelial cells.

The FDA approval of Vegzelma was based on the totality of evidence, including the pivotal phase III trial in patients with metastatic or recurrent nsNSCLC. Results showed that as a first-line treatment, Vegzelma is highly similar to the reference product in terms of efficacy, safety and pharmacokinetics.

FDA approves Xenoview (xenon Xe 129 hyperpolarized) for use with MRI for the Evaluation of Lung Ventilation

Polarean Imaging plc, the medical imaging technology company, announces that the U.S. Food and Drug Administration (“FDA”) has granted approval for its drug device combination product, Xenoview. Xenoview, prepared from the Xenon Xe 129 Gas Blend, is a hyperpolarized contrast agent indicated for use with magnetic resonance imaging (“MRI”) for the evaluation of lung ventilation in adults and pediatric patients aged 12 years and older. Xenoview has not been evaluated for use with lung perfusion imaging.

Xenoview expands the opportunity for pulmonary medicine to utilize the first and only inhaled MRI hyperpolarized contrast agent for novel visualization of lung ventilation without exposing patients to any ionizing radiation and its associated risks. The dose of Xenoview, created through the Polarean HPX hyperpolarization system, is administered in a single 10-15 second breath hold MRI procedure.

More than 30 million Americans suffer from a chronic lung disease, and there is a significant unmet need for non-invasive diagnostic technology. Xenoview can provide pulmonologists, surgeons, and other respiratory specialists with regional maps of ventilation in their patients’ lungs to assist them in managing their disease.

Xenoview is not approved for use in pediatric patients less than 12 years of age.

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Iyuzeh Ophthalmic Solution Approved for Open-Angle Glaucoma and Ocular Hypertension

Thea Pharma, Inc. (“Thea”), the U.S. subsidiary of Europe’s leading independent pharmaceutical company, Laboratoires Théa, has announced the U.S. Food and Drug Administration (FDA) approval for the New Drug Application (NDA) of Iyuzeh™ (latanoprost ophthalmic solution) 0.005% for the reduction of elevated intraocular pressure (IOP) in patients with open-angle glaucoma (OAG) or ocular hypertension (OHT).

Iyuzeh is the first and only clinically proven formulation of latanoprost available in the United States that is preservative-free. Iyuzeh is formulated without any of the preservatives commonly used in topical ocular preparations, including benzalkonium chloride (BAK). Iyuzeh has demonstrated consistent IOPlowering effects and proven tolerability across multiple trials in the U.S. and Europe.

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