An intranasal presentation of adrenaline (epinephrine) is now available for the emergency treatment of severe allergic reactions, including anaphylaxis. Neffy® is not currently listed on the Pharmaceutical Benefits Scheme (PBS), although an application has been made which is due for consideration in March.
Neffy® is formulated with dodecyl-beta-D-maltoside (DDM). This excipient functions as an absorption enhancer to promote systemic bioavailability. Studies demonstrate that Neffy® achieves adrenaline blood levels and cardiovascular effects (i.e. increased blood pressure and heart rate) within the range observed with approved adrenaline injection devices.
Real-world evidence from over 500 patients experiencing anaphylactic symptoms show a successful treatment rate of 89.2% with a single Neffy® dose. This is comparable to the rates reported in meta-analyses where around 90% of patients were successfully treated with a single intramuscular dose of adrenaline.
Practical guidance:
- Neffy® should be prescribed as part of an anaphylaxis action plan.
- There are two strengths available. The 1mg device is for patients from four years of age weighing between 15kg and 30kg; the 2mg device is for patients 30kg or more.
- Patients and carers must be instructed on how to use the device correctly. Placebo demonstrator devices can be obtained from Allergy & Anaphylaxis Australia.
- Each device is ready-to-use and contains only one dose. The nasal spray must not be primed before use.
- A second device may be used to deliver an additional dose if clinical improvement is absent or deterioration occurs after five minutes of the first dose.
- Patients should be encouraged to always carry two devices.
- Patients must seek emergency medical care after using the device.
Landiolol is a new ultra-short-acting beta-blocker for use in acute care settings. It is approved for the management of supraventricular tachycardia and for the rapid control of ventricular rate in atrial fibrillation or atrial flutter when short‑term rate control is required. It is also indicated for non‑compensatory sinus tachycardia when specific intervention is needed.
Landiolol is highly selective for β1 adrenoceptors with very low affinity for β2 receptors. This high degree of cardioselectivity minimises the impact on blood pressure and reduces the risk of bronchospasm compared with less selective beta-blockers. Landiolol has a very short half-life (~4 minutes) which supports precise dose titration and enables rapid recovery once the infusion is reduced or discontinued.
Hypotension is the most commonly observed adverse effect. This typically resolves quickly with fluid administration, dose reduction, or discontinuation. Continuous blood pressure and ECG monitoring are recommended for all patients receiving landiolol.
Vials contain 300mg landiolol hydrochloride (equivalent to 280mg landiolol). Dosing is based on the salt rather than the free base.
Ublituximab has recently been added to the Pharmaceutical Benefits Scheme (PBS) for the treatment of relapsing-remitting multiple sclerosis. Ublituximab is a monoclonal antibody that is administered as an intravenous infusion. The first two doses are given two weeks apart, with subsequent doses every 24 weeks.
The ULTIMATE I and II trials compared the efficacy of intravenous ublituximab (plus oral placebo) with oral teriflunomide (plus intravenous placebo). The primary end point was the annualised relapse rate (ARR). In the ULTIMATE I trial, ARR was 0.08 in the ublituximab group compared to 0.19 for the teriflunomide group. Similar results were seen in the ULTIMATE II trial, with an ARR of 0.09 in the ublituximab group and 0.18 in the teriflunomide group. However, no significant difference was observed in the risk of worsening of disability.
The most common adverse reactions are infections and infusion-related reactions (IRRs). Dosing is contraindicated in patients with severe active infection or severe immunocompromise. To minimise IRRs, a corticosteroid and antihistamine is administered prior to each infusion. An antipyretic may also be considered.
Although no cases of progressive multifocal leukoencephalopathy (PML) have been reported with ublituximab to date, its mechanism of action suggests a potential risk. Patients should be advised to immediately report any new or worsening neurological signs or symptoms to their doctor.
Odevixibat has recently been added to the Pharmaceutical Benefits Scheme (PBS) for the treatment of progressive familial intrahepatic cholestasis (PFIC). PFIC is a collection of rare genetic conditions of impaired bile formation and secretion. Signs and symptoms of cholestasis typically begin in infancy and can progress to liver failure.
Odevixibat acts locally in the distal ileum to inhibit the ileal bile acid transporter (IBAT). This reduces the reabsorption of bile acids, thereby increasing their clearance from the body.
The safety and efficacy of odevixibat was evaluated in a double-blind study of children with PFIC who had pruritus and elevated serum bile acids at baseline. Patients were randomly assigned to receive odevixibat (40 mcg/kg or 120 mcg/kg daily) or placebo for 24 weeks. Compared to placebo, odevixibat was associated with statistically significant improvements in pruritus. A serum bile acid response was observed in 33% of patients in the odevixibat group, while no patients met the response threshold in the placebo group. Data from the PEDFIC 2 extension study suggest these benefits are sustained with ongoing treatment.
