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Olezarsen

Olezarsen is an antisense oligonucleotide that targets the messenger RNA (mRNA) for apolipoprotein C-III (APOC3, apoC-III),[L52033] which is a key regulator of triglyceride levels in plasma, in the liver … [L52033] On December 19, 2024, olezarsen was approved by the FDA for the treatment of familial chylomicronemia syndrome.[L52028] It is also being investigated for the treatment of hyperlipidemia.

Hexaminolevulinate

Hexaminolevulinate is an optical imaging drug. In solution form it is instilled intravesically for use with photodynamic blue light cystoscopy as an adjunct to white light cystoscopy. … Hexaminolevulinate is manufactured under the brand Cysview® by Photocure ASA. In Europe, Hexaminolevulinate is marketed under the brand Hexvix®.

Chlorpropamide

Up to 80% of the single oral dose of chlorpropramide is metabolized, likely in the liver; 80-90% of the dose is excreted in urine as unchanged drug and metabolites. … Medications in this class differ in their dose, rate of absorption, duration of action, route of elimination and binding site on their target pancreatic β cell receptor.

Hydralazine

[A186841] Hydralazine is a hydrazine derivative vasodilator used alone or as adjunct therapy in the treatment of hypertension and only as adjunct therapy in the treatment of heart failure. … Originally developed in the 1950s as a malaria treatment, hydralazine showed antihypertensive ability and was soon repurposed.

(+)-menthol

Vanzacaftor

It is used alongside other CFTR correctors and CFTR potentiators to increase the quantity and function of CFTR at the cell surface in patients with cystic fibrosis. … [L52275,A179677] Vanzacaftor was first approved by the US FDA in December 2024 in combination with another CFTR corrector - [tezacaftor], which binds to a different site than vanzacaftor - and [deutivacaftor

Gefapixant

It received approval in both Japan and Switzerland in 2022 for the treatment of adult patients with RCC and UCC, and received subsequent approval in the EU in September 2023 for the same indications. … [L48596,L48591] It is the first therapy to be approved for the treatment of RCC or UCC in the EU.[L48601]

Tafenoquine

[A35671, A35690] It was discovered by the scientists at the Walter Reed Army Institute of Research in 1978 as a substitute for primaquine that would be more effective against relapsing vivax malaria. … [A35677] It was FDA approved on July 20, 2018.[L3770]

Vamorolone

[L48671,L48676] In December 2023, it was approved in the EU for the treatment of patients ≥4 years of age.[L49505] … [L48676] Vamorolone was approved by the FDA in October 2023 for the management of DMD in patients ≥2 years of age.

Cerliponase alfa

[L51329] CLN2 is a predominantly pediatric-onset neurodegenerative disease caused by a deficiency in the lysosomal enzyme TPP1, leading to progressive impairment in motor and cognitive function. … [A264354, A264369] As a recombinant human TPP1, cerliponase alfa is used as an enzyme replacement therapy to restore the levels of TPP1 in patients with CLN2.[L51339]