FDA Updates Archives - Clinic Hours

FDA approves WAYRILZ (Rilzabrutinib) for chronic ITP in adults

The U.S. FDA approved WAYRILZ (rilzabrutinib) tablets for adults with persistent or chronic immune thrombocytopenia (ITP) who have not responded to previous treatments.

MOA: kinase inhibitor

Dosage: 400 mg BD PO

Key Safety Precautions:

Infections: Monitor closely
Liver toxicity: Check bilirubin and liver enzymes
Pregnancy: May cause fetal harm; use contraception
Drug interactions: Avoid CYP3A inhibitors/inducers, PPIs; take 2 hours before antacids or H2 blockers
Organ impairment: Not for moderate/severe liver or severe kidney disease

Side Effects: Most common: diarrhea, nausea, headache, abdominal pain, and COVID-19 infection.

Reference: FDA

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FDA approves first generic Liraglutide for obesity treatment

The U.S. FDA approved Teva Pharmaceuticals’ generic version of liraglutide (Saxenda®), the first generic GLP-1 receptor agonist for chronic weight management.

Indication: It is indicated for adults with obesity (BMI ≥30) or overweight (BMI ≥27 with comorbidity) and for adolescents aged 12–17 years with obesity.

Dose: 0.6 mg once daily SC, titrated weekly to a maintenance dose of 3 mg SC daily.

Mechanism: Liraglutide mimics GLP-1, enhancing insulin secretion, slowing gastric emptying, promoting satiety, and reducing appetite.

Common side effects: nausea, vomiting, diarrhea, headache, and injection-site reactions. Serious risks include pancreatitis, gallbladder disease, and thyroid C-cell tumors.

This approval is expected to lower treatment costs, compared to the branded Saxenda (~$1,350/month), and broaden access to effective obesity therapy.

Reference: FDA.gov

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FDA approves Papzimeos (zopapogene imadenovec-drba) for recurrent respiratory papillomatosis

Papzimeos is the first and only FDA-approved therapy for the treatment of adults with RRP.

Introduction: PAPZIMEOS™ is a non-replicating adenoviral vector-based immunotherapy indicated for the treatment of adults with recurrent respiratory papillomatosis.

Dosage: 5×10¹¹ particle units (PU) per injection, given SC 4 times over 12 weeks.

Usage: Prior to first dose, surgical debulking of visible papillomas is recommended to minimize residual disease. Supplied as a single-dose vial containing 5×10¹¹ PU in 1 mL suspension.

Contraindications: None.

Warnings & Precautions: Injection-site reactions Monitor patients for at least 30 minutes after Rx. Thrombotic events.

Adverse reactions: Most common (≥5%): injection-site reactions, fatigue, chills, fever, muscle pain, and nausea.

Reference: FDA

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FDA approves novel drug for resistant hypertension based on PRECISION trial

The USA FDA has approved Aprocitentan (Brand name TRYVIO) for the treatment of resistant hypertension in combination with other antihypertensive drugs.

Dose: 12.5 mg 1OD PO.

MOA: Dual endothelin receptor antagonist.

Contraindications: Pregnancy, Hypersensitivity.

Adverse effects: Edema, anemia, ⬇️ sperm count.

Warning & precautions: ERAs cause hepatotoxicity & liver failure.

Trial: PRECISION was a multicentre, blinded, randomised, parallel-group, phase 3 studydone in Europe, North America, Asia & Australia in June 18, 2018, to April 25, 2022 by Prof Markus P Schlaich, MD et al.

Inclusion criteria: Patients with SBP > 140 mmhg who were on 3 different anti hypertensive medications including diuretics.

Objectives: The primary endpoints were changes in SBP from baseline to 4 week & from withdrawal baseline to 40 week. Secondary endpoints included 24-h ambulatory blood pressure changes.

Methods: Study consisted of 3 sequential parts:

Part 1 was 4-week double-blind, randomised, & placebo-controlled part, in which patients received aprocitentan 12·5 mg, 25 mg, or placebo in a 1:1:1 ratio.

Part 2 was a 32-week single patient blind part, in which all patients received aprocitentan 25 mg.

Part 3 was a 12-week double-blind, randomised, placebo-controlled withdrawal part, in which patients were re-randomised to aprocitentan 25 mg or placebo in a 1:1 ratio.