Odevixibat is typically dosed once daily in the morning. The capsules may be swallowed whole, or the contents sprinkled on food. Odevixibat is minimally absorbed, and the most common adverse events are diarrhoea or frequent bowel movements.
The Therapeutic Goods Administration (TGA) has updated the product warnings for GLP-1 and dual GIP/GLP-1 receptor agonists due to a potential risk of suicidal thoughts. Glucagon-like peptide‑1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) are incretins that play important roles in glucose metabolism and energy homeostasis. Medications acting as agonists at GIP and GLP-1 receptors are used in the treatment of type 2 diabetes and weight management.
These medications include:
- Semaglutide;
- Liraglutide;
- Dulaglutide; and
- Tirzepatide.
A potential signal for self-harm and suicidality prompted investigations by the TGA and international regulatory agencies. As of September 2025, the TGA had received 85 such reports, including two reports of completed suicide. While there is insufficient evidence to support a causal link, the TGA notes the complex interaction between mental illness and the disorders for which these medicines are prescribed.
Use of GLP-1 agonists has increased significantly in recent years, particularly for weight loss. The TGA advises healthcare professionals to evaluate the benefits and risks of starting or continuing these medicines in patients with suicidal ideation or a history of suicide attempts. Patients should be monitored for any new or worsening mental health symptoms and encouraged to promptly report any concerning symptoms to their healthcare professional.
The subcutaneous formulation of ocrelizumab is now available on the Pharmaceutical Benefits Scheme (PBS) for relapsing-remitting multiple sclerosis (MS). While the intravenous infusion has been PBS listed since 2018, the subcutaneous option may improve the convenience and accessibility of therapy.
Ocrelizumab is a monoclonal antibody that targets CD20-expressing B cells. It reduces the number and function of these cells which are thought to play an important role in the initiation and progression of MS.
The pivotal OPERA I and OPERA II studies demonstrated the efficacy of ocrelizumab in relapsing MS. Patients were randomly assigned to receive intravenous ocrelizumab every 24 weeks or subcutaneous interferon beta-1a three times a week. The primary end point of annualised relapse rate was 46% and 47% lower for ocrelizumab in the respective studies. Pooled analyses showed a 40% risk reduction for confirmed disability progression at 12 weeks and 24 weeks, and a 33% relative increase in confirmed disability improvement at 12 weeks. The recently published OCARINA II study found subcutaneous ocrelizumab (920mg) to be non-inferior to intravenous ocrelizumab (600mg) for drug exposure, with comparable safety and efficacy.
The usual dose of subcutaneous ocrelizumab is 920mg/23mL injected in the abdomen over ten minutes. The subcutaneous formulation contains the permeation enhancer, vorhyaluronidase alfa. This enzyme hydrolyses hyaluronic acid in the subcutaneous tissue, temporarily increasing tissue permeability and allowing a larger volume to be injected.
Both the intravenous and subcutaneous formulations are administered every six months, although the first intravenous dose is split between two infusions. Premedication with a corticosteroid and an antihistamine reduces the risk of infusion-related reactions. An antipyretic, such as paracetamol, may also be considered.
The product information documents have been updated for denosumab medicines used in the treatment of osteoporosis. These revisions strengthen existing warnings regarding the risk of multiple vertebral fractures following treatment delay or discontinuation.
Bone metabolism is a dynamic process balancing activity between bone-forming osteoblasts and bone-resorbing osteoclasts. A key regulator of this process is the RANK/RANKL/OPG pathway. Denosumab binds to RANKL, preventing it from activating the RANK receptor. This inhibits osteoclast formation and activation, effectively reducing bone resorption.
However, accelerated bone turnover and rapid loss of bone mineral density can occur following discontinuation of denosumab. While the mechanism is not completely understood, denosumab may cause osteoclasts to fission into daughter cells, called osteomorphs. These cells accumulate during denosumab treatment and can quickly fuse to form functional osteoclasts once the inhibitory effect of denosumab is removed. This may explain the increased risk of multiple vertebral fractures, particularly in patients with a history of vertebral fracture.
Denosumab is administered subcutaneously every six months. Patients should be advised of the importance of adhering to the dosing schedule as dosing delays of just 30 days have been associated with a significant increase in fracture risk. If treatment must be stopped, Healthy Bones Australia recommends patients be started on a bisphosphonate within four weeks of the next scheduled denosumab dose.
The Pharmaceutical Benefits Scheme (PBS) listing for daratumumab has been extended to include first-line treatment of multiple myeloma. Patients must be ineligible for a primary stem cell transplant and treatment must form part of triple combination therapy that is limited to daratumumab, lenalidomide, and dexamethasone.
