Triheptanoin for long chain fatty acid oxidation disorders

Leriglitazone is expected to work in patients with ALD by activating receptors called ‘PPAR gamma’, found in the mitochondria, which are components within cells that generate energy. Leriglitazone will improve the ALD pathogenic cascade by improving mitochondrial dysfunction, reducing oxidative stress and neuroinflammation, promoting demyelination and halting axonal degeneration. Hence, leriglitazone is expected to protect cells from damage and slow the progression of the disease. If licensed, leriglitazone would offer the first drug treatment option for adult ALD patients with AMN who currently have no effective therapies available.

Pegunigalsidase alfa is produced by a method known as ‘recombinant DNA technology’: it is made by cells into which a gene (DNA) has been introduced, which makes them able to produce the enzyme. The replacement enzyme has also been modified to reduce the rate at which it is removed from the body, allowing it to act for longer. Pegunigalsidase alfa was designed to increase amount of medicine in the blood and reduce the ability of a substance to provoke an immune response, thereby enhancing efficacy compared with available products. If licensed, pegunigalsidase alfa would offer an additional first-line, long -term ERT options for adult and paediatric patients (>3 yr) diagnosed with FD.

Semaglutide is in clinical development for the treatment of overweight and obese individuals. Excess weight can place stress on both mental and physical health, leading a number of complications such as depression, low self-esteem, and increased risk of heart disease, stroke and type 2 diabetes. Weight can be affected by a number of factors such as diet, physical activity, genetics and general health conditions, however diet and exercise are the two main contributing factors. Being overweight is reversible through lifestyle changes such as increased exercise, healthy diet and a net calorie deficit, along with help through counselling and medication. If weight is not able to be controlled, surgery may be required. Therefore, there is an unmet need for pharmacological therapies that target both weight and glucose control.

Ozanimod, administered orally, is a new sphingosine 1-phosphate (S1P) receptor modulator. Treatment with S1P modulators is believed to work by interfering with signalling pathways that contribute to tissue inflammation. If licensed, ozanimod will offer an additional treatment option for adult patients with moderate to severe UC who have had an inadequate response, lost response to, or were intolerant to conventional therapy or a tumour necrosis factor-alpha (TNF) antagonist. In one phase II clinical trial, ozanimod at a daily dose of 1mg resulted in a slightly higher rate of clinical remission of ulcerative colitis than placebo.