Posts in category: News

FDA Approves Beqvez (fidanacogene elaparvovec-dzkt) One-Time Gene Therapy for Adults with Hemophilia B

Beqvez™, manufactured by Pfizer for the treatment of adults with moderate to severe hemophilia B, has been approved by the FDA for patients with the following:

o Currently use factor IX (FIX) prophylaxis therapy, or
o Have current or historical life-threatening hemorrhage, or
o Have repeated, serious spontaneous bleeding episodes, and
o Do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test.

• Beqvez is a one-time treatment that is designed to enable people living with hemophilia B to produce FIX themselves rather than the current standard of care, which requires regular intravenous infusions of FIX that are often administered multiple times a week or multiple times a month.
• Hemophilia B is a rare genetic bleeding disorder that prevents normal blood clotting because of a deficiency in FIX that causes those with the disease to bleed more frequently and longer than others.
• According to the World Federation of Hemophilia, more than 38,000 people worldwide are living with hemophilia B.

Source: Pfizer Inc.

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FDA Approves Pivya (pivmecillinam) for the Treatment of Uncomplicated Urinary Tract Infections

The FDA approved Pivya tablets for the treatment of female adults with uncomplicated urinary tract infections (UTIs) caused by susceptible isolates of Escherichia coli, Proteus mirabilis and Staphylococcus saprophyticus.

• Pivya’s efficacy in treating females 18 years of age or older with uncomplicated UTIs was assessed in three controlled clinical trials comparing different Pivya dosing regimens to placebo, ibuprofen and another oral antibacterial drug.
• Patients should not use Pivya if they have a known history of severe hypersensitivity to Pivya or other beta-lactam antibacterial drugs.
• The FDA granted the approval of Pivya to UTILITY Therapeutics Ltd.

Source: UTILITY Therapeutics Ltd.

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FDA Grants Accelerated Approval for Ojemda for Relapsed or Refractory BRAF-altered Pediatric Low-Grade Glioma

The FDA has provided accelerated approval to Day One Biopharmaceuticals, Inc. for Ojemda (tovorafenib), a type II RAF inhibitor.

• pLGG is the most common brain tumor diagnosed in children, with patients suffering profound tumorand treatment-associated morbidities that can impact their life trajectory. With this approval, Day One Biopharmaceuticals has received a rare pediatric disease priority review voucher from the FDA. Until now, there had been no medicines approved for patients with pLGG driven by BRAF fusions. Ojemda is indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (pLGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.
• BRAF is the most commonly altered gene in pLGG, with up to 75 percent of children having a BRAF alteration. Ojemda is the only systemic therapy for pLGG that offers once-weekly dosing, with or without food, as a tablet or oral suspension.

Source: Day One Biopharmaceuticals, Inc.

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FDA Approves Rezenopy Nasal Spray for the Emergency Treatment of Opioid Overdose

The FDA has approved Rezenopy (naloxone hydrochloride) nasal spray 10mg for the emergency treatment of known or suspected opioid overdose, as manifested by respiratory and/or central nervous system depression in adult and pediatric patients. Rezenopy is manufactured by Summit Biosciences Inc.

• Rezenopy is a high dose naloxone hydrochloride nasal spray formulation containing 10mg of naloxone per spray available on prescription. Rezenopy nasal spray is intended for immediate administration as emergency therapy in settings where opioids may be present.
• Naloxone hydrochloride is an opioid antagonist that works to reverse the effects of opioids during an overdose, including respiratory depression, sedation, and hypotension.
• Common adverse reactions reported with Rezenopy include upper abdominal pain, nasopharyngitis and dysgeusia.

Source: FDA

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FDA Approves Lumisight™ Optical Imaging Agent to Illuminate Residual Breast Cancer Post-Lumpectomy

The FDA approved Lumicell, Inc.’s New Drug Application (NDA) for its Lumisight™ (pegulicianine) optical imaging agent and its Premarket Approval (PMA) application for Lumicell™ Direct Visualization System (DVS), together referred to as LumiSystem™.

• Lumicell, Inc., is a privately held company focused on developing innovative fluorescence-guided imaging technologies for cancerous tissue detection during surgery.
• With 84% diagnostic accuracy, LumiSystem enables surgeons to scan the breast cavity postlumpectomy, in real-time, to detect and resect residual cancer that may have otherwise been missed, potentially sparing some patients from second surgeries.
• The safety of the system was established using data from more than 700 breast cancer patients across five clinical studies at top academic and regional community cancer centers across the U.S.

Source: Lumicell, Inc.

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FDA Approves Selarsdi, a Biosimilar to Stelara

The FDA has approved Selarsdi (ustekinumab-aekn) injection for subcutaneous use, as a biosimilar to Stelara, for the treatment of moderate to severe plaque psoriasis and for active psoriatic arthritis in adults and pediatric patients 6 years and older.

