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AZX-100

Ethylmorphine

It is metabolized in the liver by ethylmorphine-N-demethylase and used as an indicator of liver function. It is not marketed in the US but is approved for use in various countries around the world. … In the US it is a schedule II drug (single-entity) and schedule III drug (in combination products).

Influenza A virus A/Brisbane/02/2018 IVR-190 (H1N1) antigen (formaldehyde inactivated)

Inactivated virus cannot replicate, and therefore cannot cause disease from infection, even in immunocompromised individuals. … In contrast, live vaccines are produced from "wild-type" or disease-causing viruses that have been attenuated, or weakened, through various laboratory techniques.

Triheptanoin

[A214817] Triheptanoin was granted FDA approval on 30 June 2020.[L14612] … [A214812,A214817] Complications in lc-FAOD patients are reduced from approximately 60% to approximately 10% with the addition of triheptanoin.

Arimoclomol

[L51384] Arimoclomol was approved by the FDA on September 20, 2024, making it the first treatment for Niemann-Pick disease, type C (NPC). … [L51389] It is also being investigated in amyotrophic lateral sclerosis (ALS) [A264414, A264429] and inclusion body myositis.[A264419]

Belantamab mafodotin

[A216756] Belantamab mafodotin was granted FDA accelerated approval on 5 August 2020 for the treatment of multiple myeloma;[L15326] however, its manufacturer began the process for withdrawal of the … In the meantime, belantamab mafodotin will be available for patients in the Risk Evaluation and Mitigation Strategy (REMS) program who can enrol in a compassionate use program.

Floxuridine

It has been used to treat hepatic metastases of gastrointestinal adenocarcinomas and for palliation in malignant neoplasms of the liver and gastrointestinal tract. … An antineoplastic antimetabolite that is metabolized to fluorouracil when administered by rapid injection. Floxuridine is available as a sterile, nonpyrogenic, lyophilized powder for reconstitution.

Bezitramide

After cases of fatal overdose in the Netherlands in 2004 the drug was withdrawn from the market. Bezitramide has never been FDA approved and is currently a schedule II drug. … Bezitramide is a narcotic analgesic which was discovered in 1961, clinically tested around the 1970's [A14383], and marketed under the name Burgodin.

Aldoxorubicin

Aldoxorubicin, an antineoplastic agents, is an albumin-binding prodrug of doxorubicin.

Eladocagene exuparvovec

[L43642] Eladocagene exuparvovec received EMA approval in July 2022. … [A253687] It was subsequently approved by the FDA in November 2024.[L51898,L51893]