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DL-alpha tocopheryl acetate

Nelarabine

[A2332] Due to the rarity of these T-cell malignancies, nelarabine was first granted orphan drug status and a fast-track designation by the FDA to address the unmet therapeutic needs of these cancers. … [A2331,AA2334,A2335] Results from 2 phase 2 studies on adult and pediatric T-ALL/T-LBL indicated that nelarabine can yield a 13% complete response (CR) rate in pediatric patients and 18% in adult patients

Propylene glycol

A clear, colorless, viscous organic solvent and diluent used in pharmaceutical preparations.

Cholecystokinin

Cholecystokinin is one of the first gastrointestinal hormones discovered, identified more than 90 years ago due to its ability to stimulate gallbladder contraction in 1928. … This hormone has also been shown to have positive effects on enteric smooth muscle contraction and on nerve activity at multiple locations in the peripheral and central nervous system.

Prasugrel

FDA approved in 2009. … Prasugrel was developed by Daiichi Sankyo Co. and is currently marketed in the United States and Canada in cooperation with Eli Lilly and Company for acute coronary syndromes planned for percutaneous coronary

Zanubrutinib

[A187958] Due to this enhanced selectivity towards BTK, zanubrutinib belongs to the second-generation BTK inhibitor drug group that also includes [acalabrutinib], which was approved by the FDA in 2017. … Zanubrutinib was granted accelerated approval by the FDA in November 2019 based on clinical trial results that demonstrated an 84% overall response rate from zanubrutinib therapy in patients with MCL,[

Evinacumab

[A229273] Loss-of-function mutations in _ANGPTL3_ have been noted to result in hypolipidemia and subsequent reductions in cardiovascular risk, whereas increases in function appear to be associated with … Evinacumab is novel in its mechanism of action compared with other lipid-lowering therapies and therefore provides a unique and synergistic therapeutic option in the treatment of HoFH.

Tolvaptan

FDA approved on May 19, 2009.

Avapritinib

Avapritinib was granted FDA approval on 9 January 2020 [L40363] and EMA approval on 24 September 2020.[L41464]

Tocofersolan

D-Alpha-tocopheryl polyethylene glycol 1000 succinate (Tocofersolan, Vedrop), has been developed in Europe as an orally bioavailable source of vitamin E in children suffering from cholestasis [L2371]. … Tocofersolan is an oral treatment of vitamin E deficiency due to digestive malabsorption in pediatric patients with congenital chronic cholestasis or hereditary chronic cholestasis.