• Introduced through a strategic partnership between Teva Pharmaceutical Industries Ltd. and Alvotech, for the exclusive commercialization of five of Alvotech’s biosimilar product candidates, Teva will be responsible for the exclusive commercialization of Selarsdi in the United States.
• Sales of Stelara in the U.S. were nearly $7 billion in 2023. The availability of a Stelara biosimilar will create opportunities for cost savings across the healthcare system and introduce additional treatment options for patients.
• In the U.S., plaque psoriasis is the most common form of psoriasis while psoriatic arthritis accounts for approximately six percent of all cases of juvenile arthritis.

Source: Teva Pharmaceutical Industries Ltd.

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FDA Approves Anktiva Plus BCG for BCG-Unresponsive Non-Muscle Invasive Bladder Cancer

ImmunityBio, Inc. has announced that the FDA has approved Anktiva plus Bacillus CalmetteGuérin (BCG) for the treatment of patients with BCG unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors.

• Anktiva is a first-in-class IL-15 agonist immunotherapy for NMIBC and received Breakthrough Therapy Designation and approval from the FDA based on the safety and efficacy outcome of complete responses (CR) and duration of complete response (DOR).
• Bladder cancer is the 10th most commonly diagnosed cancer globally, and in the U.S., the American Cancer Society estimates there will be 83,190 new cases and 16,840 deaths from bladder cancer in 2024.
• With the approval of Anktiva in combination with BCG, NMIBC, patients who would otherwise face highly invasive surgery with life long consequences have an important new therapeutic option with a long-term durable complete response.

Source: ImmunityBio, Inc.

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FDA Approves Zevtera for Bacteremia, Skin and Skin Structure Infections, and Pneumonia

The FDA has approved Zevtera® injection (ceftobiprole medocaril sodium) for the treatment of adult patients with Staphylococcus aureus bloodstream infections, including those with:

o Right-sided infective endocarditis
o Adult patients with acute bacterial skin and skin structure infections (ABSSSI)
o Adult and pediatric patients (3 months to less than 18 years old) with community-acquired bacterial pneumonia (CABP).

• Ceftobiprole, the active moiety of the prodrug ceftobiprole medocaril, is an advanced generation cephalosporin antibiotic for intravenous administration, with rapid bactericidal activity against a wide range of Gram-positive bacteria.
• Zevtera is contraindicated in patients with a known history of severe hypersensitivity to Zevtera, or to other members of the cephalosporin class.

Source: Basilea Pharmaceutica Ltd

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FDA Approves Xromi Oral Solution for Use in Pediatric Patients with Sickle Cell Anemia

Xromi has been approved by the FDA as an oral solution formulation of hydroxyurea indicated to reduce the frequency of painful crises and reduce the need for blood transfusions in pediatric patients aged 6 months of age to less than 2 years with sickle cell anemia with recurrent moderate to severe painful crises.

• Sickle cell anemia is caused by an abnormal version of hemoglobin called hemoglobin S, which leads to sickle-shaped red blood cells that can form painful clumps inside the blood vessels. These painful episodes are called sickle cell crises and are one of the most common and distressing symptoms of sickle cell disease.
• Hydroxyurea has been shown to reduce the frequency of painful episodes associated with sickle cell disease. It is thought to work by increasing levels of hemoglobin F (also called fetal hemoglobin because it is present in newborn babies) to make the red blood cells bigger, rounder, more flexible, and less likely to turn into a sickle shape.
• Xromi, manufactured by Nova Laboratories, Ltd., is supplied as a strawberry flavored oral solution containing 100 mg/mL hydroxyurea. The Xromi package contains two oral dosing syringes.

Source: FDA

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FDA Approves Risvan (risperidone) for the Treatment of Schizophrenia

Laboratorios Farmacéuticos ROVI, S.A. has announced that the U.S. Food and Drug Administration (FDA) has authorized the marketing of Risvan® (Risperidone ISM®) for the treatment of schizophrenia in adults.

Risperidone ISM® is a prolonged-release injectable antipsychotic developed and patented by ROVI for the treatment of schizophrenia in adults, which, as of the first injection, provides immediate and sustained plasmatic drug levels and does not require loading doses or supplementation with oral risperidone.

Schizophrenia is a chronic, serious, and disabling mental disorder that affects around 1% of the world population. Schizophrenia patients are characterized by a mixture of symptoms, both positive (delusional ideas, hallucinations, disorganized language, and behavior) and negative (affective flattening, speech poverty, apathy) in nature. It is estimated that between 3% and 5% of total global healthcare expenditure is spent on schizophrenia.

Read more at Drugs.com…