Daratumuab is a targeted immunotherapy that inhibits the growth of cells expressing the CD38 protein. This protein is overexpressed on the surface of cells in many haematological malignancies, including multiple myeloma. Studies have shown that daratumumab induces multiple myeloma cell death via several mechanisms, including complement-dependent cytotoxicity, antibody-dependent cell-mediated cytotoxicity, antibody-dependent cellular phagocytosis, and apoptosis.
A Phase 3 trial in patients with newly diagnosed multiple myeloma compared the safety and efficacy of daratumumab plus dexamethasone and lenalidomide to dexamethasone and lenalidomide alone. Median progression-free survival was significantly longer in the daratumumab group (44.5 months vs 17.5 months), and the risk of disease progression or death was 44% lower compared to the control group.
The most common serious adverse events reported were neutropenia, anaemia, lymphopenia, and pneumonia. Daratumumab may also cause infusion-related reactions, particularly with the first dose. Reactions can include nasal congestion, chills, and throat irritation. However, serious reactions, including anaphylaxis have been reported. Infusion-related reactions are more common with intravenous administration than subcutaneous.
Empagliflozin is available on the Pharmaceutical Benefits Scheme (PBS) for the treatment of type 2 diabetes, chronic heart failure, and chronic kidney disease (CKD). From 1 November 2025, the CKD listing expanded to cover patients with an eGFR of 20-90mL/min/1.73m2 prior to initiation.
Empagliflozin and dapagliflozin are sodium-glucose co-transporter 2 (SGLT2) inhibitors. While originally approved for the management of type 2 diabetes, studies have demonstrated that these medicines can also slow the progression of CKD regardless of diabetes status.
The primary effect of SGLT2 inhibitors is to inhibit the reabsorption of sodium and glucose in the proximal tubules. This produces glucosuria, natriuresis, and osmotic diuresis. Increased delivery of sodium to the distal tubules activates tubuloglomerular feedback mechanisms that lead to reduced intraglomerular pressure. This has been proposed as their mechanism of preserving renal function and reducing proteinuria, although additional processes may be involved.
A meta-analysis compared the safety and efficacy of SGLT-2 inhibitors, glucagon-like peptide-1 (GLP-1) agonists, and non-steroidal mineralocorticoid receptor antagonists (ns-MRA) in CKD. The authors concluded that all three medication classes are associated with significant reductions in cardiovascular and kidney disease risks in patients with CKD. However, SGLT-2 inhibitors may be the most attractive option when considering efficacy together with safety.
Empagliflozin is generally well tolerated. One of the safety concerns is the risk of ketoacidosis. The risk is minimal and largely limited to patients with diabetes. However, cases have been reported in patients without a diagnosis of diabetes. Clinical situations that may predispose to this event include acute intercurrent illness and surgery. Withholding SGLT2 inhibitors during these periods is recommended to prevent ketoacidosis in patients with diabetes. Evidence to guide perioperative dosing in patients without diabetes is currently lacking. Until further evidence is available, the Council of Australian Therapeutic Advisory Groups (CATAG) advise practitioners to refer to the Periprocedural Diabetic Ketoacidosis (DKA) with SGLT2 Inhibitor use in People with Diabetes.
The Therapeutic Goods Administration (TGA) has issued a safety update regarding the use of prilocaine/lidocaine cream (e.g. EMLA®) in infants. This follows two serious adverse events involving seizures and methaemoglobinaemia. Both cases occurred in neonates and are believed to have involved overdose.
Methaemoglobinaemia is a potentially life-threatening condition where methaemoglobin, the oxidised form of haemoglobin, accumulates in the blood. As this form of haemoglobin cannot bind oxygen, raised levels can lead to hypoxia.
Infants may be more likely to develop methaemoglobinaemia due to:
- A higher proportion of foetal haemoglobin, which is more readily oxidised to methaemoglobin compared to adult haemoglobin; and
- Lower activity of NADH cyb5r reductase, the enzyme responsible for reducing methaemoglobin back to haemoglobin.
The manufacturer advises against using EMLA® in pre-term infants with a gestational age of less than 37 weeks, and in infants up to 12 months of age who are receiving treatment with a methaemoglobin-inducing agent. Examples of methaemoglobin-inducing agents include dapsone, sulphonamides, other local anaesthetics, and nitrites and nitrates. EMLA® is also contraindicated in glucose-6-phosphate dehydrogenase deficiency and congenital or idiopathic methaemoglobinaemia as these patients have a higher risk of drug-induced methaemoglobinemia.
The EMLA® product label and package insert have been updated to emphasise the importance of adhering to the maximum recommended dose and application time. The TGA is also in the process of ensuring these updates are applied to generic products.