Findings: In patients with resistant hypertension, aprocitentan was well tolerated & superior to placebo in lowering blood pressure at 4 week with a sustained effect at 40 week.

Reference: http://clinicaltrials.gov/show/NCT03541174

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FDA approves Nivolumab for resected stage IIB/C melanoma

The US FDA has approved nivolumab for the treatment of completely resected stage IIB/C melanoma for patients aged 12 years and older, expanding the melanoma indication for the programmed death receptor-1 (PD-1) inhibitor.

Dose – For patients >40 kg, 480 mg every 4 weeks or 240 mg every 2 weeks until disease recurrence or unacceptable toxicity for up to 1 year. For pediatric patients < 40 kg, 3 mg/kg every 2 weeks or 6 mg/kg every 4 weeks until disease recurrence or unacceptable toxicity for up to 1 year.

Adverse reactions – fatigue, musculoskeletal pain, rash, diarrhea & pruritis.

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FDA approves Talquetamab for multiple myeloma

The USA FDA approves Talquetamab, a first-in-class bispecific antibody targeting the GPRC5D receptor, for heavily pretreated adults with relapsed or refractory multiple myeloma.

Indication for usage: Patients must have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

Dose: Both the weekly 0.4 mg/kg and biweekly 0.8 mg/kg doses are recommended. Check out the full regimens.

Adverse effects: CRS, dysgeusia, musculoskeletal pain, skin disorder, rash, fatigue, decreased weight, dry mouth, pyrexia, xerosis, dysphagia, upper respiratory tract infection & diarrhea.

Talquetamab was evaluated in the single-arm, open-label MonumenTAL-1 study of 187 patients who had previously been treated with at least four prior systemic therapies.

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FDA approves Sotagliflozin for heart failure

Sotagliflozin, a novel agent that inhibits sodium-glucose co transporter SGLT 1 & SGLT2 both as a treatment for adults with heart failure with or without diabetes and for T1DM & T2DM.

Dose: 200mg/400mg 1 OD.

MOA: Inhibiting SGLT2 reduces renal reabsorption of glucose and sodium which may influence several physiological functions, such as lowering both pre-and afterload of the heart and downregulating sympathetic activity. Inhibiting SGLT1 reduces intestinal absorption of glucose and sodium which likely contributes to diarrhea.

Adverse Effects: >10% UTI, Diarrhea, Hypoglycemia, Dizziness, Genital mycotic infection.

Other approved drugs are SGLT2 inhibitors empagliflozin & dapagliflozin , both of which now have labeled indications across the spectrum of LVEF.

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FDA approves Inj Buprenorphine for opioid use disorder

The US FDA has approved extended-release injection buprenorphine (Brixadi, Braeburn Inc) for the treatment of moderate to severe opioid use disorder (OUD). The medication comes in two formulations: a weekly and a monthly version. The adverse reactions include headache, constipation, nausea, injection-site erythema, injection-site pruritus, insomnia & UTI.

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FDA approves Dabrafenib with Trametinib for pediatric gliomas

The US FDA has approved dabrafenib with trametinib for children aged 1 year or older who need systemic treatment for low-grade gliomas that have a BRAF V600E mutation. Dabrafenib/trametinib is the first systemic therapy approved for frontline treatment of low-grade, BRAF-mutated pediatric gliomas, the FDA said. Dabrafenib was given orally twice daily, and trametinib was given orally once daily. Children in the chemotherapy arm received a 10-week induction course followed by eight 6-week maintenance cycles.

Adverse effects: pyrexia (66%), rash (54%), headache (40%), vomiting (38%), musculoskeletal pain (36%), fatigue (31%), dry skin (31%), diarrhea (30%), nausea (26%), epistaxis and other bleeding events (25%), abdominal pain (24%), and dermatitis acneiform (23%). The more common grade 3 or 4 laboratory abnormalities were decreased neutrophil count (20%) and increases in alanine aminotransferase (3.1%) and aspartate aminotransferase levels (3.1%).

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FDA approves new drug for Hemophilia A

The US FDA has approved efanesoctocog alfa (Altuviiio), a first-in-class, high-sustained factor VIII replacement therapy for adults and children with hemophilia A. The product is used once a week and is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as to control bleeding during surgery (perioperative management).